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In gestational trophoblastic disease (GTD), a tumor develops inside the uterus from tissue that forms after conception (the joining of sperm and egg). This tissue is made of trophoblast cells and normally surrounds the fertilized egg in the uterus. Trophoblast cells help connect the fertilized egg to the wall of the uterus and form part of the placenta (the organ that passes nutrients from the mother to the fetus). Sometimes there is a problem with the fertilized egg and trophoblast cells. Instead of a healthy fetus developing, a tumor forms. Until there are signs or symptoms of the tumor, the pregnancy will seem like a normal pregnancy. Most GTD is benign (not cancer) and does not spread, but some types become malignant (cancer) and spread to nearby tissues or distant parts of the body. Gestational trophoblastic disease (GTD) is a general term that includes different types of disease: HMs are slow-growing tumors that look like sacs of fluid. An HM is also called a molar pregnancy. The cause of hydatidiform moles is not known. HMs may be complete or partial: Most hydatidiform moles are benign, but they sometimes become cancer. Having one or more of the following risk factors increases the risk that a hydatidiform mole will become cancer: Gestational trophoblastic neoplasia (GTN) includes the following: Invasive moles are made up of trophoblast cells that grow into the muscle layer of the uterus. Invasive moles are more likely to grow and spread than a hydatidiform mole. Rarely, a complete or partial HM may become an invasive mole. Sometimes an invasive mole will disappear without treatment. A choriocarcinoma is a malignant tumor that forms from trophoblast cells and spreads to the muscle layer of the uterus and nearby blood vessels. It may also spread to other parts of the body, such as the brain, lungs, liver, kidney, spleen, intestines, pelvis, or vagina. A choriocarcinoma is more likely to form in women who have had any of the following: A placental-site trophoblastic tumor (PSTT) is a rare type of gestational trophoblastic neoplasia that forms where the placenta attaches to the uterus. The tumor forms from trophoblast cells and spreads into the muscle of the uterus and into blood vessels. It may also spread to the lungs, pelvis, or lymph nodes. A PSTT grows very slowly and signs or symptoms may appear months or years after a normal pregnancy. An epithelioid trophoblastic tumor (ETT) is a very rare type of gestational trophoblastic neoplasia that may be benign or malignant. When the tumor is malignant, it may spread to the lungs. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk to your doctor if you think you may be at risk. Risk factors for GTD include the following: These and other signs and symptoms may be caused by gestational trophoblastic disease or by other conditions. Check with your doctor if you have any of the following: GTD sometimes causes an overactive thyroid. Signs and symptoms of an overactive thyroid include the following: The following tests and procedures may be used: Gestational trophoblastic disease usually can be cured. Treatment and prognosis depend on the following: Treatment options also depend on whether the woman wishes to become pregnant in the future. The process used to find out the extent or spread of cancer is called staging, The information gathered from the staging process helps determine the stage of disease. For gestational trophoblastic neoplasia (GTN), stage is one of the factors used to plan treatment. The following tests and procedures may be done to help find out the stage of the disease: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if choriocarcinoma spreads to the lung, the cancer cells in the lung are actually choriocarcinoma cells. The disease is metastatic choriocarcinoma, not lung cancer. Hydatidiform moles (HM) are found in the uterus only and do not spread to other parts of the body. In stage I, the tumor is in the uterus only. In stage II, the tumor has spread beyond the uterus to the ovary, fallopian tube, vagina, and/or the connective tissues around the uterus. In stage III, the tumor has spread to the lung, with or without spread to the ovary, fallopian tube, vagina, and/or the connective tissues around the uterus. In stage IV, the tumor has spread to distant parts of the body other than the lungs. Invasive moles and choriocarcinomas are treated based on risk groups. The stage of the invasive mole or choriocarcinoma is one factor used to determine risk group. Other factors include the following: There are two risk groups for invasive moles and choriocarcinomas: low risk and high risk. Patients with low-risk disease usually receive less aggressive treatment than patients with high-risk disease. Placental-site trophoblastic tumor (PSTT) and epithelioid trophoblastic tumor (ETT) treatments depend on the stage of disease. Recurrent gestational trophoblastic neoplasia (GTN) is cancer that has recurred (come back) after it has been treated. The cancer may come back in the uterus or in other parts of the body. Gestational trophoblastic neoplasia that does not respond to treatment is called resistant GTN. Different types of treatment are available for patients with gestational trophoblastic disease. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Before starting treatment, patients may want to think about taking part in a clinical trial. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. Choosing the most appropriate cancer treatment is a decision that ideally involves the patient, family, and health care team. The doctor may remove the cancer using one of the following operations: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated, or whether the tumor is low-risk or high-risk. Combination chemotherapy is treatment using more than one anticancer drug. See Drugs Approved for Gestational Trophoblastic Disease for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of gestational trophoblastic disease being treated. External radiation therapy is used to treat gestational trophoblastic disease. Information about ongoing clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Blood levels of beta human chorionic gonadotropin (beta-hCG) will be checked for up to 6 months after treatment has ended. This is because a beta-hCG level that is higher than normal may mean that the tumor has not responded to treatment or it has become cancer. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of a hydatidiform mole may include the following: After surgery, beta human chorionic gonadotropin (beta-hCG) blood tests are done every week until the beta-hCG level returns to normal. Patients also have follow-up doctor visits monthly for up to 6 months. If the level of beta-hCG does not return to normal or increases, it may mean the hydatidiform mole was not completely removed and it has become cancer. Pregnancy causes beta-hCG levels to increase, so your doctor will ask you not to become pregnant until follow-up is finished. For disease that remains after surgery, treatment is usually chemotherapy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of low-risk gestational trophoblastic neoplasia (GTN) (invasive mole or choriocarcinoma) may include the following: If the level of beta-hCG in the blood does not return to normal or the tumor spreads to distant parts of the body, chemotherapy regimens used for high-risk metastatic GTN are given. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of high-risk metastatic gestational trophoblastic neoplasia (invasive mole or choriocarcinoma) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of stage I placental-site gestational trophoblastic tumors and epithelioid trophoblastic tumors may include the following: Treatment of stage II placental-site gestational trophoblastic tumors and epithelioid trophoblastic tumors may include the following: Treatment of stage III and IV placental-site gestational trophoblastic tumors and epithelioid trophoblastic tumors may include following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of recurrent or resistant gestational trophoblastic tumor may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about gestational trophoblastic tumors and neoplasia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of gestational trophoblastic disease. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Gestational Trophoblastic Disease Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/gestational-trophoblastic/patient/gtd-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389509] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Gestational Trophoblastic Disease Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? 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what research (or clinical trials) is being done for Gestational Trophoblastic Disease ?

New types of treatment are being tested in clinical trials. Information about ongoing clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

These tumors may be benign (not cancer) or malignant (cancer). There are many types of vascular tumors. The most common type of childhood vascular tumor is infantile hemangioma, which is a benign tumor that usually goes away on its own. Because malignant vascular tumors are rare in children, there is not a lot of information about what treatment works best. The following tests and procedures may be used: Benign tumors are not cancer. This summary has information about the following benign vascular tumors: Intermediate tumors that are locally aggressive often spread to the area around the tumor. This summary has information about the following locally aggressive vascular tumors: Intermediate (rarely metastasizing) tumors sometimes spread to other parts of the body. This summary has information about the following vascular tumors that rarely metastasize: Malignant tumors are cancer. This summary has information about the following malignant vascular tumors: Different types of treatment are available for children with vascular tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because vascular tumors in children are rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with cancer and who specialize in certain areas of medicine. These may include the following specialists: Beta-blockers are drugs that decrease blood pressure and heart rate. When used in patients with vascular tumors, beta-blockers may help shrink the tumors. Beta-blocker therapy may be given by vein (IV), by mouth, or placed on the skin (topical). The way the beta-blocker therapy is given depends on the type of vascular tumor and where the tumor first formed. The beta-blocker propranolol is usually the first treatment for hemangiomas. Infants younger than 4 weeks, or who have an underlying condition, or who are treated with IV propranolol may need to have their treatment started in a hospital. Propranolol is also used to treat benign vascular tumor of liver and kaposiform hemangioendothelioma. Other beta-blockers used to treat vascular tumors include atenolol, nadolol, and timolol. Infantile hemangioma may also be treated with propranolol and steroid therapy or propranolol and topical beta-blocker therapy. See the drug information summary on Propranolol Hydrochloride for more information. The following types of surgery may be used to remove many types of vascular tumors: The type of surgery used depends on the type of vascular tumor and where the tumor formed in the body. For malignant tumors, after the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Photocoagulation is the use of an intense beam of light, such as a laser, to seal off blood vessels or destroy tissue. It is used to treat pyogenic granuloma. Cryotherapy is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as abnormal blood vessels in pyogenic granuloma. This type of treatment is also called cryosurgery. For more information, see Cryosurgery to Treat Cancer. Embolization is a procedure that uses particles, such as tiny gelatin sponges or beads, to block blood vessels in the liver. It may be used to treat some benign vascular tumors of the liver and kaposiform hemangioendothelioma. Chemotherapy is a treatment that uses drugs to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach tumor cells throughout the body. Sometimes more than one anticancer drug is given. This is called combination chemotherapy. Sclerotherapy is a treatment used to destroy the blood vessel that leads to the tumor and the tumor. A liquid is injected into the blood vessel, causing it to scar and break down. Over time, the destroyed blood vessel is absorbed into normal tissue. The blood flows through nearby healthy veins instead. Sclerotherapy is used in the treatment of epithelioid hemangioma. Radiation therapy is a treatment that uses high-energy x-rays or other types of radiation to kill tumor cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with the tumor. It is used to treat some vascular tumors. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific tumor cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Different types of targeted therapy are being used or studied to treat childhood vascular tumors: Immunotherapy is a treatment that uses the patient’s immune system to fight disease. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against disease. This tumor treatment is a type of biologic therapy. The following types of immunotherapy are being used in the treatment of childhood vascular tumors: There are two types of immune checkpoint inhibitor therapy: Other drugs used to treat childhood vascular tumors or manage their effects include the following: Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Some treatments, such as chemotherapy and radiation therapy, cause side effects that continue or appear months or years after treatment has ended. These are called late effects. Late effects of treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the possible late effects caused by some treatments. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.) For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the research process. Clinical trials are done to find out if new treatments are safe and effective or better than the standard treatment. Many of today's standard treatments are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way disease will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose tumors have not gotten better. There are also clinical trials that test new ways to stop tumors from recurring (coming back) or reduce the side effects of treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the vascular tumor may be repeated. Some tests will be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the tumor has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Infantile hemangiomas are the most common type of benign vascular tumor in children. Infantile hemangiomas form when immature cells that are meant to form blood vessels form a tumor instead. An infantile hemangioma may also be called a "strawberry mark." These tumors are not usually seen at birth but appear when the infant is 3 to 6 weeks old. Most hemangiomas get bigger for about 5 months and then stop growing. The hemangiomas slowly fade away over the next several years, but a red mark or loose or wrinkled skin may remain. It is rare for an infantile hemangioma to come back after it has faded away. Infantile hemangiomas may be on the skin, in the tissue below the skin, and/or in an organ. They are usually on the head and neck but can be anywhere on or in the body. Hemangiomas may appear as a single lesion, one or more lesions spread over a larger area of the body, or multiple lesions in more than one part of the body. Lesions that are spread over a larger area of the body or multiple lesions are more likely to cause problems. Infantile hemangioma with minimal or arrested growth (IH-MAG) is a certain type of infantile hemangioma that is seen at birth and does not tend to get bigger. The lesion appears as light and dark areas of redness in the skin. The lesions are usually on the lower body but can be on the head and neck. Hemangiomas of this type go away over time without treatment. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Infantile hemangiomas are more common in the following: Other risk factors for infantile hemangiomas include the following: Having more than one hemangioma or an airway or ophthalmic hemangioma increases the risk of having other health problems. Signs and Symptoms Infantile hemangiomas may cause any of the following signs and symptoms. Check with your child’s doctor if your child has any of the following: Although most infantile hemangiomas are nothing to worry about, if your child develops any lumps or red or blue marks on the skin check with your child's doctor. He or she can refer the child to a specialist if needed. Diagnostic Tests A physical exam and history are usually all that are needed to diagnose infantile hemangiomas. If there is something about the tumor that looks unusual, a biopsy may be done. If the hemangioma is deeper inside the body with no change to the skin, or the lesions are spread across a large area of the body, an ultrasound may be done. Infants with five or more hemangiomas on the skin should have an ultrasound of the liver done to check for a liver hemangioma. See the General Information section for a description of these tests and procedures. If the hemangiomas are part of a syndrome, more tests may be done, such as an echocardiogram, MRI, magnetic resonance angiogram, and eye exam. Treatment Most hemangiomas fade and shrink without treatment. If the hemangioma is large or causing other health problems, treatment may include the following: Congenital hemangioma is a benign vascular tumor that begins forming before birth and is fully formed when the baby is born. They're usually on the skin but can be in another organ. A congenital hemangioma may occur as a rash of purple spots and the skin around the spot may be lighter. There are three types of congenital hemangiomas: Diagnostic Tests See the General Information section for a description of tests and procedures, such as ultrasound, used to diagnose congenital hemangioma. Treatment Treatment of rapidly involuting congenital hemangioma and partial involuting congenital hemangioma may include the following: Treatment of non-involuting congenital hemangioma may include the following: Benign vascular tumors of the liver may be focal vascular lesions (a single lesion in one area of the liver), multiple liver lesions (more than one lesion in one area of the liver), or diffuse liver lesions (more than one lesion in more than one area of the liver). The liver has many functions, including filtering blood and making proteins needed for blood clotting. Sometimes, blood that normally flows through the liver is blocked or slowed by the tumor. This sends blood directly to the heart without going through the liver and is called a liver shunt. This can cause heart failure and problems with blood clotting. Focal Vascular Lesions Focal vascular lesions are usually rapidly involuting congenital hemangiomas or non-involuting congenital hemangiomas. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose focal vascular lesions of the liver. Treatment Treatment of focal vascular lesions of the liver depends on whether symptoms occur and may include the following: Multiple and Diffuse Liver Lesions Multifocal and diffuse lesions of the liver are usually infantile hemangiomas. Diffuse lesions of the liver can cause serious effects, including problems with the thyroid gland and heart. The liver can enlarge, press on other organs, and cause more symptoms. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose multifocal or diffuse benign vascular lesions. Treatment Treatment of multifocal liver lesions may include the following: Treatment of diffuse liver lesions may include the following: If a vascular lesion of the liver does not respond to standard treatments, a biopsy may be done to see if the tumor has become malignant. Spindle cell hemangiomas contain cells called spindle cells. Under a microscope, spindle cells look long and slender. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Spindle cell hemangiomas are likely to occur in children with the following syndromes: Signs Spindle cell hemangiomas appear on or under the skin. They are painful red-brown or bluish lesions that usually appear on the arms or legs. They can begin as one lesion and develop into more lesions over years. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose spindle cell hemangioma. Treatment There is no standard treatment for spindle cell hemangiomas. Treatment may include the following: Spindle cell hemangiomas may come back after surgery. Epithelioid hemangiomas usually form on or in the skin, especially the head, but can occur in other areas, such as bone. Signs and Symptoms Epithelioid hemangiomas are sometimes caused by injury. On the skin, they may appear as firm pink to red bumps and may be itchy. Epithelioid hemangioma of the bone may cause swelling, pain, and weakened bone in the affected area. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose epithelioid hemangioma. Treatment There is no standard treatment for epithelioid hemangiomas. Treatment may include the following: Epithelioid hemangiomas often come back after treatment. Pyogenic granuloma is also called lobular capillary hemangioma. It is most common in older children and young adults but may occur at any age. The lesions are sometimes caused by injury or from the use of certain medicines, including birth control pills and retinoids. They may also form for no known reason inside capillaries (the smallest blood vessels), arteries, veins, or other places on the body. Some lesions may be associated with capillary malformations. Usually there is only one lesion, but sometimes multiple lesions occur in the same area or the lesions may spread to other areas of the body. Signs Pyogenic granulomas are raised, bright red lesions that may be small or large and smooth or bumpy. They grow quickly over weeks to months and may bleed a lot. These lesions are usually on the surface of the skin, but may form in the tissues below the skin and look like other vascular lesions. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose pyogenic granuloma. Treatment Some pyogenic granulomas go away without treatment. Other pyogenic granulomas need treatment that may include the following: Pyogenic granulomas often come back after treatment. Angiofibromas are rare. They are benign skin lesions that usually occur with a condition called tuberous sclerosis (an inherited disorder that causes skin lesions, seizures, and mental disabilities). Signs Angiofibromas appear as red bumps on the face. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose angiofibroma. Treatment Treatment of angiofibromas may include the following: Juvenile nasopharyngeal angiofibromas are benign tumors but they can grow into nearby tissue. They begin in the nasal cavity and may spread to the nasopharynx, the paranasal sinuses, the bone around the eyes, and sometimes to the brain. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose juvenile nasopharyngeal angiofibroma. Treatment Treatment of juvenile nasopharyngeal angiofibromas may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Kaposiform hemangioendotheliomas and tufted angiomas are blood vessel tumors that occur in infants or young children. These tumors can cause Kasabach-Merritt phenomenon, a condition in which the blood is not able to clot and serious bleeding may occur. In Kasabach-Merritt phenomenon, the tumor traps and destroys platelets (blood-clotting cells). Then there aren't enough platelets in the blood when needed to stop bleeding. This type of vascular tumor is not related to Kaposi sarcoma. Signs and Symptoms Kaposiform hemangioendotheliomas and tufted angiomas usually occur on the skin of the arms and legs, but may also form in deeper tissues, such as muscle or bone, or in the chest, abdomen, head, or neck. Signs and symptoms may include the following: Patients who have kaposiform hemangioendothelioma and tufted angioma may have anemia (weakness, feeling tired, or looking pale). Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose kaposiform hemangioendothelioma and tufted angioma. If a physical exam and MRI clearly show the tumor is a kaposiform hemangioendothelioma or a tufted angioma, a biopsy may not be needed. A biopsy is not always done because serious bleeding can occur. Treatment Treatment of kaposiform hemangioendotheliomas and tufted angiomas depends on the child's symptoms. Infection, delay in treatment, and surgery can cause bleeding that is life-threatening. Kaposiform hemangioendotheliomas and tufted angiomas are best treated by a vascular anomaly specialist. Treatment for uncomplicated kaposiform hemangioendotheliomas and tufted angiomas may include the following: Treatment for complicated kaposiform hemangioendotheliomas and tufted angiomas may include the following: Even with treatment, these tumors do not fully go away and can come back. Pain and inflammation may get worse with age, often around the time of puberty. Long-term effects include chronic pain, heart failure, bone problems, and lymphedema (the build up of lymph fluid in tissues). For information about the treatments listed below, see the Treatment Option Overview section. Pseudomyogenic hemangioendothelioma can occur in children, but is most common in men aged 20 to 50 years. These tumors are rare, and usually occur on or under the skin, or in bone. They may spread to nearby tissue but usually do not spread to other parts of the body. In most cases, there are multiple tumors. Signs and Symptoms Pseudomyogenic hemangioendotheliomas may appear as a lump in soft tissue or may cause pain in the affected area. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose pseudomyogenic hemangioendothelioma. Treatment Treatment of pseudomyogenic hemangioendotheliomas may include the following: Because pseudomyogenic hemangioendothelioma is so rare in children, treatment options are based on clinical trials in adults. Retiform hemangioendotheliomas are slow growing, flat tumors that occur in young adults and sometimes children. These tumors usually occur on or under the skin of the arms, legs, and trunk. These tumors usually do not spread to other parts of the body. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose retiform hemangioendothelioma. Treatment Treatment of retiform hemangioendotheliomas may include the following: Retiform hemangioendothelioma may come back after treatment. Papillary intralymphatic angioendotheliomas are also called Dabska tumors. These tumors form in or under the skin anywhere on the body. Lymph nodes are sometimes affected. Signs Papillary intralymphatic angioendotheliomas may appear as firm, raised, purplish bumps, which may be small or large. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose papillary intralymphatic angioendothelioma. Treatment Treatment of papillary intralymphatic angioendotheliomas may include the following: Composite hemangioendotheliomas have features of both benign and malignant vascular tumors. These tumors usually occur on or under the skin on the arms or legs. They may also occur on the head, neck, or chest. Composite hemangioendotheliomas are not likely to metastasize (spread) but they may come back in the same place. When the tumors metastasize, they usually spread to nearby lymph nodes. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose composite hemangioendothelioma and find out whether the tumor has spread. Treatment Treatment of composite hemangioendotheliomas may include the following: Kaposi sarcoma is a cancer that causes lesions to grow in the skin; the mucous membranes lining the mouth, nose, and throat; lymph nodes; or other organs. It is caused by the Kaposi sarcoma herpes virus (KSHV). In the United States, it occurs most often in children who have a weak immune system caused by rare immune system disorders, HIV infection, or drugs used in organ transplants. Signs Signs in children may include the following: Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose Kaposi sarcoma. Treatment Treatment of Kaposi sarcoma may include the following: Because Kaposi sarcoma is so rare in children, some treatment options are based on clinical trials in adults. See the PDQ summary on Kaposi Sarcoma Treatment for information about Kaposi sarcoma in adults. For information about the treatments listed below, see the Treatment Option Overview section. Epithelioid hemangioendotheliomas can occur in children, but are most common in adults aged 30 to 50 years. They usually occur in the liver, lung, or in bone. They may be fast growing or slow growing. In about a third of cases, the tumor spreads to other parts of the body very quickly. Signs and Symptoms Signs and symptoms depend on where the tumor is in the body. Check with your child's doctor if your child has any of the following: Tumors that occur in the liver or soft tissue may also cause signs and symptoms. Diagnostic Tests Epithelioid hemangioendotheliomas in the liver are found with CT scans and MRI scans. See the General Information section for a description of these tests and procedures used to diagnose epithelioid hemangioendothelioma and find out whether the tumor has spread. X-rays may also be done. Treatment Treatment of slow-growing epithelioid hemangioendotheliomas includes the following: Treatment of fast-growing epithelioid hemangioendotheliomas may include the following: Angiosarcomas are fast-growing tumors that form in blood vessels or lymph vessels in any part of the body, usually in soft tissue. Most angiosarcomas are in or near the skin. Those in deeper soft tissue can form in the liver, spleen, and lung. These tumors are very rare in children. Children sometimes have more than one tumor in the skin and/or liver. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Risk factors for angiosarcomas include the following: Signs Signs of angiosarcoma depend on where the tumor is and may include the following: Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose angiosarcoma and find out whether the tumor has spread. Treatment Treatment of angiosarcoma may include the following: For more information about childhood vascular tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood vascular tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. 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What is (are) Childhood Vascular Tumors ?

Key Points - Childhood vascular tumors form from cells that make blood vessels or lymph vessels. - Tests are used to detect (find) and diagnose childhood vascular tumors. - Childhood vascular tumors may be classified into four groups. - Benign tumors - Intermediate (locally aggressive) tumors - Intermediate (rarely metastasizing) tumors - Malignant tumors Childhood vascular tumors form from cells that make blood vessels or lymph vessels. Vascular tumors can form from abnormal blood vessel or lymph vessel cells anywhere in the body. They may be benign (not cancer) or malignant (cancer). There are many types of vascular tumors. The most common type of childhood vascular tumor is hemangioma, which is a benign tumor that usually goes away on its own. Because malignant vascular tumors are rare in children, there is not a lot of information about what treatment works best. Childhood vascular tumors may be classified into four groups. Benign tumors Benign tumors are not cancer. This summary has information about the following benign vascular tumors: - Infantile hemangioma. - Congenital hemangioma. - Benign vascular tumors of the liver. - Spindle cell hemangioma. - Epithelioid hemangioma. - Pyogenic granuloma (lobular capillary hemangioma). - Angiofibroma. - Juvenile nasopharyngeal angiofibroma. Intermediate (locally aggressive) tumors Intermediate tumors that are locally aggressive often spread to the area around the tumor. This summary has information about the following locally aggressive vascular tumors: - Kaposiform hemangioendothelioma and tufted angioma. Intermediate (rarely metastasizing) tumors Intermediate (rarely metastasizing) tumors sometimes spread to other parts of the body. This summary has information about the following vascular tumors that rarely metastasize: - Retiform hemangioendothelioma. - Papillary intralymphatic angioendothelioma. - Composite hemangioendothelioma. - Kaposi sarcoma. Malignant tumors Malignant tumors are cancer. This summary has information about the following malignant vascular tumors: - Epithelioid hemangioendothelioma. - Angiosarcoma of soft tissue. Benign Tumors Infantile Hemangioma Infantile hemangiomas are the most common type of benign vascular tumor in children. An infantile hemangioma may also be called a &quot;strawberry mark.&quot; Immature cells that are meant to form blood vessels form a tumor instead. These tumors are not usually seen at birth but appear when the infant is 3 to 6 weeks old. Most hemangiomas get bigger for about 5 months, then stop growing and slowly fade away completely during the next several years. It is rare for them to come back. Hemangiomas may be on the skin, in the tissue below the skin, and/or in an organ. They are usually on the head and neck but can be anywhere on or in the body. Hemangiomas may appear as a single lesion, one or more lesions spread over a larger area of the body, or multiple lesions in more than one part of the body. Lesions that are spread over a larger area of the body or multiple lesions are more likely to cause problems. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesnt mean that you will not get the disease. Talk with your child&apos;s doctor if you think your child may be at risk. Infantile hemangiomas are more common in the following: - Girls. - Whites. - Premature babies. - Twins, triplets, or other multiple births. - Babies of mothers who are older at time of the pregnancy or who have problems with the placenta during pregnancy. Other risk factors for infantile hemangiomas include the following: - Having certain syndromes. - PHACE syndrome: A syndrome in which the hemangioma spreads across a large area of the body (usually the head or face). Other health problems involving the large blood vessels, heart, eyes, and/or brain may also occur. - LUMBAR/PELVIS/SACRAL syndrome: A syndrome in which the hemangioma spreads across a large area of the lower back. Other health problems that affect the urinary system, genitals, rectum, anus, brain, spinal cord, and nerve function may also occur. Having more than one hemangioma or an airway hemangioma increases the risk of having other health problems. - Multiple hemangiomas: Having more than five hemangiomas on the skin is a sign that there may be hemangiomas in an organ, most commonly the liver. Heart, muscle, and thyroid gland problems can also occur. - Airway hemangiomas: Hemangiomas in the airway usually occur along with a large, beard-shaped area of hemangioma on the face (from the ears, around the mouth, lower chin, and front of neck). It is important for airway hemangiomas to be treated before the child has trouble breathing. Signs and Symptoms Infantile hemangiomas may cause any of the following signs and symptoms. Check with your childs doctor if your child has any of the following: - Skin lesions: An area of spidery veins or lightened or discolored skin may appear before the hemangioma does. Hemangiomas occur as firm, warm, bright red-blue lesions on the skin. Lesions that form ulcers are also painful. Later, as the hemangiomas go away, they begin fading in the center before flattening and losing color. - Lesions below the skin: Lesions that grow under the skin in the fat may appear blue or purple. If the lesions are deep enough under the skin surface, they may not be seen. - Lesions in an organ: There may be no signs that hemangiomas have formed on an organ. Although most infantile hemangiomas are nothing to worry about, if your child develops any lumps or red or blue marks on the skin check with your child&apos;s doctor. He or she can recommend a specialist if needed. Diagnostic Tests A physical exam and history are usually all that are needed to diagnose infantile hemangiomas. If there is something about the tumor that looks unusual, a biopsy may be done. If the hemangioma is deeper inside the body with no change to the skin, or the lesions are spread across a large area of the body, an ultrasound or MRI may be done. See the General Information section for a description of these tests and procedures. If the hemangiomas are part of a syndrome, more tests may be done such as an echocardiogram, magnetic resonance angiogram, and eye exam. Treatment Most hemangiomas fade and shrink without treatment. If the hemangioma is large or causing other health problems, treatment may include the following: - Propranolol or other beta-blocker therapy. - Steroid therapy, before beta-blocker therapy is begun or when beta-blockers cannot be used. - Pulsed dye laser surgery, for hemangiomas that have ulcers or have not gone away. - Surgery (excision) for hemangiomas that have ulcers, cause vision problems, or have not gone away. - Topical beta-blocker therapy for hemangiomas that are in one area of the skin. - Combined therapy, such as propranolol and steroid therapy or propranolol and topical beta-blocker therapy. Congenital Hemangioma Congenital hemangioma is a benign vascular tumor that begins forming before birth and is fully formed when the baby is born. They&apos;re usually on the skin but can be in another organ. There are three types of congenital hemangiomas: - Rapidly Involuting Congenital Hemangioma: These tumors go away on their own 12 to 15 months after birth. They can form ulcers, bleed, and cause temporary heart and blood clotting problems. The skin may look a little different even after the hemangiomas go away. - Partial Involuting Congenital Hemangioma: These tumors do not go away completely. - Non-Involuting Congenital Hemangioma: These tumors never go away on their own. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose congenital hemangioma. Treatment Treatment of rapidly involuting congenital hemangioma and partial involuting congenital hemangioma may include the following: - Observation only. Treatment of non-involuting congenital hemangioma may include the following: - Surgery to remove the tumor depending on where it is and whether it is causing symptoms. Benign Vascular Tumors of the Liver Benign vascular tumors of the liver may be focal (a single lesion in one area of the liver), multifocal (multiple lesions in one area of the liver), or diffuse (multiple lesions in more than one area of the liver). The liver has many functions, including filtering blood and making proteins needed for blood clotting. Sometimes, blood that normally flows through the liver is blocked or slowed by the tumor. This sends blood directly to the heart without going through the liver and is called a liver shunt. This can cause heart failure and problems with blood clotting. Focal Tumors Focal tumors are usually rapidly involuting congenital hemangiomas or non-involuting congenital hemangiomas. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose focal benign vascular tumors. Treatment Treatment of focal tumors of the liver depends on whether symptoms occur and may include the following: - Observation. - Drugs to manage symptoms, including heart failure and blood clotting problems. - Embolization of the liver to manage symptoms, including heart failure. Multifocal and Diffuse Tumors Multifocal and diffuse tumors of the liver are usually infantile hemangiomas. Diffuse tumors of the liver can cause serious effects, including problems with the thyroid gland and heart. The liver can enlarge, press on other organs, and cause more symptoms. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose multifocal or diffuse benign vascular tumors. Treatment Treatment of multifocal and diffuse liver tumors may include the following: - Observation for multifocal tumors of the liver that do not cause symptoms. - Beta-blocker therapy (propranolol). - Chemotherapy. - Steroid therapy. - Total hepatectomy and liver transplant, when the tumors do not respond to drug therapy. This is only done when the tumors have spread widely in the liver and more than one organ has failed. If a vascular tumor of the liver does not respond to standard treatments, a biopsy may be done to see if the tumor has become malignant. Spindle Cell Hemangioma Spindle cell hemangiomas contain cells called spindle cells. Under a microscope, spindle cells look long and slender. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesnt mean that you will not get the disease. Talk with your child&apos;s doctor if you think your child may be at risk. Spindle cell hemangiomas are likely to occur in children with the following syndromes: - Maffucci syndrome, which affects cartilage and skin. - Klippel-Trenaunay syndrome, which affects blood vessels, soft tissues, and bones. Signs Spindle cell hemangiomas appear on or under the skin. They are painful red-brown or bluish lesions that usually appear on the arms or legs. They can begin as one lesion and develop into more lesions over years. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose spindle cell hemangioma. Treatment There is no standard treatment for spindle cell hemangiomas. Treatment may include the following: - Surgery to remove the tumor. Spindle cell hemangiomas may come back after surgery. Epithelioid Hemangioma Epithelioid hemangiomas usually form on or in the skin, especially the head, but can occur in other areas, such as bone. Signs and Symptoms Epithelioid hemangiomas are sometimes caused by injury. On the skin, they may appear as firm pink to red bumps and may be itchy. Epithelioid hemangioma of the bone may cause swelling, pain, and weakened bone in the affected area. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose epithelioid hemangioma. Treatment There is no standard treatment for epithelioid hemangiomas. Treatment may include the following: - Surgery (curettage or resection). - Sclerotherapy. - Radiation therapy in rare cases. Epithelioid hemangiomas often come back after treatment. Pyogenic Granuloma Pyogenic granuloma is also called lobular capillary hemangioma. It is most common in older children and young adults but may occur at any age. The lesions are sometimes caused by injury or from the use of certain medicines, including birth control pills and retinoids. They may also form for no known reason inside capillaries (the smallest blood vessels) or other places on the body. Usually there is only one lesion, but sometimes multiple lesions occur in the same area or the lesions may spread to other areas of the body. Signs Pyogenic granulomas are raised, bright red lesions that may be small or large and smooth or bumpy. They grow quickly over weeks to months and may bleed a lot. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose pyogenic granuloma. Treatment Some pyogenic granulomas go away without treatment. Other pyogenic granulomas need treatment that may include the following: - Surgery (excision or curettage) to remove the lesion. - Laser photocoagulation. Pyogenic granulomas often come back after treatment. Angiofibroma Angiofibromas are rare. They are benign skin lesions that usually occur with a condition called tuberous sclerosis (an inherited disorder that causes skin lesions, seizures, and mental disabilities). Signs Angiofibromas appear as red bumps on the face. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose angiofibroma. Treatment Treatment of angiofibromas may include the following: - Surgery (excision) to remove the tumor. - Laser therapy. - Targeted therapy (sirolimus). Juvenile Nasopharyngeal Angiofibroma Juvenile nasopharyngeal angiofibromas are benign tumors but they can invade nearby tissue. They begin in the nasal cavity and may spread to the nasopharynx, the paranasal sinuses, the bone around the eyes, and sometimes to the brain. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose juvenile nasopharyngeal angiofibroma. Treatment Treatment of juvenile nasopharyngeal angiofibromas may include the following: - Surgery (excision) to remove the tumor. - Radiation therapy. - Chemotherapy. - Immunotherapy (interferon). - Targeted therapy (sirolimus). Intermediate Tumors that Spread Locally Kaposiform Hemangioendothelioma and Tufted Angioma Kaposiform hemangioendotheliomas and tufted angiomas are blood vessel tumors that occur in infants or during early childhood. These tumors can cause Kasabach-Merritt phenomenon, a condition in which the blood is not able to clot and serious bleeding may occur. In Kasabach-Merritt phenomenon the tumor traps and destroys platelets (blood-clotting cells). Then there aren&apos;t enough platelets in the blood when needed to stop bleeding. This type of vascular tumor is not related to Kaposi sarcoma. Signs and Symptoms Kaposiform hemangioendotheliomas and tufted angiomas usually occur on the skin of the arms and legs, but may also form in deeper tissues, such as muscle or bone. Signs and symptoms may include the following: - Firm, painful areas of skin that look bruised. - Purple or brownish-red areas of skin. - Easy bruising. - Bleeding more than the usual amount from mucous membranes, wounds, and other tissues. - Anemia (weakness, feeling tired, or looking pale). Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose kaposiform hemangioendothelioma. If a physical exam and MRI clearly show the tumor is a kaposiform hemangioendothelioma or a tufted angioma, a biopsy may not be needed. A biopsy is not always done because serious bleeding can occur. Treatment Treatment of kaposiform hemangioendotheliomas and tufted angiomas depends on the child&apos;s symptoms. Infection, delay in treatment, and surgery can cause bleeding that is life-threatening. Kaposiform hemangioendotheliomas and tufted angiomas are best treated by a vascular anomaly specialist. Treatment and supportive care to manage bleeding may include the following: - Steroid therapy which may be followed by chemotherapy. - Non-steroidal anti-inflammatory drugs (NSAID), such as aspirin. - Immunotherapy (interferon). - Antifibrinolytic therapy to improve blood clotting. - Chemotherapy with one or more anticancer drugs. - Beta-blocker therapy (propranolol). - Surgery (excision) to remove the tumor, with or without embolization. - Targeted therapy (sirolimus). - A clinical trial of targeted therapy (sirolimus) and steroid therapy. Even with treatment, these tumors do not fully go away and can come back. Long-term effects include chronic pain, heart failure, bone problems, and lymphedema (the build up of lymph fluid in tissues). Intermediate Tumors that Rarely Spread Retiform Hemangioendothelioma Retiform hemangioendotheliomas are slow growing, flat tumors that occur in young adults and sometimes children. These tumors usually occur on or under the skin of the arms, legs, and trunk. These tumors often come back after treatment, but they usually do not spread to other parts of the body. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose retiform hemangioendothelioma. Treatment Treatment of retiform hemangioendotheliomas may include the following: - Surgery (excision) to remove the tumor. Follow up will include monitoring to see if the tumor comes back. - Radiation therapy and chemotherapy when surgery cannot be done or when the tumor has come back. Papillary Intralymphatic Angioendothelioma Papillary intralymphatic angioendotheliomas are also called Dabska tumors. These tumors form in or under the skin anywhere on the body. The tumors contain channels that look like lymph vessels. Lymph nodes are sometimes affected. Signs Papillary intralymphatic angioendotheliomas may appear as firm, raised, purplish bumps, which may be small or large. These tumors grow slowly over time. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose papillary intralymphatic angioendothelioma. Treatment Treatment of papillary intralymphatic angioendotheliomas may include the following: - Surgery (excision) to remove the tumor. Composite Hemangioendothelioma Composite hemangioendotheliomas have features of both benign and malignant vascular tumors. These tumors usually occur on or under the skin on the arms or legs. They may also occur on the head, neck, or chest. Composite hemangioendotheliomas are not likely to metastasize (spread) but they may come back in the same place. When the tumors metastasize, they usually spread to nearby lymph nodes. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose composite hemangioendothelioma and find out whether the tumor has spread. Treatment Treatment of composite hemangioendotheliomas may include the following: - Surgery to remove the tumor. - Radiation therapy and chemotherapy for tumors that have spread. Kaposi Sarcoma Kaposi sarcoma is a cancer that causes lesions to grow in the skin; the mucous membranes lining the mouth, nose, and throat; lymph nodes; or other organs. It is caused by the Kaposi sarcoma herpes virus (KSHV). In the United States, it usually occurs in people who have a weak immune system caused by AIDS or by drugs used in organ transplants. It is very rare in children and can be caused by HIV infection and rare immune system disorders. Signs Signs in children may include the following: - Lesions in the skin, mouth, or throat. Skin lesions are red, purple, or brown and change from flat, to raised, to scaly areas called plaques, to nodules. - Swollen lymph nodes. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose Kaposi sarcoma. Treatment Treatment of Kaposi sarcoma may include the following: - Chemotherapy. See the PDQ summary on Kaposi Sarcoma Treatment for information about Kaposi sarcoma in adults. Malignant Tumors Epithelioid Hemangioendothelioma Epithelioid hemangioendotheliomas can occur in children, but are most common in adults between 30 and 50 years of age. They usually occur in the liver, lung, or in bone. They may be either fast growing or slow growing. In about a third of cases, the tumor spreads to other parts of the body very quickly. Signs and Symptoms Signs and symptoms depend on where the tumor is: - On the skin, the tumors can be raised and rounded or flat, red-brown patches that feel warm. - In the lung, there may be no early symptoms. Signs and symptoms that occur may include: - Chest pain. - Spitting up blood. - Anemia (weakness, feeling tired, or looking pale). - Trouble breathing (from scarred lung tissue). - In bone, the tumors can cause breaks. Diagnostic Tests Epithelioid hemangioendotheliomas in the liver are found with CT scans and MRI scans. See the General Information section for a description of these tests and procedures used to diagnose epithelioid hemangioendothelioma and find out whether the tumor has spread. X-rays may also be done. Treatment Treatment of slow-growing epithelioid hemangioendotheliomas includes the following: - Observation. Treatment of fast-growing epithelioid hemangioendotheliomas may include the following: - Surgery to remove the tumor when possible. - Immunotherapy (interferon) and targeted therapy (thalidomide, sorafenib, pazopanib, sirolimus) for tumors that are likely to spread. - Chemotherapy. - Total hepatectomy and liver transplant when the tumor is in the liver. Angiosarcoma of the Soft Tissue Angiosarcomas are fast-growing tumors that form in blood vessels or lymph vessels in any part of the body, usually in soft tissue. Most angiosarcomas are in or near the skin. Those in deeper soft tissue can form in the liver, spleen, and lung. These tumors are very rare in children. Children sometimes have more than one tumor in the skin and/or liver. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesnt mean that you will not get the disease. Talk with your child&apos;s doctor if you think your child may be at risk. Risk factors for angiosarcomas include the following: - Being exposed to radiation. - Chronic (long-term) lymphedema, a condition in which extra lymph fluid builds up in tissues and causes swelling. - Having a benign vascular tumor. A benign tumor, such as a hemangioma, may become an angiosarcoma but this rare. Signs Signs of angiosarcoma depend on where the tumor is and may include the following: - Red patches on the skin that bleed easily. - Purple tumors. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose angiosarcoma and find out whether the tumor has spread. Treatment Treatment of angiosarcoma may include the following: - Surgery to completely remove the tumor. - A combination of surgery, chemotherapy, and radiation therapy for angiosarcomas that have spread. - Targeted therapy (bevacizumab) and chemotherapy for angiosarcomas that began as infantile hemangiomas. - A clinical trial of targeted therapy, radiation therapy, and surgery with or without chemotherapy.

These tumors may be benign (not cancer) or malignant (cancer). There are many types of vascular tumors. The most common type of childhood vascular tumor is infantile hemangioma, which is a benign tumor that usually goes away on its own. Because malignant vascular tumors are rare in children, there is not a lot of information about what treatment works best. The following tests and procedures may be used: Benign tumors are not cancer. This summary has information about the following benign vascular tumors: Intermediate tumors that are locally aggressive often spread to the area around the tumor. This summary has information about the following locally aggressive vascular tumors: Intermediate (rarely metastasizing) tumors sometimes spread to other parts of the body. This summary has information about the following vascular tumors that rarely metastasize: Malignant tumors are cancer. This summary has information about the following malignant vascular tumors: Different types of treatment are available for children with vascular tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because vascular tumors in children are rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with cancer and who specialize in certain areas of medicine. These may include the following specialists: Beta-blockers are drugs that decrease blood pressure and heart rate. When used in patients with vascular tumors, beta-blockers may help shrink the tumors. Beta-blocker therapy may be given by vein (IV), by mouth, or placed on the skin (topical). The way the beta-blocker therapy is given depends on the type of vascular tumor and where the tumor first formed. The beta-blocker propranolol is usually the first treatment for hemangiomas. Infants younger than 4 weeks, or who have an underlying condition, or who are treated with IV propranolol may need to have their treatment started in a hospital. Propranolol is also used to treat benign vascular tumor of liver and kaposiform hemangioendothelioma. Other beta-blockers used to treat vascular tumors include atenolol, nadolol, and timolol. Infantile hemangioma may also be treated with propranolol and steroid therapy or propranolol and topical beta-blocker therapy. See the drug information summary on Propranolol Hydrochloride for more information. The following types of surgery may be used to remove many types of vascular tumors: The type of surgery used depends on the type of vascular tumor and where the tumor formed in the body. For malignant tumors, after the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Photocoagulation is the use of an intense beam of light, such as a laser, to seal off blood vessels or destroy tissue. It is used to treat pyogenic granuloma. Cryotherapy is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as abnormal blood vessels in pyogenic granuloma. This type of treatment is also called cryosurgery. For more information, see Cryosurgery to Treat Cancer. Embolization is a procedure that uses particles, such as tiny gelatin sponges or beads, to block blood vessels in the liver. It may be used to treat some benign vascular tumors of the liver and kaposiform hemangioendothelioma. Chemotherapy is a treatment that uses drugs to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach tumor cells throughout the body. Sometimes more than one anticancer drug is given. This is called combination chemotherapy. Sclerotherapy is a treatment used to destroy the blood vessel that leads to the tumor and the tumor. A liquid is injected into the blood vessel, causing it to scar and break down. Over time, the destroyed blood vessel is absorbed into normal tissue. The blood flows through nearby healthy veins instead. Sclerotherapy is used in the treatment of epithelioid hemangioma. Radiation therapy is a treatment that uses high-energy x-rays or other types of radiation to kill tumor cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with the tumor. It is used to treat some vascular tumors. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific tumor cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Different types of targeted therapy are being used or studied to treat childhood vascular tumors: Immunotherapy is a treatment that uses the patient’s immune system to fight disease. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against disease. This tumor treatment is a type of biologic therapy. The following types of immunotherapy are being used in the treatment of childhood vascular tumors: There are two types of immune checkpoint inhibitor therapy: Other drugs used to treat childhood vascular tumors or manage their effects include the following: Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Some treatments, such as chemotherapy and radiation therapy, cause side effects that continue or appear months or years after treatment has ended. These are called late effects. Late effects of treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the possible late effects caused by some treatments. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.) For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the research process. Clinical trials are done to find out if new treatments are safe and effective or better than the standard treatment. Many of today's standard treatments are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way disease will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose tumors have not gotten better. There are also clinical trials that test new ways to stop tumors from recurring (coming back) or reduce the side effects of treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the vascular tumor may be repeated. Some tests will be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the tumor has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Infantile hemangiomas are the most common type of benign vascular tumor in children. Infantile hemangiomas form when immature cells that are meant to form blood vessels form a tumor instead. An infantile hemangioma may also be called a "strawberry mark." These tumors are not usually seen at birth but appear when the infant is 3 to 6 weeks old. Most hemangiomas get bigger for about 5 months and then stop growing. The hemangiomas slowly fade away over the next several years, but a red mark or loose or wrinkled skin may remain. It is rare for an infantile hemangioma to come back after it has faded away. Infantile hemangiomas may be on the skin, in the tissue below the skin, and/or in an organ. They are usually on the head and neck but can be anywhere on or in the body. Hemangiomas may appear as a single lesion, one or more lesions spread over a larger area of the body, or multiple lesions in more than one part of the body. Lesions that are spread over a larger area of the body or multiple lesions are more likely to cause problems. Infantile hemangioma with minimal or arrested growth (IH-MAG) is a certain type of infantile hemangioma that is seen at birth and does not tend to get bigger. The lesion appears as light and dark areas of redness in the skin. The lesions are usually on the lower body but can be on the head and neck. Hemangiomas of this type go away over time without treatment. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Infantile hemangiomas are more common in the following: Other risk factors for infantile hemangiomas include the following: Having more than one hemangioma or an airway or ophthalmic hemangioma increases the risk of having other health problems. Signs and Symptoms Infantile hemangiomas may cause any of the following signs and symptoms. Check with your child’s doctor if your child has any of the following: Although most infantile hemangiomas are nothing to worry about, if your child develops any lumps or red or blue marks on the skin check with your child's doctor. He or she can refer the child to a specialist if needed. Diagnostic Tests A physical exam and history are usually all that are needed to diagnose infantile hemangiomas. If there is something about the tumor that looks unusual, a biopsy may be done. If the hemangioma is deeper inside the body with no change to the skin, or the lesions are spread across a large area of the body, an ultrasound may be done. Infants with five or more hemangiomas on the skin should have an ultrasound of the liver done to check for a liver hemangioma. See the General Information section for a description of these tests and procedures. If the hemangiomas are part of a syndrome, more tests may be done, such as an echocardiogram, MRI, magnetic resonance angiogram, and eye exam. Treatment Most hemangiomas fade and shrink without treatment. If the hemangioma is large or causing other health problems, treatment may include the following: Congenital hemangioma is a benign vascular tumor that begins forming before birth and is fully formed when the baby is born. They're usually on the skin but can be in another organ. A congenital hemangioma may occur as a rash of purple spots and the skin around the spot may be lighter. There are three types of congenital hemangiomas: Diagnostic Tests See the General Information section for a description of tests and procedures, such as ultrasound, used to diagnose congenital hemangioma. Treatment Treatment of rapidly involuting congenital hemangioma and partial involuting congenital hemangioma may include the following: Treatment of non-involuting congenital hemangioma may include the following: Benign vascular tumors of the liver may be focal vascular lesions (a single lesion in one area of the liver), multiple liver lesions (more than one lesion in one area of the liver), or diffuse liver lesions (more than one lesion in more than one area of the liver). The liver has many functions, including filtering blood and making proteins needed for blood clotting. Sometimes, blood that normally flows through the liver is blocked or slowed by the tumor. This sends blood directly to the heart without going through the liver and is called a liver shunt. This can cause heart failure and problems with blood clotting. Focal Vascular Lesions Focal vascular lesions are usually rapidly involuting congenital hemangiomas or non-involuting congenital hemangiomas. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose focal vascular lesions of the liver. Treatment Treatment of focal vascular lesions of the liver depends on whether symptoms occur and may include the following: Multiple and Diffuse Liver Lesions Multifocal and diffuse lesions of the liver are usually infantile hemangiomas. Diffuse lesions of the liver can cause serious effects, including problems with the thyroid gland and heart. The liver can enlarge, press on other organs, and cause more symptoms. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose multifocal or diffuse benign vascular lesions. Treatment Treatment of multifocal liver lesions may include the following: Treatment of diffuse liver lesions may include the following: If a vascular lesion of the liver does not respond to standard treatments, a biopsy may be done to see if the tumor has become malignant. Spindle cell hemangiomas contain cells called spindle cells. Under a microscope, spindle cells look long and slender. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Spindle cell hemangiomas are likely to occur in children with the following syndromes: Signs Spindle cell hemangiomas appear on or under the skin. They are painful red-brown or bluish lesions that usually appear on the arms or legs. They can begin as one lesion and develop into more lesions over years. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose spindle cell hemangioma. Treatment There is no standard treatment for spindle cell hemangiomas. Treatment may include the following: Spindle cell hemangiomas may come back after surgery. Epithelioid hemangiomas usually form on or in the skin, especially the head, but can occur in other areas, such as bone. Signs and Symptoms Epithelioid hemangiomas are sometimes caused by injury. On the skin, they may appear as firm pink to red bumps and may be itchy. Epithelioid hemangioma of the bone may cause swelling, pain, and weakened bone in the affected area. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose epithelioid hemangioma. Treatment There is no standard treatment for epithelioid hemangiomas. Treatment may include the following: Epithelioid hemangiomas often come back after treatment. Pyogenic granuloma is also called lobular capillary hemangioma. It is most common in older children and young adults but may occur at any age. The lesions are sometimes caused by injury or from the use of certain medicines, including birth control pills and retinoids. They may also form for no known reason inside capillaries (the smallest blood vessels), arteries, veins, or other places on the body. Some lesions may be associated with capillary malformations. Usually there is only one lesion, but sometimes multiple lesions occur in the same area or the lesions may spread to other areas of the body. Signs Pyogenic granulomas are raised, bright red lesions that may be small or large and smooth or bumpy. They grow quickly over weeks to months and may bleed a lot. These lesions are usually on the surface of the skin, but may form in the tissues below the skin and look like other vascular lesions. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose pyogenic granuloma. Treatment Some pyogenic granulomas go away without treatment. Other pyogenic granulomas need treatment that may include the following: Pyogenic granulomas often come back after treatment. Angiofibromas are rare. They are benign skin lesions that usually occur with a condition called tuberous sclerosis (an inherited disorder that causes skin lesions, seizures, and mental disabilities). Signs Angiofibromas appear as red bumps on the face. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose angiofibroma. Treatment Treatment of angiofibromas may include the following: Juvenile nasopharyngeal angiofibromas are benign tumors but they can grow into nearby tissue. They begin in the nasal cavity and may spread to the nasopharynx, the paranasal sinuses, the bone around the eyes, and sometimes to the brain. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose juvenile nasopharyngeal angiofibroma. Treatment Treatment of juvenile nasopharyngeal angiofibromas may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Kaposiform hemangioendotheliomas and tufted angiomas are blood vessel tumors that occur in infants or young children. These tumors can cause Kasabach-Merritt phenomenon, a condition in which the blood is not able to clot and serious bleeding may occur. In Kasabach-Merritt phenomenon, the tumor traps and destroys platelets (blood-clotting cells). Then there aren't enough platelets in the blood when needed to stop bleeding. This type of vascular tumor is not related to Kaposi sarcoma. Signs and Symptoms Kaposiform hemangioendotheliomas and tufted angiomas usually occur on the skin of the arms and legs, but may also form in deeper tissues, such as muscle or bone, or in the chest, abdomen, head, or neck. Signs and symptoms may include the following: Patients who have kaposiform hemangioendothelioma and tufted angioma may have anemia (weakness, feeling tired, or looking pale). Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose kaposiform hemangioendothelioma and tufted angioma. If a physical exam and MRI clearly show the tumor is a kaposiform hemangioendothelioma or a tufted angioma, a biopsy may not be needed. A biopsy is not always done because serious bleeding can occur. Treatment Treatment of kaposiform hemangioendotheliomas and tufted angiomas depends on the child's symptoms. Infection, delay in treatment, and surgery can cause bleeding that is life-threatening. Kaposiform hemangioendotheliomas and tufted angiomas are best treated by a vascular anomaly specialist. Treatment for uncomplicated kaposiform hemangioendotheliomas and tufted angiomas may include the following: Treatment for complicated kaposiform hemangioendotheliomas and tufted angiomas may include the following: Even with treatment, these tumors do not fully go away and can come back. Pain and inflammation may get worse with age, often around the time of puberty. Long-term effects include chronic pain, heart failure, bone problems, and lymphedema (the build up of lymph fluid in tissues). For information about the treatments listed below, see the Treatment Option Overview section. Pseudomyogenic hemangioendothelioma can occur in children, but is most common in men aged 20 to 50 years. These tumors are rare, and usually occur on or under the skin, or in bone. They may spread to nearby tissue but usually do not spread to other parts of the body. In most cases, there are multiple tumors. Signs and Symptoms Pseudomyogenic hemangioendotheliomas may appear as a lump in soft tissue or may cause pain in the affected area. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose pseudomyogenic hemangioendothelioma. Treatment Treatment of pseudomyogenic hemangioendotheliomas may include the following: Because pseudomyogenic hemangioendothelioma is so rare in children, treatment options are based on clinical trials in adults. Retiform hemangioendotheliomas are slow growing, flat tumors that occur in young adults and sometimes children. These tumors usually occur on or under the skin of the arms, legs, and trunk. These tumors usually do not spread to other parts of the body. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose retiform hemangioendothelioma. Treatment Treatment of retiform hemangioendotheliomas may include the following: Retiform hemangioendothelioma may come back after treatment. Papillary intralymphatic angioendotheliomas are also called Dabska tumors. These tumors form in or under the skin anywhere on the body. Lymph nodes are sometimes affected. Signs Papillary intralymphatic angioendotheliomas may appear as firm, raised, purplish bumps, which may be small or large. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose papillary intralymphatic angioendothelioma. Treatment Treatment of papillary intralymphatic angioendotheliomas may include the following: Composite hemangioendotheliomas have features of both benign and malignant vascular tumors. These tumors usually occur on or under the skin on the arms or legs. They may also occur on the head, neck, or chest. Composite hemangioendotheliomas are not likely to metastasize (spread) but they may come back in the same place. When the tumors metastasize, they usually spread to nearby lymph nodes. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose composite hemangioendothelioma and find out whether the tumor has spread. Treatment Treatment of composite hemangioendotheliomas may include the following: Kaposi sarcoma is a cancer that causes lesions to grow in the skin; the mucous membranes lining the mouth, nose, and throat; lymph nodes; or other organs. It is caused by the Kaposi sarcoma herpes virus (KSHV). In the United States, it occurs most often in children who have a weak immune system caused by rare immune system disorders, HIV infection, or drugs used in organ transplants. Signs Signs in children may include the following: Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose Kaposi sarcoma. Treatment Treatment of Kaposi sarcoma may include the following: Because Kaposi sarcoma is so rare in children, some treatment options are based on clinical trials in adults. See the PDQ summary on Kaposi Sarcoma Treatment for information about Kaposi sarcoma in adults. For information about the treatments listed below, see the Treatment Option Overview section. Epithelioid hemangioendotheliomas can occur in children, but are most common in adults aged 30 to 50 years. They usually occur in the liver, lung, or in bone. They may be fast growing or slow growing. In about a third of cases, the tumor spreads to other parts of the body very quickly. Signs and Symptoms Signs and symptoms depend on where the tumor is in the body. Check with your child's doctor if your child has any of the following: Tumors that occur in the liver or soft tissue may also cause signs and symptoms. Diagnostic Tests Epithelioid hemangioendotheliomas in the liver are found with CT scans and MRI scans. See the General Information section for a description of these tests and procedures used to diagnose epithelioid hemangioendothelioma and find out whether the tumor has spread. X-rays may also be done. Treatment Treatment of slow-growing epithelioid hemangioendotheliomas includes the following: Treatment of fast-growing epithelioid hemangioendotheliomas may include the following: Angiosarcomas are fast-growing tumors that form in blood vessels or lymph vessels in any part of the body, usually in soft tissue. Most angiosarcomas are in or near the skin. Those in deeper soft tissue can form in the liver, spleen, and lung. These tumors are very rare in children. Children sometimes have more than one tumor in the skin and/or liver. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Risk factors for angiosarcomas include the following: Signs Signs of angiosarcoma depend on where the tumor is and may include the following: Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose angiosarcoma and find out whether the tumor has spread. Treatment Treatment of angiosarcoma may include the following: For more information about childhood vascular tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood vascular tumors. It is meant to inform and help patients, families, and caregivers. 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How to diagnose Childhood Vascular Tumors ?

Tests are used to detect (find) and diagnose childhood vascular tumors. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps, lesions, or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to be looked at later. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). - Biopsy : The removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for signs of cancer. A biopsy is not always needed to diagnose a vascular tumor.

These tumors may be benign (not cancer) or malignant (cancer). There are many types of vascular tumors. The most common type of childhood vascular tumor is infantile hemangioma, which is a benign tumor that usually goes away on its own. Because malignant vascular tumors are rare in children, there is not a lot of information about what treatment works best. The following tests and procedures may be used: Benign tumors are not cancer. This summary has information about the following benign vascular tumors: Intermediate tumors that are locally aggressive often spread to the area around the tumor. This summary has information about the following locally aggressive vascular tumors: Intermediate (rarely metastasizing) tumors sometimes spread to other parts of the body. This summary has information about the following vascular tumors that rarely metastasize: Malignant tumors are cancer. This summary has information about the following malignant vascular tumors: Different types of treatment are available for children with vascular tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because vascular tumors in children are rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with cancer and who specialize in certain areas of medicine. These may include the following specialists: Beta-blockers are drugs that decrease blood pressure and heart rate. When used in patients with vascular tumors, beta-blockers may help shrink the tumors. Beta-blocker therapy may be given by vein (IV), by mouth, or placed on the skin (topical). The way the beta-blocker therapy is given depends on the type of vascular tumor and where the tumor first formed. The beta-blocker propranolol is usually the first treatment for hemangiomas. Infants younger than 4 weeks, or who have an underlying condition, or who are treated with IV propranolol may need to have their treatment started in a hospital. Propranolol is also used to treat benign vascular tumor of liver and kaposiform hemangioendothelioma. Other beta-blockers used to treat vascular tumors include atenolol, nadolol, and timolol. Infantile hemangioma may also be treated with propranolol and steroid therapy or propranolol and topical beta-blocker therapy. See the drug information summary on Propranolol Hydrochloride for more information. The following types of surgery may be used to remove many types of vascular tumors: The type of surgery used depends on the type of vascular tumor and where the tumor formed in the body. For malignant tumors, after the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Photocoagulation is the use of an intense beam of light, such as a laser, to seal off blood vessels or destroy tissue. It is used to treat pyogenic granuloma. Cryotherapy is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as abnormal blood vessels in pyogenic granuloma. This type of treatment is also called cryosurgery. For more information, see Cryosurgery to Treat Cancer. Embolization is a procedure that uses particles, such as tiny gelatin sponges or beads, to block blood vessels in the liver. It may be used to treat some benign vascular tumors of the liver and kaposiform hemangioendothelioma. Chemotherapy is a treatment that uses drugs to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach tumor cells throughout the body. Sometimes more than one anticancer drug is given. This is called combination chemotherapy. Sclerotherapy is a treatment used to destroy the blood vessel that leads to the tumor and the tumor. A liquid is injected into the blood vessel, causing it to scar and break down. Over time, the destroyed blood vessel is absorbed into normal tissue. The blood flows through nearby healthy veins instead. Sclerotherapy is used in the treatment of epithelioid hemangioma. Radiation therapy is a treatment that uses high-energy x-rays or other types of radiation to kill tumor cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with the tumor. It is used to treat some vascular tumors. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific tumor cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Different types of targeted therapy are being used or studied to treat childhood vascular tumors: Immunotherapy is a treatment that uses the patient’s immune system to fight disease. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against disease. This tumor treatment is a type of biologic therapy. The following types of immunotherapy are being used in the treatment of childhood vascular tumors: There are two types of immune checkpoint inhibitor therapy: Other drugs used to treat childhood vascular tumors or manage their effects include the following: Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Some treatments, such as chemotherapy and radiation therapy, cause side effects that continue or appear months or years after treatment has ended. These are called late effects. Late effects of treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the possible late effects caused by some treatments. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.) For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the research process. Clinical trials are done to find out if new treatments are safe and effective or better than the standard treatment. Many of today's standard treatments are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way disease will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose tumors have not gotten better. There are also clinical trials that test new ways to stop tumors from recurring (coming back) or reduce the side effects of treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the vascular tumor may be repeated. Some tests will be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the tumor has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Infantile hemangiomas are the most common type of benign vascular tumor in children. Infantile hemangiomas form when immature cells that are meant to form blood vessels form a tumor instead. An infantile hemangioma may also be called a "strawberry mark." These tumors are not usually seen at birth but appear when the infant is 3 to 6 weeks old. Most hemangiomas get bigger for about 5 months and then stop growing. The hemangiomas slowly fade away over the next several years, but a red mark or loose or wrinkled skin may remain. It is rare for an infantile hemangioma to come back after it has faded away. Infantile hemangiomas may be on the skin, in the tissue below the skin, and/or in an organ. They are usually on the head and neck but can be anywhere on or in the body. Hemangiomas may appear as a single lesion, one or more lesions spread over a larger area of the body, or multiple lesions in more than one part of the body. Lesions that are spread over a larger area of the body or multiple lesions are more likely to cause problems. Infantile hemangioma with minimal or arrested growth (IH-MAG) is a certain type of infantile hemangioma that is seen at birth and does not tend to get bigger. The lesion appears as light and dark areas of redness in the skin. The lesions are usually on the lower body but can be on the head and neck. Hemangiomas of this type go away over time without treatment. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Infantile hemangiomas are more common in the following: Other risk factors for infantile hemangiomas include the following: Having more than one hemangioma or an airway or ophthalmic hemangioma increases the risk of having other health problems. Signs and Symptoms Infantile hemangiomas may cause any of the following signs and symptoms. Check with your child’s doctor if your child has any of the following: Although most infantile hemangiomas are nothing to worry about, if your child develops any lumps or red or blue marks on the skin check with your child's doctor. He or she can refer the child to a specialist if needed. Diagnostic Tests A physical exam and history are usually all that are needed to diagnose infantile hemangiomas. If there is something about the tumor that looks unusual, a biopsy may be done. If the hemangioma is deeper inside the body with no change to the skin, or the lesions are spread across a large area of the body, an ultrasound may be done. Infants with five or more hemangiomas on the skin should have an ultrasound of the liver done to check for a liver hemangioma. See the General Information section for a description of these tests and procedures. If the hemangiomas are part of a syndrome, more tests may be done, such as an echocardiogram, MRI, magnetic resonance angiogram, and eye exam. Treatment Most hemangiomas fade and shrink without treatment. If the hemangioma is large or causing other health problems, treatment may include the following: Congenital hemangioma is a benign vascular tumor that begins forming before birth and is fully formed when the baby is born. They're usually on the skin but can be in another organ. A congenital hemangioma may occur as a rash of purple spots and the skin around the spot may be lighter. There are three types of congenital hemangiomas: Diagnostic Tests See the General Information section for a description of tests and procedures, such as ultrasound, used to diagnose congenital hemangioma. Treatment Treatment of rapidly involuting congenital hemangioma and partial involuting congenital hemangioma may include the following: Treatment of non-involuting congenital hemangioma may include the following: Benign vascular tumors of the liver may be focal vascular lesions (a single lesion in one area of the liver), multiple liver lesions (more than one lesion in one area of the liver), or diffuse liver lesions (more than one lesion in more than one area of the liver). The liver has many functions, including filtering blood and making proteins needed for blood clotting. Sometimes, blood that normally flows through the liver is blocked or slowed by the tumor. This sends blood directly to the heart without going through the liver and is called a liver shunt. This can cause heart failure and problems with blood clotting. Focal Vascular Lesions Focal vascular lesions are usually rapidly involuting congenital hemangiomas or non-involuting congenital hemangiomas. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose focal vascular lesions of the liver. Treatment Treatment of focal vascular lesions of the liver depends on whether symptoms occur and may include the following: Multiple and Diffuse Liver Lesions Multifocal and diffuse lesions of the liver are usually infantile hemangiomas. Diffuse lesions of the liver can cause serious effects, including problems with the thyroid gland and heart. The liver can enlarge, press on other organs, and cause more symptoms. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose multifocal or diffuse benign vascular lesions. Treatment Treatment of multifocal liver lesions may include the following: Treatment of diffuse liver lesions may include the following: If a vascular lesion of the liver does not respond to standard treatments, a biopsy may be done to see if the tumor has become malignant. Spindle cell hemangiomas contain cells called spindle cells. Under a microscope, spindle cells look long and slender. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Spindle cell hemangiomas are likely to occur in children with the following syndromes: Signs Spindle cell hemangiomas appear on or under the skin. They are painful red-brown or bluish lesions that usually appear on the arms or legs. They can begin as one lesion and develop into more lesions over years. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose spindle cell hemangioma. Treatment There is no standard treatment for spindle cell hemangiomas. Treatment may include the following: Spindle cell hemangiomas may come back after surgery. Epithelioid hemangiomas usually form on or in the skin, especially the head, but can occur in other areas, such as bone. Signs and Symptoms Epithelioid hemangiomas are sometimes caused by injury. On the skin, they may appear as firm pink to red bumps and may be itchy. Epithelioid hemangioma of the bone may cause swelling, pain, and weakened bone in the affected area. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose epithelioid hemangioma. Treatment There is no standard treatment for epithelioid hemangiomas. Treatment may include the following: Epithelioid hemangiomas often come back after treatment. Pyogenic granuloma is also called lobular capillary hemangioma. It is most common in older children and young adults but may occur at any age. The lesions are sometimes caused by injury or from the use of certain medicines, including birth control pills and retinoids. They may also form for no known reason inside capillaries (the smallest blood vessels), arteries, veins, or other places on the body. Some lesions may be associated with capillary malformations. Usually there is only one lesion, but sometimes multiple lesions occur in the same area or the lesions may spread to other areas of the body. Signs Pyogenic granulomas are raised, bright red lesions that may be small or large and smooth or bumpy. They grow quickly over weeks to months and may bleed a lot. These lesions are usually on the surface of the skin, but may form in the tissues below the skin and look like other vascular lesions. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose pyogenic granuloma. Treatment Some pyogenic granulomas go away without treatment. Other pyogenic granulomas need treatment that may include the following: Pyogenic granulomas often come back after treatment. Angiofibromas are rare. They are benign skin lesions that usually occur with a condition called tuberous sclerosis (an inherited disorder that causes skin lesions, seizures, and mental disabilities). Signs Angiofibromas appear as red bumps on the face. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose angiofibroma. Treatment Treatment of angiofibromas may include the following: Juvenile nasopharyngeal angiofibromas are benign tumors but they can grow into nearby tissue. They begin in the nasal cavity and may spread to the nasopharynx, the paranasal sinuses, the bone around the eyes, and sometimes to the brain. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose juvenile nasopharyngeal angiofibroma. Treatment Treatment of juvenile nasopharyngeal angiofibromas may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Kaposiform hemangioendotheliomas and tufted angiomas are blood vessel tumors that occur in infants or young children. These tumors can cause Kasabach-Merritt phenomenon, a condition in which the blood is not able to clot and serious bleeding may occur. In Kasabach-Merritt phenomenon, the tumor traps and destroys platelets (blood-clotting cells). Then there aren't enough platelets in the blood when needed to stop bleeding. This type of vascular tumor is not related to Kaposi sarcoma. Signs and Symptoms Kaposiform hemangioendotheliomas and tufted angiomas usually occur on the skin of the arms and legs, but may also form in deeper tissues, such as muscle or bone, or in the chest, abdomen, head, or neck. Signs and symptoms may include the following: Patients who have kaposiform hemangioendothelioma and tufted angioma may have anemia (weakness, feeling tired, or looking pale). Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose kaposiform hemangioendothelioma and tufted angioma. If a physical exam and MRI clearly show the tumor is a kaposiform hemangioendothelioma or a tufted angioma, a biopsy may not be needed. A biopsy is not always done because serious bleeding can occur. Treatment Treatment of kaposiform hemangioendotheliomas and tufted angiomas depends on the child's symptoms. Infection, delay in treatment, and surgery can cause bleeding that is life-threatening. Kaposiform hemangioendotheliomas and tufted angiomas are best treated by a vascular anomaly specialist. Treatment for uncomplicated kaposiform hemangioendotheliomas and tufted angiomas may include the following: Treatment for complicated kaposiform hemangioendotheliomas and tufted angiomas may include the following: Even with treatment, these tumors do not fully go away and can come back. Pain and inflammation may get worse with age, often around the time of puberty. Long-term effects include chronic pain, heart failure, bone problems, and lymphedema (the build up of lymph fluid in tissues). For information about the treatments listed below, see the Treatment Option Overview section. Pseudomyogenic hemangioendothelioma can occur in children, but is most common in men aged 20 to 50 years. These tumors are rare, and usually occur on or under the skin, or in bone. They may spread to nearby tissue but usually do not spread to other parts of the body. In most cases, there are multiple tumors. Signs and Symptoms Pseudomyogenic hemangioendotheliomas may appear as a lump in soft tissue or may cause pain in the affected area. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose pseudomyogenic hemangioendothelioma. Treatment Treatment of pseudomyogenic hemangioendotheliomas may include the following: Because pseudomyogenic hemangioendothelioma is so rare in children, treatment options are based on clinical trials in adults. Retiform hemangioendotheliomas are slow growing, flat tumors that occur in young adults and sometimes children. These tumors usually occur on or under the skin of the arms, legs, and trunk. These tumors usually do not spread to other parts of the body. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose retiform hemangioendothelioma. Treatment Treatment of retiform hemangioendotheliomas may include the following: Retiform hemangioendothelioma may come back after treatment. Papillary intralymphatic angioendotheliomas are also called Dabska tumors. These tumors form in or under the skin anywhere on the body. Lymph nodes are sometimes affected. Signs Papillary intralymphatic angioendotheliomas may appear as firm, raised, purplish bumps, which may be small or large. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose papillary intralymphatic angioendothelioma. Treatment Treatment of papillary intralymphatic angioendotheliomas may include the following: Composite hemangioendotheliomas have features of both benign and malignant vascular tumors. These tumors usually occur on or under the skin on the arms or legs. They may also occur on the head, neck, or chest. Composite hemangioendotheliomas are not likely to metastasize (spread) but they may come back in the same place. When the tumors metastasize, they usually spread to nearby lymph nodes. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose composite hemangioendothelioma and find out whether the tumor has spread. Treatment Treatment of composite hemangioendotheliomas may include the following: Kaposi sarcoma is a cancer that causes lesions to grow in the skin; the mucous membranes lining the mouth, nose, and throat; lymph nodes; or other organs. It is caused by the Kaposi sarcoma herpes virus (KSHV). In the United States, it occurs most often in children who have a weak immune system caused by rare immune system disorders, HIV infection, or drugs used in organ transplants. Signs Signs in children may include the following: Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose Kaposi sarcoma. Treatment Treatment of Kaposi sarcoma may include the following: Because Kaposi sarcoma is so rare in children, some treatment options are based on clinical trials in adults. See the PDQ summary on Kaposi Sarcoma Treatment for information about Kaposi sarcoma in adults. For information about the treatments listed below, see the Treatment Option Overview section. Epithelioid hemangioendotheliomas can occur in children, but are most common in adults aged 30 to 50 years. They usually occur in the liver, lung, or in bone. They may be fast growing or slow growing. In about a third of cases, the tumor spreads to other parts of the body very quickly. Signs and Symptoms Signs and symptoms depend on where the tumor is in the body. Check with your child's doctor if your child has any of the following: Tumors that occur in the liver or soft tissue may also cause signs and symptoms. Diagnostic Tests Epithelioid hemangioendotheliomas in the liver are found with CT scans and MRI scans. See the General Information section for a description of these tests and procedures used to diagnose epithelioid hemangioendothelioma and find out whether the tumor has spread. X-rays may also be done. Treatment Treatment of slow-growing epithelioid hemangioendotheliomas includes the following: Treatment of fast-growing epithelioid hemangioendotheliomas may include the following: Angiosarcomas are fast-growing tumors that form in blood vessels or lymph vessels in any part of the body, usually in soft tissue. Most angiosarcomas are in or near the skin. Those in deeper soft tissue can form in the liver, spleen, and lung. These tumors are very rare in children. Children sometimes have more than one tumor in the skin and/or liver. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Risk factors for angiosarcomas include the following: Signs Signs of angiosarcoma depend on where the tumor is and may include the following: Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose angiosarcoma and find out whether the tumor has spread. Treatment Treatment of angiosarcoma may include the following: For more information about childhood vascular tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood vascular tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. 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What are the treatments for Childhood Vascular Tumors ?

Key Points - There are different types of treatment for childhood vascular tumors. - Children with childhood vascular tumors should have their treatment planned by a team of health care providers who are experts in treating cancer in children. - Some treatments cause side effects months or years after treatment has ended. - Eleven types of standard treatment are used: - Beta-blocker therapy - Surgery - Photocoagulation - Embolization - Chemotherapy - Sclerotherapy - Radiation therapy - Targeted therapy - Immunotherapy - Other drug therapy - Observation - New types of treatment are being tested in clinical trials. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for childhood vascular tumors. Different types of treatment are available for children with vascular tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because vascular tumors in children are rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Children with childhood vascular tumors should have their treatment planned by a team of health care providers who are experts in treating cancer in children. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with cancer and who specialize in certain areas of medicine. These may include the following specialists: - Pediatric vascular anomaly specialist (expert in treating children with vascular tumors). - Pediatric surgeon. - Orthopedic surgeon. - Radiation oncologist. - Pediatric nurse specialist. - Rehabilitation specialist. - Psychologist. - Social worker. Some treatments cause side effects months or years after treatment has ended. Some treatments, such as chemotherapy and radiation therapy, cause side effects that continue or appear months or years after treatment has ended. These are called late effects. Late effects of treatment may include: - Physical problems. - Changes in mood, feelings, thinking, learning, or memory. - Second cancers (new types of cancer). Some late effects may be treated or controlled. It is important to talk with your child&apos;s doctors about the possible late effects caused by some treatments. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Eleven types of standard treatment are used: Beta-blocker therapy Beta-blockers are drugs that decrease blood pressure and heart rate. When used in patients with vascular tumors, beta-blockers may help shrink the tumors. Beta-blocker therapy may be given by vein (IV), by mouth, or placed on the skin (topical). The way the beta-blocker therapy is given depends on the type of vascular tumor and where the tumor first formed. The beta-blocker propranolol is usually the first treatment for hemangiomas. Infants treated with IV propranolol may need to have their treatment started in a hospital. Propranolol is also used to treat benign vascular tumor of liver and kaposiform hemangioendothelioma. Other beta-blockers used to treat vascular tumors include atenolol, nadolol, and timolol. Infantile hemangioma may also be treated with propranolol and steroid therapy or propranolol and topical beta-blocker therapy. See the drug information summary on Propranolol Hydrochloride for more information. Surgery The following types of surgery may be used to remove many types of vascular tumors: - Excision: Surgery to remove the entire tumor and some of the healthy tissue around it. - Laser surgery: A surgical procedure that uses a laser beam (a narrow beam of intense light) as a knife to make bloodless cuts in tissue or to remove a skin lesion such as a tumor. Surgery with a pulsed dye laser may be used for some hemangiomas. This type of laser uses a beam of light that targets blood vessels in the skin. The light is changed into heat and the blood vessels are destroyed without damaging nearby skin. - Total hepatectomy and liver transplant: A surgical procedure to remove the entire liver followed by a transplant of a healthy liver from a donor. - Curettage: A procedure in which abnormal tissue is removed using a small, spoon-shaped instrument called a curette. The type of surgery used depends on the type of vascular tumor and where the tumor formed in the body. For malignant tumors, even if the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the tumor will come back, is called adjuvant therapy. Photocoagulation Photocoagulation is the use of an intense beam of light, such as a laser, to seal off blood vessels or destroy tissue. It is used to treat pyogenic granuloma. Embolization Embolization is a procedure that uses particles, such as tiny gelatin sponges or beads, to block blood vessels in the liver. It may be used to treat some benign vascular tumors of the liver and kaposiform hemangioendothelioma. Chemotherapy Chemotherapy is a treatment that uses drugs to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach tumor cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect tumor cells in those areas (regional chemotherapy). Chemotherapy for hemangiomas may also be given topically (applied to the skin in a cream or lotion). The way the chemotherapy is given depends on the type of the vascular tumor being treated. Sclerotherapy Sclerotherapy is a treatment used to destroy the blood vessel with the tumor. A liquid is injected into the blood vessel, causing it to scar and break down. Over time, the destroyed blood vessel is absorbed into normal tissue. The blood flows through nearby healthy veins instead. Sclerotherapy is used in the treatment of epithelioid hemangioma. Radiation therapy Radiation therapy is a treatment that uses high-energy x-rays or other types of radiation to kill tumor cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the tumor. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the tumor. The way the radiation therapy is given depends on the type of the vascular tumor being treated. External radiation is used to treat some vascular tumors. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to attack specific tumor cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Angiogenesis inhibitors are a type of targeted therapy. - Angiogenesis inhibitors are drugs that stop cells from dividing and prevent the growth of new blood vessels that tumors need to grow. The targeted therapy drugs thalidomide, sorafenib, pazopanib, sirolimus, and bevacizumab are angiogenesis inhibitors used to treat childhood vascular tumors. Immunotherapy Immunotherapy is a treatment that uses the patients immune system to fight disease. Substances made by the body or made in a laboratory are used to boost, direct, or restore the bodys natural defenses against disease. Interferon is a type of immunotherapy used to treat childhood vascular tumors. It interferes with the division of tumor cells and can slow tumor growth. It is used in the treatment of juvenile nasopharyngeal angiofibroma, kaposiform hemangioendothelioma, and epithelioid hemangioendothelioma. Other drug therapy Other drugs used to treat childhood vascular tumors or manage their effects include the following: - Steroid therapy: Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Steroid drugs help shrink some vascular tumors. - Non-steroidal anti-inflammatory drugs (NSAIDs): NSAIDs are commonly used to decrease fever, swelling, pain, and redness. Examples of NSAIDs are aspirin, ibuprofen, and naproxen. In the treatment of vascular tumors, NSAIDs can increase the flow of blood through the tumors and decrease the chance that an unwanted blood clot will form. - Antifibrinolytic therapy: These drugs help the blood clot in patients who have Kasabach-Merritt syndrome. Fibrin is the main protein in a blood clot that helps stop bleeding and heal wounds. Some vascular tumors cause fibrin to break down and the patient&apos;s blood does not clot normally, causing uncontrolled bleeding. Antifibrinolytics help prevent the breakdown of fibrin. Observation Observation is closely monitoring a patients condition without giving any treatment until signs or symptoms appear or change. New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the research process. Clinical trials are done to find out if new treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way disease will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose tumors have not gotten better. There are also clinical trials that test new ways to stop tumors from recurring (coming back) or reduce the side effects of treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the vascular tumor may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child&apos;s condition has changed or if the tumor has recurred (come back). These tests are sometimes called follow-up tests or check-ups.

These tumors may be benign (not cancer) or malignant (cancer). There are many types of vascular tumors. The most common type of childhood vascular tumor is infantile hemangioma, which is a benign tumor that usually goes away on its own. Because malignant vascular tumors are rare in children, there is not a lot of information about what treatment works best. The following tests and procedures may be used: Benign tumors are not cancer. This summary has information about the following benign vascular tumors: Intermediate tumors that are locally aggressive often spread to the area around the tumor. This summary has information about the following locally aggressive vascular tumors: Intermediate (rarely metastasizing) tumors sometimes spread to other parts of the body. This summary has information about the following vascular tumors that rarely metastasize: Malignant tumors are cancer. This summary has information about the following malignant vascular tumors: Different types of treatment are available for children with vascular tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because vascular tumors in children are rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with cancer and who specialize in certain areas of medicine. These may include the following specialists: Beta-blockers are drugs that decrease blood pressure and heart rate. When used in patients with vascular tumors, beta-blockers may help shrink the tumors. Beta-blocker therapy may be given by vein (IV), by mouth, or placed on the skin (topical). The way the beta-blocker therapy is given depends on the type of vascular tumor and where the tumor first formed. The beta-blocker propranolol is usually the first treatment for hemangiomas. Infants younger than 4 weeks, or who have an underlying condition, or who are treated with IV propranolol may need to have their treatment started in a hospital. Propranolol is also used to treat benign vascular tumor of liver and kaposiform hemangioendothelioma. Other beta-blockers used to treat vascular tumors include atenolol, nadolol, and timolol. Infantile hemangioma may also be treated with propranolol and steroid therapy or propranolol and topical beta-blocker therapy. See the drug information summary on Propranolol Hydrochloride for more information. The following types of surgery may be used to remove many types of vascular tumors: The type of surgery used depends on the type of vascular tumor and where the tumor formed in the body. For malignant tumors, after the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Photocoagulation is the use of an intense beam of light, such as a laser, to seal off blood vessels or destroy tissue. It is used to treat pyogenic granuloma. Cryotherapy is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as abnormal blood vessels in pyogenic granuloma. This type of treatment is also called cryosurgery. For more information, see Cryosurgery to Treat Cancer. Embolization is a procedure that uses particles, such as tiny gelatin sponges or beads, to block blood vessels in the liver. It may be used to treat some benign vascular tumors of the liver and kaposiform hemangioendothelioma. Chemotherapy is a treatment that uses drugs to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach tumor cells throughout the body. Sometimes more than one anticancer drug is given. This is called combination chemotherapy. Sclerotherapy is a treatment used to destroy the blood vessel that leads to the tumor and the tumor. A liquid is injected into the blood vessel, causing it to scar and break down. Over time, the destroyed blood vessel is absorbed into normal tissue. The blood flows through nearby healthy veins instead. Sclerotherapy is used in the treatment of epithelioid hemangioma. Radiation therapy is a treatment that uses high-energy x-rays or other types of radiation to kill tumor cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with the tumor. It is used to treat some vascular tumors. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific tumor cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Different types of targeted therapy are being used or studied to treat childhood vascular tumors: Immunotherapy is a treatment that uses the patient’s immune system to fight disease. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against disease. This tumor treatment is a type of biologic therapy. The following types of immunotherapy are being used in the treatment of childhood vascular tumors: There are two types of immune checkpoint inhibitor therapy: Other drugs used to treat childhood vascular tumors or manage their effects include the following: Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Some treatments, such as chemotherapy and radiation therapy, cause side effects that continue or appear months or years after treatment has ended. These are called late effects. Late effects of treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the possible late effects caused by some treatments. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.) For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the research process. Clinical trials are done to find out if new treatments are safe and effective or better than the standard treatment. Many of today's standard treatments are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way disease will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose tumors have not gotten better. There are also clinical trials that test new ways to stop tumors from recurring (coming back) or reduce the side effects of treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the vascular tumor may be repeated. Some tests will be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the tumor has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Infantile hemangiomas are the most common type of benign vascular tumor in children. Infantile hemangiomas form when immature cells that are meant to form blood vessels form a tumor instead. An infantile hemangioma may also be called a "strawberry mark." These tumors are not usually seen at birth but appear when the infant is 3 to 6 weeks old. Most hemangiomas get bigger for about 5 months and then stop growing. The hemangiomas slowly fade away over the next several years, but a red mark or loose or wrinkled skin may remain. It is rare for an infantile hemangioma to come back after it has faded away. Infantile hemangiomas may be on the skin, in the tissue below the skin, and/or in an organ. They are usually on the head and neck but can be anywhere on or in the body. Hemangiomas may appear as a single lesion, one or more lesions spread over a larger area of the body, or multiple lesions in more than one part of the body. Lesions that are spread over a larger area of the body or multiple lesions are more likely to cause problems. Infantile hemangioma with minimal or arrested growth (IH-MAG) is a certain type of infantile hemangioma that is seen at birth and does not tend to get bigger. The lesion appears as light and dark areas of redness in the skin. The lesions are usually on the lower body but can be on the head and neck. Hemangiomas of this type go away over time without treatment. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Infantile hemangiomas are more common in the following: Other risk factors for infantile hemangiomas include the following: Having more than one hemangioma or an airway or ophthalmic hemangioma increases the risk of having other health problems. Signs and Symptoms Infantile hemangiomas may cause any of the following signs and symptoms. Check with your child’s doctor if your child has any of the following: Although most infantile hemangiomas are nothing to worry about, if your child develops any lumps or red or blue marks on the skin check with your child's doctor. He or she can refer the child to a specialist if needed. Diagnostic Tests A physical exam and history are usually all that are needed to diagnose infantile hemangiomas. If there is something about the tumor that looks unusual, a biopsy may be done. If the hemangioma is deeper inside the body with no change to the skin, or the lesions are spread across a large area of the body, an ultrasound may be done. Infants with five or more hemangiomas on the skin should have an ultrasound of the liver done to check for a liver hemangioma. See the General Information section for a description of these tests and procedures. If the hemangiomas are part of a syndrome, more tests may be done, such as an echocardiogram, MRI, magnetic resonance angiogram, and eye exam. Treatment Most hemangiomas fade and shrink without treatment. If the hemangioma is large or causing other health problems, treatment may include the following: Congenital hemangioma is a benign vascular tumor that begins forming before birth and is fully formed when the baby is born. They're usually on the skin but can be in another organ. A congenital hemangioma may occur as a rash of purple spots and the skin around the spot may be lighter. There are three types of congenital hemangiomas: Diagnostic Tests See the General Information section for a description of tests and procedures, such as ultrasound, used to diagnose congenital hemangioma. Treatment Treatment of rapidly involuting congenital hemangioma and partial involuting congenital hemangioma may include the following: Treatment of non-involuting congenital hemangioma may include the following: Benign vascular tumors of the liver may be focal vascular lesions (a single lesion in one area of the liver), multiple liver lesions (more than one lesion in one area of the liver), or diffuse liver lesions (more than one lesion in more than one area of the liver). The liver has many functions, including filtering blood and making proteins needed for blood clotting. Sometimes, blood that normally flows through the liver is blocked or slowed by the tumor. This sends blood directly to the heart without going through the liver and is called a liver shunt. This can cause heart failure and problems with blood clotting. Focal Vascular Lesions Focal vascular lesions are usually rapidly involuting congenital hemangiomas or non-involuting congenital hemangiomas. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose focal vascular lesions of the liver. Treatment Treatment of focal vascular lesions of the liver depends on whether symptoms occur and may include the following: Multiple and Diffuse Liver Lesions Multifocal and diffuse lesions of the liver are usually infantile hemangiomas. Diffuse lesions of the liver can cause serious effects, including problems with the thyroid gland and heart. The liver can enlarge, press on other organs, and cause more symptoms. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose multifocal or diffuse benign vascular lesions. Treatment Treatment of multifocal liver lesions may include the following: Treatment of diffuse liver lesions may include the following: If a vascular lesion of the liver does not respond to standard treatments, a biopsy may be done to see if the tumor has become malignant. Spindle cell hemangiomas contain cells called spindle cells. Under a microscope, spindle cells look long and slender. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Spindle cell hemangiomas are likely to occur in children with the following syndromes: Signs Spindle cell hemangiomas appear on or under the skin. They are painful red-brown or bluish lesions that usually appear on the arms or legs. They can begin as one lesion and develop into more lesions over years. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose spindle cell hemangioma. Treatment There is no standard treatment for spindle cell hemangiomas. Treatment may include the following: Spindle cell hemangiomas may come back after surgery. Epithelioid hemangiomas usually form on or in the skin, especially the head, but can occur in other areas, such as bone. Signs and Symptoms Epithelioid hemangiomas are sometimes caused by injury. On the skin, they may appear as firm pink to red bumps and may be itchy. Epithelioid hemangioma of the bone may cause swelling, pain, and weakened bone in the affected area. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose epithelioid hemangioma. Treatment There is no standard treatment for epithelioid hemangiomas. Treatment may include the following: Epithelioid hemangiomas often come back after treatment. Pyogenic granuloma is also called lobular capillary hemangioma. It is most common in older children and young adults but may occur at any age. The lesions are sometimes caused by injury or from the use of certain medicines, including birth control pills and retinoids. They may also form for no known reason inside capillaries (the smallest blood vessels), arteries, veins, or other places on the body. Some lesions may be associated with capillary malformations. Usually there is only one lesion, but sometimes multiple lesions occur in the same area or the lesions may spread to other areas of the body. Signs Pyogenic granulomas are raised, bright red lesions that may be small or large and smooth or bumpy. They grow quickly over weeks to months and may bleed a lot. These lesions are usually on the surface of the skin, but may form in the tissues below the skin and look like other vascular lesions. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose pyogenic granuloma. Treatment Some pyogenic granulomas go away without treatment. Other pyogenic granulomas need treatment that may include the following: Pyogenic granulomas often come back after treatment. Angiofibromas are rare. They are benign skin lesions that usually occur with a condition called tuberous sclerosis (an inherited disorder that causes skin lesions, seizures, and mental disabilities). Signs Angiofibromas appear as red bumps on the face. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose angiofibroma. Treatment Treatment of angiofibromas may include the following: Juvenile nasopharyngeal angiofibromas are benign tumors but they can grow into nearby tissue. They begin in the nasal cavity and may spread to the nasopharynx, the paranasal sinuses, the bone around the eyes, and sometimes to the brain. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose juvenile nasopharyngeal angiofibroma. Treatment Treatment of juvenile nasopharyngeal angiofibromas may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Kaposiform hemangioendotheliomas and tufted angiomas are blood vessel tumors that occur in infants or young children. These tumors can cause Kasabach-Merritt phenomenon, a condition in which the blood is not able to clot and serious bleeding may occur. In Kasabach-Merritt phenomenon, the tumor traps and destroys platelets (blood-clotting cells). Then there aren't enough platelets in the blood when needed to stop bleeding. This type of vascular tumor is not related to Kaposi sarcoma. Signs and Symptoms Kaposiform hemangioendotheliomas and tufted angiomas usually occur on the skin of the arms and legs, but may also form in deeper tissues, such as muscle or bone, or in the chest, abdomen, head, or neck. Signs and symptoms may include the following: Patients who have kaposiform hemangioendothelioma and tufted angioma may have anemia (weakness, feeling tired, or looking pale). Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose kaposiform hemangioendothelioma and tufted angioma. If a physical exam and MRI clearly show the tumor is a kaposiform hemangioendothelioma or a tufted angioma, a biopsy may not be needed. A biopsy is not always done because serious bleeding can occur. Treatment Treatment of kaposiform hemangioendotheliomas and tufted angiomas depends on the child's symptoms. Infection, delay in treatment, and surgery can cause bleeding that is life-threatening. Kaposiform hemangioendotheliomas and tufted angiomas are best treated by a vascular anomaly specialist. Treatment for uncomplicated kaposiform hemangioendotheliomas and tufted angiomas may include the following: Treatment for complicated kaposiform hemangioendotheliomas and tufted angiomas may include the following: Even with treatment, these tumors do not fully go away and can come back. Pain and inflammation may get worse with age, often around the time of puberty. Long-term effects include chronic pain, heart failure, bone problems, and lymphedema (the build up of lymph fluid in tissues). For information about the treatments listed below, see the Treatment Option Overview section. Pseudomyogenic hemangioendothelioma can occur in children, but is most common in men aged 20 to 50 years. These tumors are rare, and usually occur on or under the skin, or in bone. They may spread to nearby tissue but usually do not spread to other parts of the body. In most cases, there are multiple tumors. Signs and Symptoms Pseudomyogenic hemangioendotheliomas may appear as a lump in soft tissue or may cause pain in the affected area. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose pseudomyogenic hemangioendothelioma. Treatment Treatment of pseudomyogenic hemangioendotheliomas may include the following: Because pseudomyogenic hemangioendothelioma is so rare in children, treatment options are based on clinical trials in adults. Retiform hemangioendotheliomas are slow growing, flat tumors that occur in young adults and sometimes children. These tumors usually occur on or under the skin of the arms, legs, and trunk. These tumors usually do not spread to other parts of the body. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose retiform hemangioendothelioma. Treatment Treatment of retiform hemangioendotheliomas may include the following: Retiform hemangioendothelioma may come back after treatment. Papillary intralymphatic angioendotheliomas are also called Dabska tumors. These tumors form in or under the skin anywhere on the body. Lymph nodes are sometimes affected. Signs Papillary intralymphatic angioendotheliomas may appear as firm, raised, purplish bumps, which may be small or large. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose papillary intralymphatic angioendothelioma. Treatment Treatment of papillary intralymphatic angioendotheliomas may include the following: Composite hemangioendotheliomas have features of both benign and malignant vascular tumors. These tumors usually occur on or under the skin on the arms or legs. They may also occur on the head, neck, or chest. Composite hemangioendotheliomas are not likely to metastasize (spread) but they may come back in the same place. When the tumors metastasize, they usually spread to nearby lymph nodes. Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose composite hemangioendothelioma and find out whether the tumor has spread. Treatment Treatment of composite hemangioendotheliomas may include the following: Kaposi sarcoma is a cancer that causes lesions to grow in the skin; the mucous membranes lining the mouth, nose, and throat; lymph nodes; or other organs. It is caused by the Kaposi sarcoma herpes virus (KSHV). In the United States, it occurs most often in children who have a weak immune system caused by rare immune system disorders, HIV infection, or drugs used in organ transplants. Signs Signs in children may include the following: Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose Kaposi sarcoma. Treatment Treatment of Kaposi sarcoma may include the following: Because Kaposi sarcoma is so rare in children, some treatment options are based on clinical trials in adults. See the PDQ summary on Kaposi Sarcoma Treatment for information about Kaposi sarcoma in adults. For information about the treatments listed below, see the Treatment Option Overview section. Epithelioid hemangioendotheliomas can occur in children, but are most common in adults aged 30 to 50 years. They usually occur in the liver, lung, or in bone. They may be fast growing or slow growing. In about a third of cases, the tumor spreads to other parts of the body very quickly. Signs and Symptoms Signs and symptoms depend on where the tumor is in the body. Check with your child's doctor if your child has any of the following: Tumors that occur in the liver or soft tissue may also cause signs and symptoms. Diagnostic Tests Epithelioid hemangioendotheliomas in the liver are found with CT scans and MRI scans. See the General Information section for a description of these tests and procedures used to diagnose epithelioid hemangioendothelioma and find out whether the tumor has spread. X-rays may also be done. Treatment Treatment of slow-growing epithelioid hemangioendotheliomas includes the following: Treatment of fast-growing epithelioid hemangioendotheliomas may include the following: Angiosarcomas are fast-growing tumors that form in blood vessels or lymph vessels in any part of the body, usually in soft tissue. Most angiosarcomas are in or near the skin. Those in deeper soft tissue can form in the liver, spleen, and lung. These tumors are very rare in children. Children sometimes have more than one tumor in the skin and/or liver. Risk Factors Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get the disease; not having risk factors doesn’t mean that you will not get the disease. Talk with your child's doctor if you think your child may be at risk. Risk factors for angiosarcomas include the following: Signs Signs of angiosarcoma depend on where the tumor is and may include the following: Diagnostic Tests See the General Information section for a description of tests and procedures used to diagnose angiosarcoma and find out whether the tumor has spread. Treatment Treatment of angiosarcoma may include the following: For more information about childhood vascular tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood vascular tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Vascular Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/soft-tissue-sarcoma/patient/child-vascular-tumors-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 27253005] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. 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what research (or clinical trials) is being done for Childhood Vascular Tumors ?

New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the research process. Clinical trials are done to find out if new treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way disease will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose tumors have not gotten better. There are also clinical trials that test new ways to stop tumors from recurring (coming back) or reduce the side effects of treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Osteosarcoma usually starts in osteoblasts, which are a type of bone cell that becomes new bone. Osteosarcoma is most common in adolescents. It commonly forms in the ends of the long bones of the body, which include bones of the arms and legs. In children and adolescents, it often forms in the long bones, near the knee. Rarely, osteosarcoma may be found in soft tissue or organs in the chest or abdomen. Osteosarcoma is the most common type of bone cancer. UPS (formerly called malignant fibrous histiocytoma [MFH]) is a rare type of bone cancer that usually starts in soft tissue, but it may form in bone. In bone, UPS cells look similar to osteosarcoma under a microscope. UPS is treated like osteosarcoma. Ewing sarcoma is another kind of bone cancer, but it is not covered in this summary. For more information, see Ewing Sarcoma Treatment. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop osteosarcoma, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Risk factors for osteosarcoma include the following: These and other signs and symptoms may be caused by osteosarcoma or UPS or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Imaging tests are done before a biopsy. Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. It is important that the biopsy be done by a surgeon who is an expert in treating cancer of the bone. It is best if that surgeon is also the one who removes the tumor. The biopsy and the surgery to remove the tumor are planned together. The way the biopsy is done affects which type of surgery can be done later. The type of biopsy that is done will be based on the size of the tumor and where it is in the body. There are two types of biopsy that may be used: The following test may be done on the tissue that is removed: The prognosis may be affected by certain factors before and after treatment. The prognosis of untreated osteosarcoma and UPS may depend on the following: After osteosarcoma or UPS is treated, prognosis also depends on the following: Treatment options for osteosarcoma and UPS depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. Most patients with osteosarcoma and UPS are grouped according to whether cancer is localized or metastatic. The following tests and procedures may be used to find out if the cancer has spread: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if osteosarcoma spreads to the lung, the cancer cells in the lung are actually osteosarcoma cells. The disease is metastatic osteosarcoma, not lung cancer. The cancer may recur (come back) in the bone or in other parts of the body. Osteosarcoma and UPS most often recur in the lung, bone, or both. When osteosarcoma recurs, it is usually within 18 months after treatment is completed. Different types of treatment are available for children with osteosarcoma or UPS of bone. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating osteosarcoma and UPS and who specialize in certain areas of medicine. These may include the following specialists: For information about types and monitoring of late effects in childhood and adolescent cancer survivors, see Late Effects of Treatment for Childhood Cancer. Surgery to remove the entire tumor will be done when possible. Chemotherapy may be given before surgery to make the tumor smaller. This is called neoadjuvant chemotherapy. Chemotherapy is given so less bone tissue needs to be removed and there are fewer problems after surgery. The following types of surgery may be done: Studies have shown that survival is the same whether the first surgery done is a limb-sparing surgery or an amputation. After the doctor removes all the cancer that can be seen at the time of the surgery, patients are given chemotherapy to kill any cancer cells that are left in the area where the tumor was removed or that have spread to other parts of the body. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is the use of more than one anticancer drug. Chemotherapy is usually given before and after surgery to remove the primary tumor. For more information, see Drugs Approved for Bone Cancer. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Osteosarcoma and UPS cells are not killed easily by external radiation therapy. It may be used when a small amount of cancer is left after surgery or used together with other treatments. Samarium is a radioactive drug that targets areas where bone cells are growing, such as tumor cells in bone. It helps relieve pain caused by cancer in the bone and it also kills blood cells in the bone marrow. It is used to treat osteosarcoma that has come back after treatment in a different bone. Treatment with samarium may be followed by stem cell transplant. Before treatment with samarium, stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After treatment with samarium is complete, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Information about ongoing clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed localized osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Lung Metastasis When osteosarcoma or UPS spreads, it usually spreads to the lung. Treatment of newly diagnosed osteosarcoma and UPS with lung metastasis may include the following: Bone Metastasis or Bone with Lung Metastasis Newly diagnosed osteosarcoma and UPS may spread to a distant bone and/or the lung. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent osteosarcoma and UPS of bone may include the following: Treatment depends on the area and type of recurrence, for example: Clinical trials for treatment of recurrent osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about osteosarcoma and UPS, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of osteosarcoma and undifferentiated pleomorphic sarcoma (UPS) (formerly called malignant fibrous histiocytoma [MFH]) of bone. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Osteosarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/bone/patient/osteosarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389380] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Osteosarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Osteosarcoma and Malignant Fibrous Histiocytoma of Bone ?

Key Points - Osteosarcoma and malignant fibrous histiocytoma (MFH) of the bone are diseases in which malignant (cancer) cells form in bone. - Having past treatment with radiation can increase the risk of osteosarcoma. - Signs and symptoms of osteosarcoma and MFH include swelling over a bone or a bony part of the body and joint pain. - Imaging tests are used to detect (find) osteosarcoma and MFH. - A biopsy is done to diagnose osteosarcoma. - Certain factors affect prognosis (chance of recovery) and treatment options. Osteosarcoma and malignant fibrous histiocytoma (MFH) of the bone are diseases in which malignant (cancer) cells form in bone. Osteosarcoma usually starts in osteoblasts, which are a type of bone cell that becomes new bone tissue. Osteosarcoma is most common in adolescents. It commonly forms in the ends of the long bones of the body, which include bones of the arms and legs. In children and adolescents, it often forms in the bones near the knee. Rarely, osteosarcoma may be found in soft tissue or organs in the chest or abdomen. Osteosarcoma is the most common type of bone cancer. Malignant fibrous histiocytoma (MFH) of bone is a rare tumor of the bone. It is treated like osteosarcoma. Ewing sarcoma is another kind of bone cancer, but it is not covered in this summary. See the PDQ summary about Ewing Sarcoma Treatment for more information.

Osteosarcoma usually starts in osteoblasts, which are a type of bone cell that becomes new bone. Osteosarcoma is most common in adolescents. It commonly forms in the ends of the long bones of the body, which include bones of the arms and legs. In children and adolescents, it often forms in the long bones, near the knee. Rarely, osteosarcoma may be found in soft tissue or organs in the chest or abdomen. Osteosarcoma is the most common type of bone cancer. UPS (formerly called malignant fibrous histiocytoma [MFH]) is a rare type of bone cancer that usually starts in soft tissue, but it may form in bone. In bone, UPS cells look similar to osteosarcoma under a microscope. UPS is treated like osteosarcoma. Ewing sarcoma is another kind of bone cancer, but it is not covered in this summary. For more information, see Ewing Sarcoma Treatment. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop osteosarcoma, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Risk factors for osteosarcoma include the following: These and other signs and symptoms may be caused by osteosarcoma or UPS or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Imaging tests are done before a biopsy. Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. It is important that the biopsy be done by a surgeon who is an expert in treating cancer of the bone. It is best if that surgeon is also the one who removes the tumor. The biopsy and the surgery to remove the tumor are planned together. The way the biopsy is done affects which type of surgery can be done later. The type of biopsy that is done will be based on the size of the tumor and where it is in the body. There are two types of biopsy that may be used: The following test may be done on the tissue that is removed: The prognosis may be affected by certain factors before and after treatment. The prognosis of untreated osteosarcoma and UPS may depend on the following: After osteosarcoma or UPS is treated, prognosis also depends on the following: Treatment options for osteosarcoma and UPS depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. Most patients with osteosarcoma and UPS are grouped according to whether cancer is localized or metastatic. The following tests and procedures may be used to find out if the cancer has spread: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if osteosarcoma spreads to the lung, the cancer cells in the lung are actually osteosarcoma cells. The disease is metastatic osteosarcoma, not lung cancer. The cancer may recur (come back) in the bone or in other parts of the body. Osteosarcoma and UPS most often recur in the lung, bone, or both. When osteosarcoma recurs, it is usually within 18 months after treatment is completed. Different types of treatment are available for children with osteosarcoma or UPS of bone. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating osteosarcoma and UPS and who specialize in certain areas of medicine. These may include the following specialists: For information about types and monitoring of late effects in childhood and adolescent cancer survivors, see Late Effects of Treatment for Childhood Cancer. Surgery to remove the entire tumor will be done when possible. Chemotherapy may be given before surgery to make the tumor smaller. This is called neoadjuvant chemotherapy. Chemotherapy is given so less bone tissue needs to be removed and there are fewer problems after surgery. The following types of surgery may be done: Studies have shown that survival is the same whether the first surgery done is a limb-sparing surgery or an amputation. After the doctor removes all the cancer that can be seen at the time of the surgery, patients are given chemotherapy to kill any cancer cells that are left in the area where the tumor was removed or that have spread to other parts of the body. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is the use of more than one anticancer drug. Chemotherapy is usually given before and after surgery to remove the primary tumor. For more information, see Drugs Approved for Bone Cancer. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Osteosarcoma and UPS cells are not killed easily by external radiation therapy. It may be used when a small amount of cancer is left after surgery or used together with other treatments. Samarium is a radioactive drug that targets areas where bone cells are growing, such as tumor cells in bone. It helps relieve pain caused by cancer in the bone and it also kills blood cells in the bone marrow. It is used to treat osteosarcoma that has come back after treatment in a different bone. Treatment with samarium may be followed by stem cell transplant. Before treatment with samarium, stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After treatment with samarium is complete, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Information about ongoing clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed localized osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Lung Metastasis When osteosarcoma or UPS spreads, it usually spreads to the lung. Treatment of newly diagnosed osteosarcoma and UPS with lung metastasis may include the following: Bone Metastasis or Bone with Lung Metastasis Newly diagnosed osteosarcoma and UPS may spread to a distant bone and/or the lung. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent osteosarcoma and UPS of bone may include the following: Treatment depends on the area and type of recurrence, for example: Clinical trials for treatment of recurrent osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about osteosarcoma and UPS, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of osteosarcoma and undifferentiated pleomorphic sarcoma (UPS) (formerly called malignant fibrous histiocytoma [MFH]) of bone. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Osteosarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/bone/patient/osteosarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389380] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Osteosarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Osteosarcoma and Malignant Fibrous Histiocytoma of Bone? ?

Having past treatment with radiation can increase the risk of osteosarcoma. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesnt mean that you will not get cancer. Talk with your child&apos;s doctor if you think your child may be at risk. Risk factors for osteosarcoma include the following: - Past treatment with radiation therapy. - Past treatment with anticancer drugs called alkylating agents. - Having a certain change in the retinoblastoma gene. - Having certain conditions, such as the following: - Bloom syndrome. - Diamond-Blackfan anemia. - Li-Fraumeni syndrome. - Paget disease. - Hereditary retinoblastoma. - Rothmund-Thomson syndrome. - Werner syndrome.

Osteosarcoma usually starts in osteoblasts, which are a type of bone cell that becomes new bone. Osteosarcoma is most common in adolescents. It commonly forms in the ends of the long bones of the body, which include bones of the arms and legs. In children and adolescents, it often forms in the long bones, near the knee. Rarely, osteosarcoma may be found in soft tissue or organs in the chest or abdomen. Osteosarcoma is the most common type of bone cancer. UPS (formerly called malignant fibrous histiocytoma [MFH]) is a rare type of bone cancer that usually starts in soft tissue, but it may form in bone. In bone, UPS cells look similar to osteosarcoma under a microscope. UPS is treated like osteosarcoma. Ewing sarcoma is another kind of bone cancer, but it is not covered in this summary. For more information, see Ewing Sarcoma Treatment. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop osteosarcoma, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Risk factors for osteosarcoma include the following: These and other signs and symptoms may be caused by osteosarcoma or UPS or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Imaging tests are done before a biopsy. Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. It is important that the biopsy be done by a surgeon who is an expert in treating cancer of the bone. It is best if that surgeon is also the one who removes the tumor. The biopsy and the surgery to remove the tumor are planned together. The way the biopsy is done affects which type of surgery can be done later. The type of biopsy that is done will be based on the size of the tumor and where it is in the body. There are two types of biopsy that may be used: The following test may be done on the tissue that is removed: The prognosis may be affected by certain factors before and after treatment. The prognosis of untreated osteosarcoma and UPS may depend on the following: After osteosarcoma or UPS is treated, prognosis also depends on the following: Treatment options for osteosarcoma and UPS depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. Most patients with osteosarcoma and UPS are grouped according to whether cancer is localized or metastatic. The following tests and procedures may be used to find out if the cancer has spread: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if osteosarcoma spreads to the lung, the cancer cells in the lung are actually osteosarcoma cells. The disease is metastatic osteosarcoma, not lung cancer. The cancer may recur (come back) in the bone or in other parts of the body. Osteosarcoma and UPS most often recur in the lung, bone, or both. When osteosarcoma recurs, it is usually within 18 months after treatment is completed. Different types of treatment are available for children with osteosarcoma or UPS of bone. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating osteosarcoma and UPS and who specialize in certain areas of medicine. These may include the following specialists: For information about types and monitoring of late effects in childhood and adolescent cancer survivors, see Late Effects of Treatment for Childhood Cancer. Surgery to remove the entire tumor will be done when possible. Chemotherapy may be given before surgery to make the tumor smaller. This is called neoadjuvant chemotherapy. Chemotherapy is given so less bone tissue needs to be removed and there are fewer problems after surgery. The following types of surgery may be done: Studies have shown that survival is the same whether the first surgery done is a limb-sparing surgery or an amputation. After the doctor removes all the cancer that can be seen at the time of the surgery, patients are given chemotherapy to kill any cancer cells that are left in the area where the tumor was removed or that have spread to other parts of the body. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is the use of more than one anticancer drug. Chemotherapy is usually given before and after surgery to remove the primary tumor. For more information, see Drugs Approved for Bone Cancer. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Osteosarcoma and UPS cells are not killed easily by external radiation therapy. It may be used when a small amount of cancer is left after surgery or used together with other treatments. Samarium is a radioactive drug that targets areas where bone cells are growing, such as tumor cells in bone. It helps relieve pain caused by cancer in the bone and it also kills blood cells in the bone marrow. It is used to treat osteosarcoma that has come back after treatment in a different bone. Treatment with samarium may be followed by stem cell transplant. Before treatment with samarium, stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After treatment with samarium is complete, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Information about ongoing clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed localized osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Lung Metastasis When osteosarcoma or UPS spreads, it usually spreads to the lung. Treatment of newly diagnosed osteosarcoma and UPS with lung metastasis may include the following: Bone Metastasis or Bone with Lung Metastasis Newly diagnosed osteosarcoma and UPS may spread to a distant bone and/or the lung. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent osteosarcoma and UPS of bone may include the following: Treatment depends on the area and type of recurrence, for example: Clinical trials for treatment of recurrent osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about osteosarcoma and UPS, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of osteosarcoma and undifferentiated pleomorphic sarcoma (UPS) (formerly called malignant fibrous histiocytoma [MFH]) of bone. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Osteosarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/bone/patient/osteosarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389380] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Osteosarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Osteosarcoma and Malignant Fibrous Histiocytoma of Bone ?

Signs and symptoms of osteosarcoma and MFH include swelling over a bone or a bony part of the body and joint pain. These and other signs and symptoms may be caused by osteosarcoma or MFH or by other conditions. Check with a doctor if your child has any of the following: - Swelling over a bone or bony part of the body. - Pain in a bone or joint. - A bone that breaks for no known reason.

Osteosarcoma usually starts in osteoblasts, which are a type of bone cell that becomes new bone. Osteosarcoma is most common in adolescents. It commonly forms in the ends of the long bones of the body, which include bones of the arms and legs. In children and adolescents, it often forms in the long bones, near the knee. Rarely, osteosarcoma may be found in soft tissue or organs in the chest or abdomen. Osteosarcoma is the most common type of bone cancer. UPS (formerly called malignant fibrous histiocytoma [MFH]) is a rare type of bone cancer that usually starts in soft tissue, but it may form in bone. In bone, UPS cells look similar to osteosarcoma under a microscope. UPS is treated like osteosarcoma. Ewing sarcoma is another kind of bone cancer, but it is not covered in this summary. For more information, see Ewing Sarcoma Treatment. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop osteosarcoma, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Risk factors for osteosarcoma include the following: These and other signs and symptoms may be caused by osteosarcoma or UPS or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Imaging tests are done before a biopsy. Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. It is important that the biopsy be done by a surgeon who is an expert in treating cancer of the bone. It is best if that surgeon is also the one who removes the tumor. The biopsy and the surgery to remove the tumor are planned together. The way the biopsy is done affects which type of surgery can be done later. The type of biopsy that is done will be based on the size of the tumor and where it is in the body. There are two types of biopsy that may be used: The following test may be done on the tissue that is removed: The prognosis may be affected by certain factors before and after treatment. The prognosis of untreated osteosarcoma and UPS may depend on the following: After osteosarcoma or UPS is treated, prognosis also depends on the following: Treatment options for osteosarcoma and UPS depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. Most patients with osteosarcoma and UPS are grouped according to whether cancer is localized or metastatic. The following tests and procedures may be used to find out if the cancer has spread: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if osteosarcoma spreads to the lung, the cancer cells in the lung are actually osteosarcoma cells. The disease is metastatic osteosarcoma, not lung cancer. The cancer may recur (come back) in the bone or in other parts of the body. Osteosarcoma and UPS most often recur in the lung, bone, or both. When osteosarcoma recurs, it is usually within 18 months after treatment is completed. Different types of treatment are available for children with osteosarcoma or UPS of bone. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating osteosarcoma and UPS and who specialize in certain areas of medicine. These may include the following specialists: For information about types and monitoring of late effects in childhood and adolescent cancer survivors, see Late Effects of Treatment for Childhood Cancer. Surgery to remove the entire tumor will be done when possible. Chemotherapy may be given before surgery to make the tumor smaller. This is called neoadjuvant chemotherapy. Chemotherapy is given so less bone tissue needs to be removed and there are fewer problems after surgery. The following types of surgery may be done: Studies have shown that survival is the same whether the first surgery done is a limb-sparing surgery or an amputation. After the doctor removes all the cancer that can be seen at the time of the surgery, patients are given chemotherapy to kill any cancer cells that are left in the area where the tumor was removed or that have spread to other parts of the body. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is the use of more than one anticancer drug. Chemotherapy is usually given before and after surgery to remove the primary tumor. For more information, see Drugs Approved for Bone Cancer. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Osteosarcoma and UPS cells are not killed easily by external radiation therapy. It may be used when a small amount of cancer is left after surgery or used together with other treatments. Samarium is a radioactive drug that targets areas where bone cells are growing, such as tumor cells in bone. It helps relieve pain caused by cancer in the bone and it also kills blood cells in the bone marrow. It is used to treat osteosarcoma that has come back after treatment in a different bone. Treatment with samarium may be followed by stem cell transplant. Before treatment with samarium, stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After treatment with samarium is complete, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Information about ongoing clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed localized osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Lung Metastasis When osteosarcoma or UPS spreads, it usually spreads to the lung. Treatment of newly diagnosed osteosarcoma and UPS with lung metastasis may include the following: Bone Metastasis or Bone with Lung Metastasis Newly diagnosed osteosarcoma and UPS may spread to a distant bone and/or the lung. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent osteosarcoma and UPS of bone may include the following: Treatment depends on the area and type of recurrence, for example: Clinical trials for treatment of recurrent osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about osteosarcoma and UPS, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of osteosarcoma and undifferentiated pleomorphic sarcoma (UPS) (formerly called malignant fibrous histiocytoma [MFH]) of bone. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Osteosarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/bone/patient/osteosarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389380] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Osteosarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Osteosarcoma and Malignant Fibrous Histiocytoma of Bone ?

Imaging tests are used to detect (find) osteosarcoma and MFH. Imaging tests are done before the biopsy. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - X-ray : An x-ray of the organs and bones inside the body. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). A biopsy is done to diagnose osteosarcoma. Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. It is important that the biopsy be done by a surgeon who is an expert in treating cancer of the bone. It is best if that surgeon is also the one who removes the tumor. The biopsy and the surgery to remove the tumor are planned together. The way the biopsy is done affects which type of surgery can be done later. The type of biopsy that is done will be based on the size of the tumor and where it is in the body. There are two types of biopsy that may be used: - Core biopsy : The removal of tissue using a wide needle. - Incisional biopsy : The removal of part of a lump or a sample of tissue that doesn&apos;t look normal. The following test may be done on the tissue that is removed: - Light and electron microscopy : A laboratory test in which cells in a sample of tissue are viewed under regular and high-powered microscopes to look for certain changes in the cells.

Osteosarcoma usually starts in osteoblasts, which are a type of bone cell that becomes new bone. Osteosarcoma is most common in adolescents. It commonly forms in the ends of the long bones of the body, which include bones of the arms and legs. In children and adolescents, it often forms in the long bones, near the knee. Rarely, osteosarcoma may be found in soft tissue or organs in the chest or abdomen. Osteosarcoma is the most common type of bone cancer. UPS (formerly called malignant fibrous histiocytoma [MFH]) is a rare type of bone cancer that usually starts in soft tissue, but it may form in bone. In bone, UPS cells look similar to osteosarcoma under a microscope. UPS is treated like osteosarcoma. Ewing sarcoma is another kind of bone cancer, but it is not covered in this summary. For more information, see Ewing Sarcoma Treatment. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop osteosarcoma, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Risk factors for osteosarcoma include the following: These and other signs and symptoms may be caused by osteosarcoma or UPS or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Imaging tests are done before a biopsy. Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. It is important that the biopsy be done by a surgeon who is an expert in treating cancer of the bone. It is best if that surgeon is also the one who removes the tumor. The biopsy and the surgery to remove the tumor are planned together. The way the biopsy is done affects which type of surgery can be done later. The type of biopsy that is done will be based on the size of the tumor and where it is in the body. There are two types of biopsy that may be used: The following test may be done on the tissue that is removed: The prognosis may be affected by certain factors before and after treatment. The prognosis of untreated osteosarcoma and UPS may depend on the following: After osteosarcoma or UPS is treated, prognosis also depends on the following: Treatment options for osteosarcoma and UPS depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. Most patients with osteosarcoma and UPS are grouped according to whether cancer is localized or metastatic. The following tests and procedures may be used to find out if the cancer has spread: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if osteosarcoma spreads to the lung, the cancer cells in the lung are actually osteosarcoma cells. The disease is metastatic osteosarcoma, not lung cancer. The cancer may recur (come back) in the bone or in other parts of the body. Osteosarcoma and UPS most often recur in the lung, bone, or both. When osteosarcoma recurs, it is usually within 18 months after treatment is completed. Different types of treatment are available for children with osteosarcoma or UPS of bone. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating osteosarcoma and UPS and who specialize in certain areas of medicine. These may include the following specialists: For information about types and monitoring of late effects in childhood and adolescent cancer survivors, see Late Effects of Treatment for Childhood Cancer. Surgery to remove the entire tumor will be done when possible. Chemotherapy may be given before surgery to make the tumor smaller. This is called neoadjuvant chemotherapy. Chemotherapy is given so less bone tissue needs to be removed and there are fewer problems after surgery. The following types of surgery may be done: Studies have shown that survival is the same whether the first surgery done is a limb-sparing surgery or an amputation. After the doctor removes all the cancer that can be seen at the time of the surgery, patients are given chemotherapy to kill any cancer cells that are left in the area where the tumor was removed or that have spread to other parts of the body. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is the use of more than one anticancer drug. Chemotherapy is usually given before and after surgery to remove the primary tumor. For more information, see Drugs Approved for Bone Cancer. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Osteosarcoma and UPS cells are not killed easily by external radiation therapy. It may be used when a small amount of cancer is left after surgery or used together with other treatments. Samarium is a radioactive drug that targets areas where bone cells are growing, such as tumor cells in bone. It helps relieve pain caused by cancer in the bone and it also kills blood cells in the bone marrow. It is used to treat osteosarcoma that has come back after treatment in a different bone. Treatment with samarium may be followed by stem cell transplant. Before treatment with samarium, stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After treatment with samarium is complete, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Information about ongoing clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed localized osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Lung Metastasis When osteosarcoma or UPS spreads, it usually spreads to the lung. Treatment of newly diagnosed osteosarcoma and UPS with lung metastasis may include the following: Bone Metastasis or Bone with Lung Metastasis Newly diagnosed osteosarcoma and UPS may spread to a distant bone and/or the lung. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent osteosarcoma and UPS of bone may include the following: Treatment depends on the area and type of recurrence, for example: Clinical trials for treatment of recurrent osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about osteosarcoma and UPS, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of osteosarcoma and undifferentiated pleomorphic sarcoma (UPS) (formerly called malignant fibrous histiocytoma [MFH]) of bone. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Osteosarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/bone/patient/osteosarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389380] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Osteosarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Osteosarcoma and Malignant Fibrous Histiocytoma of Bone ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) is affected by certain factors before and after treatment. The prognosis of untreated osteosarcoma and MFH depends on the following: - Where the tumor is in the body and whether tumors formed in more than one bone. - The size of the tumor. - Whether the cancer has spread to other parts of the body and where it has spread. - The type of tumor (based on how the cancer cells look under a microscope). - The patient&apos;s age and weight at diagnosis. - Whether the tumor has caused a break in the bone. - Whether the patient has certain genetic diseases. After osteosarcoma or MFH is treated, prognosis also depends on the following: - How much of the cancer was killed by chemotherapy. - How much of the tumor was taken out by surgery. - Whether chemotherapy is delayed for more than 3 weeks after surgery takes place. - Whether the cancer has recurred (come back) within 2 years of diagnosis. Treatment options for osteosarcoma and MFH depend on the following: - Where the tumor is in the body. - The size of the tumor. - The stage of the cancer. - Whether the bones are still growing. - The patient&apos;s age and general health. - The desire of the patient and family for the patient to be able to participate in activities such as sports or have a certain appearance. - Whether the cancer is newly diagnosed or has recurred after treatment.

Osteosarcoma usually starts in osteoblasts, which are a type of bone cell that becomes new bone. Osteosarcoma is most common in adolescents. It commonly forms in the ends of the long bones of the body, which include bones of the arms and legs. In children and adolescents, it often forms in the long bones, near the knee. Rarely, osteosarcoma may be found in soft tissue or organs in the chest or abdomen. Osteosarcoma is the most common type of bone cancer. UPS (formerly called malignant fibrous histiocytoma [MFH]) is a rare type of bone cancer that usually starts in soft tissue, but it may form in bone. In bone, UPS cells look similar to osteosarcoma under a microscope. UPS is treated like osteosarcoma. Ewing sarcoma is another kind of bone cancer, but it is not covered in this summary. For more information, see Ewing Sarcoma Treatment. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop osteosarcoma, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Risk factors for osteosarcoma include the following: These and other signs and symptoms may be caused by osteosarcoma or UPS or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Imaging tests are done before a biopsy. Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. It is important that the biopsy be done by a surgeon who is an expert in treating cancer of the bone. It is best if that surgeon is also the one who removes the tumor. The biopsy and the surgery to remove the tumor are planned together. The way the biopsy is done affects which type of surgery can be done later. The type of biopsy that is done will be based on the size of the tumor and where it is in the body. There are two types of biopsy that may be used: The following test may be done on the tissue that is removed: The prognosis may be affected by certain factors before and after treatment. The prognosis of untreated osteosarcoma and UPS may depend on the following: After osteosarcoma or UPS is treated, prognosis also depends on the following: Treatment options for osteosarcoma and UPS depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. Most patients with osteosarcoma and UPS are grouped according to whether cancer is localized or metastatic. The following tests and procedures may be used to find out if the cancer has spread: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if osteosarcoma spreads to the lung, the cancer cells in the lung are actually osteosarcoma cells. The disease is metastatic osteosarcoma, not lung cancer. The cancer may recur (come back) in the bone or in other parts of the body. Osteosarcoma and UPS most often recur in the lung, bone, or both. When osteosarcoma recurs, it is usually within 18 months after treatment is completed. Different types of treatment are available for children with osteosarcoma or UPS of bone. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating osteosarcoma and UPS and who specialize in certain areas of medicine. These may include the following specialists: For information about types and monitoring of late effects in childhood and adolescent cancer survivors, see Late Effects of Treatment for Childhood Cancer. Surgery to remove the entire tumor will be done when possible. Chemotherapy may be given before surgery to make the tumor smaller. This is called neoadjuvant chemotherapy. Chemotherapy is given so less bone tissue needs to be removed and there are fewer problems after surgery. The following types of surgery may be done: Studies have shown that survival is the same whether the first surgery done is a limb-sparing surgery or an amputation. After the doctor removes all the cancer that can be seen at the time of the surgery, patients are given chemotherapy to kill any cancer cells that are left in the area where the tumor was removed or that have spread to other parts of the body. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is the use of more than one anticancer drug. Chemotherapy is usually given before and after surgery to remove the primary tumor. For more information, see Drugs Approved for Bone Cancer. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Osteosarcoma and UPS cells are not killed easily by external radiation therapy. It may be used when a small amount of cancer is left after surgery or used together with other treatments. Samarium is a radioactive drug that targets areas where bone cells are growing, such as tumor cells in bone. It helps relieve pain caused by cancer in the bone and it also kills blood cells in the bone marrow. It is used to treat osteosarcoma that has come back after treatment in a different bone. Treatment with samarium may be followed by stem cell transplant. Before treatment with samarium, stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After treatment with samarium is complete, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Information about ongoing clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed localized osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Lung Metastasis When osteosarcoma or UPS spreads, it usually spreads to the lung. Treatment of newly diagnosed osteosarcoma and UPS with lung metastasis may include the following: Bone Metastasis or Bone with Lung Metastasis Newly diagnosed osteosarcoma and UPS may spread to a distant bone and/or the lung. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent osteosarcoma and UPS of bone may include the following: Treatment depends on the area and type of recurrence, for example: Clinical trials for treatment of recurrent osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about osteosarcoma and UPS, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of osteosarcoma and undifferentiated pleomorphic sarcoma (UPS) (formerly called malignant fibrous histiocytoma [MFH]) of bone. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Osteosarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/bone/patient/osteosarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389380] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Osteosarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Osteosarcoma and Malignant Fibrous Histiocytoma of Bone ?

Key Points - After osteosarcoma or malignant fibrous histiocytoma (MFH) has been diagnosed, tests are done to find out if cancer cells have spread to other parts of the body. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - Osteosarcoma and MFH are described as either localized or metastatic. After osteosarcoma or malignant fibrous histiocytoma (MFH) has been diagnosed, tests are done to find out if cancer cells have spread to other parts of the body. The process used to find out if cancer has spread to other parts of the body is called staging. For osteosarcoma and malignant fibrous histiocytoma (MFH), most patients are grouped according to whether cancer is found in only one part of the body or has spread. The following tests and procedures may be used: - X-ray : An x-ray of the organs, such as the chest, and bones inside the body. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. X-rays will be taken of the chest and the area where the tumor formed. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the chest, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. Pictures will be taken of the chest and the area where the tumor formed. - PET-CT scan : A procedure that combines the pictures from a positron emission tomography (PET) scan and a computed tomography (CT) scan. The PET and CT scans are done at the same time on the same machine. The pictures from both scans are combined to make a more detailed picture than either test would make by itself. A PET scan is a procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). - Bone scan : A procedure to check if there are rapidly dividing cells, such as cancer cells, in the bone. A very small amount of radioactive material is injected into a vein and travels through the bloodstream. The radioactive material collects in the bones and is detected by a scanner. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if osteosarcoma spreads to the lung, the cancer cells in the lung are actually osteosarcoma cells. The disease is metastatic osteosarcoma, not lung cancer. Osteosarcoma and MFH are described as either localized or metastatic. - Localized osteosarcoma or MFH has not spread out of the bone where the cancer started. There may be one or more areas of cancer in the bone that can be removed during surgery. - Metastatic osteosarcoma or MFH has spread from the bone in which the cancer began to other parts of the body. The cancer most often spreads to the lungs. It may also spread to other bones.

Osteosarcoma usually starts in osteoblasts, which are a type of bone cell that becomes new bone. Osteosarcoma is most common in adolescents. It commonly forms in the ends of the long bones of the body, which include bones of the arms and legs. In children and adolescents, it often forms in the long bones, near the knee. Rarely, osteosarcoma may be found in soft tissue or organs in the chest or abdomen. Osteosarcoma is the most common type of bone cancer. UPS (formerly called malignant fibrous histiocytoma [MFH]) is a rare type of bone cancer that usually starts in soft tissue, but it may form in bone. In bone, UPS cells look similar to osteosarcoma under a microscope. UPS is treated like osteosarcoma. Ewing sarcoma is another kind of bone cancer, but it is not covered in this summary. For more information, see Ewing Sarcoma Treatment. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop osteosarcoma, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Risk factors for osteosarcoma include the following: These and other signs and symptoms may be caused by osteosarcoma or UPS or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Imaging tests are done before a biopsy. Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. It is important that the biopsy be done by a surgeon who is an expert in treating cancer of the bone. It is best if that surgeon is also the one who removes the tumor. The biopsy and the surgery to remove the tumor are planned together. The way the biopsy is done affects which type of surgery can be done later. The type of biopsy that is done will be based on the size of the tumor and where it is in the body. There are two types of biopsy that may be used: The following test may be done on the tissue that is removed: The prognosis may be affected by certain factors before and after treatment. The prognosis of untreated osteosarcoma and UPS may depend on the following: After osteosarcoma or UPS is treated, prognosis also depends on the following: Treatment options for osteosarcoma and UPS depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. Most patients with osteosarcoma and UPS are grouped according to whether cancer is localized or metastatic. The following tests and procedures may be used to find out if the cancer has spread: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if osteosarcoma spreads to the lung, the cancer cells in the lung are actually osteosarcoma cells. The disease is metastatic osteosarcoma, not lung cancer. The cancer may recur (come back) in the bone or in other parts of the body. Osteosarcoma and UPS most often recur in the lung, bone, or both. When osteosarcoma recurs, it is usually within 18 months after treatment is completed. Different types of treatment are available for children with osteosarcoma or UPS of bone. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating osteosarcoma and UPS and who specialize in certain areas of medicine. These may include the following specialists: For information about types and monitoring of late effects in childhood and adolescent cancer survivors, see Late Effects of Treatment for Childhood Cancer. Surgery to remove the entire tumor will be done when possible. Chemotherapy may be given before surgery to make the tumor smaller. This is called neoadjuvant chemotherapy. Chemotherapy is given so less bone tissue needs to be removed and there are fewer problems after surgery. The following types of surgery may be done: Studies have shown that survival is the same whether the first surgery done is a limb-sparing surgery or an amputation. After the doctor removes all the cancer that can be seen at the time of the surgery, patients are given chemotherapy to kill any cancer cells that are left in the area where the tumor was removed or that have spread to other parts of the body. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is the use of more than one anticancer drug. Chemotherapy is usually given before and after surgery to remove the primary tumor. For more information, see Drugs Approved for Bone Cancer. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Osteosarcoma and UPS cells are not killed easily by external radiation therapy. It may be used when a small amount of cancer is left after surgery or used together with other treatments. Samarium is a radioactive drug that targets areas where bone cells are growing, such as tumor cells in bone. It helps relieve pain caused by cancer in the bone and it also kills blood cells in the bone marrow. It is used to treat osteosarcoma that has come back after treatment in a different bone. Treatment with samarium may be followed by stem cell transplant. Before treatment with samarium, stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After treatment with samarium is complete, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Information about ongoing clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed localized osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Lung Metastasis When osteosarcoma or UPS spreads, it usually spreads to the lung. Treatment of newly diagnosed osteosarcoma and UPS with lung metastasis may include the following: Bone Metastasis or Bone with Lung Metastasis Newly diagnosed osteosarcoma and UPS may spread to a distant bone and/or the lung. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent osteosarcoma and UPS of bone may include the following: Treatment depends on the area and type of recurrence, for example: Clinical trials for treatment of recurrent osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about osteosarcoma and UPS, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of osteosarcoma and undifferentiated pleomorphic sarcoma (UPS) (formerly called malignant fibrous histiocytoma [MFH]) of bone. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Osteosarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/bone/patient/osteosarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389380] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Osteosarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Osteosarcoma and Malignant Fibrous Histiocytoma of Bone ?

Key Points - There are different types of treatment for patients with osteosarcoma or malignant fibrous histiocytoma (MFH) of bone. - Children with osteosarcoma or MFH should have their treatment planned by a team of health care providers who are experts in treating cancer in children. - Treatment for osteosarcoma or malignant fibrous histiocytoma may cause side effects. - Four types of standard treatment are used: - Surgery - Chemotherapy - Radiation therapy - Samarium - New types of treatment are being tested in clinical trials. - Targeted therapy - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with osteosarcoma or malignant fibrous histiocytoma (MFH) of bone. Different types of treatment are available for children with osteosarcoma or malignant fibrous histiocytoma (MFH) of bone. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Children with osteosarcoma or MFH should have their treatment planned by a team of health care providers who are experts in treating cancer in children. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating osteosarcoma and MFH and who specialize in certain areas of medicine. These may include the following specialists: - Pediatrician. - Orthopedic surgeon. - Radiation oncologist. - Rehabilitation specialist. - Pediatric nurse specialist. - Social worker. - Psychologist. Treatment for osteosarcoma or malignant fibrous histiocytoma may cause side effects. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: - Physical problems. - Changes in mood, feelings, thinking, learning, or memory. - Second cancers (new types of cancer). Some late effects may be treated or controlled. It is important to talk with your child&apos;s doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Four types of standard treatment are used: Surgery Surgery to remove the entire tumor will be done when possible. Chemotherapy may be given before surgery to make the tumor smaller. This is called neoadjuvant chemotherapy. Chemotherapy is given so less bone tissue needs to be removed and there are fewer problems after surgery. The following types of surgery may be done: - Wide local excision: Surgery to remove the cancer and some healthy tissue around it. - Limb-sparing surgery: Removal of the tumor in a limb (arm or leg) without amputation, so the use and appearance of the limb is saved. Most patients with osteosarcoma in a limb can be treated with limb-sparing surgery. The tumor is removed by wide local excision. Tissue and bone that are removed may be replaced with a graft using tissue and bone taken from another part of the patient&apos;s body, or with an implant such as artificial bone. If a fracture is found at diagnosis or during chemotherapy before surgery, limb-sparing surgery may still be possible in some cases. If the surgeon is not able to remove all of the tumor and enough healthy tissue around it, an amputation may be done. - Amputation: Surgery to remove part or all of an arm or leg. This may be done when it is not possible to remove all of the tumor in limb-sparing surgery. The patient may be fitted with a prosthesis (artificial limb) after amputation. - Rotationplasty: Surgery to remove the tumor and the knee joint. The part of the leg that remains below the knee is then attached to the part of the leg that remains above the knee, with the foot facing backward and the ankle acting as a knee. A prosthesis may then be attached to the foot. Studies have shown that survival is the same whether the first surgery done is a limb-sparing surgery or an amputation. Even if the doctor removes all the cancer that can be seen at the time of the surgery, patients are also given chemotherapy after surgery to kill any cancer cells that are left in the area where the tumor was removed or that have spread to other parts of the body. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is the use of more than one anticancer drug. The way the chemotherapy is given depends on the type and stage of the cancer being treated. In the treatment of osteosarcoma and malignant fibrous histiocytosis of bone, chemotherapy is usually given before and after surgery to remove the primary tumor. See Drugs Approved for Bone Cancer for more information. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated. Osteosarcoma and MFH cells are not killed easily by external radiation therapy. It may be used when a small amount of cancer is left after surgery or used together with other treatments. Samarium Samarium is a radioactive drug that targets areas where bone cells are growing, such as tumor cells in bone. It helps relieve pain caused by cancer in the bone and it also kills blood cells in the bone marrow. It also is used to treat osteosarcoma that has come back after treatment in a different bone. Treatment with samarium may be followed by stem cell transplant. Before treatment with samarium, stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After treatment with samarium is complete, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body&apos;s blood cells. New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about ongoing clinical trials is available from the NCI website. Targeted therapy Targeted therapy is a treatment that uses drugs or other substances to find and attack specific cancer cells without harming normal cells. Kinase inhibitor therapy and monoclonal antibody therapy are types of targeted therapy being studied in clinical trials for osteosarcoma. Kinase inhibitor therapy blocks a protein needed for cancer cells to divide. Sorafenib is a type of kinase inhibitor therapy being studied for the treatment of recurrent osteosarcoma. Monoclonal antibody therapy is a cancer treatment that uses antibodies made in the laboratory, from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Denosumab, dinutuximab, and glembatumumab are monoclonal antibodies being studied for the treatment of recurrent osteosarcoma. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child&apos;s condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Osteosarcoma and Malignant Fibrous Histiocytoma of Bone Localized Osteosarcoma and Malignant Fibrous Histiocytoma of Bone Treatment may include the following: - Surgery. Combination chemotherapy is usually given before and after surgery. - Surgery followed by radiation therapy when the tumor cannot be completely removed by surgery. Check the list of NCI-supported cancer clinical trials that are now accepting patients with localized osteosarcoma and localized childhood malignant fibrous histiocytoma of bone. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Metastatic Osteosarcoma and Malignant Fibrous Histiocytoma of Bone Lung Metastasis When osteosarcoma or malignant fibrous histiocytoma (MFH) spreads, it usually spreads to the lung. Treatment of osteosarcoma and MFH with lung metastasis may include the following: - Combination chemotherapy followed by surgery to remove the primary cancer and the cancer that has spread to the lung. Bone Metastasis or Bone with Lung Metastasis Osteosarcoma and malignant fibrous histiocytoma may spread to a distant bone and/or the lung. Treatment may include the following: - Combination chemotherapy followed by surgery to remove the primary tumor and the cancer that has spread to other parts of the body. More chemotherapy is given after surgery. - Surgery to remove the primary tumor followed by chemotherapy and surgery to remove cancer that has spread to other parts of the body. Check the list of NCI-supported cancer clinical trials that are now accepting patients with metastatic osteosarcoma and metastatic childhood malignant fibrous histiocytoma of bone. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Recurrent Osteosarcoma and Malignant Fibrous Histiocytoma of Bone Treatment of recurrent osteosarcoma and malignant fibrous histiocytoma of bone may include the following: - Surgery to remove the tumor with or without chemotherapy. - Samarium with or without stem cell transplant using the patient&apos;s own stem cells, as palliative treatment to relieve pain and improve the quality of life. - A clinical trial of new types of treatment for patients whose cancer cannot be removed by surgery. These may include targeted therapy such as kinase inhibitor therapy or monoclonal antibody therapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with recurrent osteosarcoma and recurrent childhood malignant fibrous histiocytoma of bone. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website.

Osteosarcoma usually starts in osteoblasts, which are a type of bone cell that becomes new bone. Osteosarcoma is most common in adolescents. It commonly forms in the ends of the long bones of the body, which include bones of the arms and legs. In children and adolescents, it often forms in the long bones, near the knee. Rarely, osteosarcoma may be found in soft tissue or organs in the chest or abdomen. Osteosarcoma is the most common type of bone cancer. UPS (formerly called malignant fibrous histiocytoma [MFH]) is a rare type of bone cancer that usually starts in soft tissue, but it may form in bone. In bone, UPS cells look similar to osteosarcoma under a microscope. UPS is treated like osteosarcoma. Ewing sarcoma is another kind of bone cancer, but it is not covered in this summary. For more information, see Ewing Sarcoma Treatment. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop osteosarcoma, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Risk factors for osteosarcoma include the following: These and other signs and symptoms may be caused by osteosarcoma or UPS or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Imaging tests are done before a biopsy. Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. It is important that the biopsy be done by a surgeon who is an expert in treating cancer of the bone. It is best if that surgeon is also the one who removes the tumor. The biopsy and the surgery to remove the tumor are planned together. The way the biopsy is done affects which type of surgery can be done later. The type of biopsy that is done will be based on the size of the tumor and where it is in the body. There are two types of biopsy that may be used: The following test may be done on the tissue that is removed: The prognosis may be affected by certain factors before and after treatment. The prognosis of untreated osteosarcoma and UPS may depend on the following: After osteosarcoma or UPS is treated, prognosis also depends on the following: Treatment options for osteosarcoma and UPS depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. Most patients with osteosarcoma and UPS are grouped according to whether cancer is localized or metastatic. The following tests and procedures may be used to find out if the cancer has spread: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if osteosarcoma spreads to the lung, the cancer cells in the lung are actually osteosarcoma cells. The disease is metastatic osteosarcoma, not lung cancer. The cancer may recur (come back) in the bone or in other parts of the body. Osteosarcoma and UPS most often recur in the lung, bone, or both. When osteosarcoma recurs, it is usually within 18 months after treatment is completed. Different types of treatment are available for children with osteosarcoma or UPS of bone. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating osteosarcoma and UPS and who specialize in certain areas of medicine. These may include the following specialists: For information about types and monitoring of late effects in childhood and adolescent cancer survivors, see Late Effects of Treatment for Childhood Cancer. Surgery to remove the entire tumor will be done when possible. Chemotherapy may be given before surgery to make the tumor smaller. This is called neoadjuvant chemotherapy. Chemotherapy is given so less bone tissue needs to be removed and there are fewer problems after surgery. The following types of surgery may be done: Studies have shown that survival is the same whether the first surgery done is a limb-sparing surgery or an amputation. After the doctor removes all the cancer that can be seen at the time of the surgery, patients are given chemotherapy to kill any cancer cells that are left in the area where the tumor was removed or that have spread to other parts of the body. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is the use of more than one anticancer drug. Chemotherapy is usually given before and after surgery to remove the primary tumor. For more information, see Drugs Approved for Bone Cancer. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Osteosarcoma and UPS cells are not killed easily by external radiation therapy. It may be used when a small amount of cancer is left after surgery or used together with other treatments. Samarium is a radioactive drug that targets areas where bone cells are growing, such as tumor cells in bone. It helps relieve pain caused by cancer in the bone and it also kills blood cells in the bone marrow. It is used to treat osteosarcoma that has come back after treatment in a different bone. Treatment with samarium may be followed by stem cell transplant. Before treatment with samarium, stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After treatment with samarium is complete, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Information about ongoing clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed localized osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Lung Metastasis When osteosarcoma or UPS spreads, it usually spreads to the lung. Treatment of newly diagnosed osteosarcoma and UPS with lung metastasis may include the following: Bone Metastasis or Bone with Lung Metastasis Newly diagnosed osteosarcoma and UPS may spread to a distant bone and/or the lung. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent osteosarcoma and UPS of bone may include the following: Treatment depends on the area and type of recurrence, for example: Clinical trials for treatment of recurrent osteosarcoma and UPS of bone may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about osteosarcoma and UPS, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of osteosarcoma and undifferentiated pleomorphic sarcoma (UPS) (formerly called malignant fibrous histiocytoma [MFH]) of bone. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Osteosarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/bone/patient/osteosarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389380] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Osteosarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Osteosarcoma and Malignant Fibrous Histiocytoma of Bone ?

New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about ongoing clinical trials is available from the NCI website. Targeted therapy Targeted therapy is a treatment that uses drugs or other substances to find and attack specific cancer cells without harming normal cells. Kinase inhibitor therapy and monoclonal antibody therapy are types of targeted therapy being studied in clinical trials for osteosarcoma. Kinase inhibitor therapy blocks a protein needed for cancer cells to divide. Sorafenib is a type of kinase inhibitor therapy being studied for the treatment of recurrent osteosarcoma. Monoclonal antibody therapy is a cancer treatment that uses antibodies made in the laboratory, from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Denosumab, dinutuximab, and glembatumumab are monoclonal antibodies being studied for the treatment of recurrent osteosarcoma. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child&apos;s condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups.

The breast is made up of lobes and ducts. Each breast has 15 to 20 sections called lobes. Each lobe has many smaller sections called lobules. Lobules end in dozens of tiny bulbs that can make milk. The lobes, lobules, and bulbs are linked by thin tubes called ducts. EnlargeAnatomy of the female breast. The nipple and areola are shown on the outside of the breast. The lymph nodes, lobes, lobules, ducts, and other parts of the inside of the breast are also shown. Each breast also has blood vessels and lymph vessels. The lymph vessels carry an almost colorless, watery fluid called lymph. Lymph vessels carry lymph between lymph nodes. Lymph nodes are small, bean-shaped structures found throughout the body. They filter lymph and store white blood cells that help fight infection and disease. Groups of lymph nodes are found near the breast in the axilla (under the arm), above the collarbone, and in the chest. Breast cancer occurs about once in every 3,000 pregnancies. It occurs most often in women aged 32 to 38 years. Because many women are choosing to delay having children, it is likely that the number of new cases of breast cancer during pregnancy will increase. These and other signs may be caused by breast cancer or by other conditions. Check with your doctor if you have any of the following: The breasts usually get larger, tender, or lumpy in women who are pregnant, nursing, or have just given birth. This occurs because of normal hormone changes that take place during pregnancy. These changes can make small lumps difficult to detect. The breasts may also become denser. It is more difficult to detect breast cancer in women with dense breasts using mammography. Because these breast changes can delay diagnosis, breast cancer is often found at a later stage in these women. To detect breast cancer, pregnant and nursing women should examine their breasts themselves. Women should also receive clinical breast exams during their regular prenatal and postnatal check-ups. Talk to your doctor if you notice any changes in your breasts that you do not expect or that worry you. The following tests and procedures may be used: There are three types of breast biopsies: Decisions about the best treatment are based on the results of these tests and the trimester of the pregnancy. The tests give information about: Tests may include the following: The prognosis and treatment options depend on the following: The process used to find out if the cancer has spread within the breast or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some procedures may expose the fetus to harmful radiation or dyes. These procedures are done only if absolutely necessary. Certain actions, such as using a lead-lined shield to cover the abdomen, are used to help protect the fetus from radiation as much as possible. The following tests and procedures may be used to stage breast cancer during pregnancy: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if breast cancer spreads to the bone, the cancer cells in the bone are actually breast cancer cells. The disease is metastatic breast cancer, not bone cancer. To plan the best treatment and understand your prognosis, it is important to know the breast cancer stage. There are 3 types of breast cancer stage groups: For breast cancer, the TNM system describes the tumor as follows: When the lymph nodes are removed by surgery and studied under a microscope by a pathologist, pathologic staging is used to describe the lymph nodes. The pathologic staging of lymph nodes is described below. or cancer has spread to 4 to 9 axillary lymph nodes and cancer in at least one of the lymph nodes is larger than 2 millimeters. Cancer has also spread to lymph nodes near the breastbone on the same side of the body as the primary tumor, and the cancer is larger than 0.2 millimeters and is found by sentinel lymph node biopsy. When the lymph nodes are checked using mammography or ultrasound, it is called clinical staging. The clinical staging of lymph nodes is not described here. The grading system describes a tumor based on how abnormal the cancer cells and tissue look under a microscope and how quickly the cancer cells are likely to grow and spread. Low-grade cancer cells look more like normal cells and tend to grow and spread more slowly than high-grade cancer cells. To describe how abnormal the cancer cells and tissue are, the pathologist will assess the following three features: For each feature, the pathologist assigns a score of 1 to 3; a score of “1” means the cells and tumor tissue look the most like normal cells and tissue, and a score of “3” means the cells and tissue look the most abnormal. The scores for each feature are added together to get a total score between 3 and 9. Three grades are possible: Healthy breast cells, and some breast cancer cells, have receptors (biomarkers) that attach to the hormones estrogen and progesterone. These hormones are needed for healthy cells, and some breast cancer cells, to grow and divide. To check for these biomarkers, samples of tissue containing breast cancer cells are removed during a biopsy or surgery. The samples are tested in a laboratory to see whether the breast cancer cells have estrogen or progesterone receptors. Another type of receptor (biomarker) that is found on the surface of all breast cancer cells is called HER2. HER2 receptors are needed for the breast cancer cells to grow and divide. For breast cancer, biomarker testing includes the following: Sometimes the breast cancer cells will be described as triple negative or triple positive. It is important to know the estrogen receptor, progesterone receptor, and HER2 receptor status to choose the best treatment. There are drugs that can stop the receptors from attaching to the hormones estrogen and progesterone and stop the cancer from growing. Other drugs may be used to block the HER2 receptors on the surface of the breast cancer cells and stop the cancer from growing. Here are 3 examples that combine the TNM system, the grading system, and the biomarker status to find out the Pathological Prognostic breast cancer stage for a woman whose first treatment was surgery: If the tumor size is 30 millimeters (T2), has not spread to nearby lymph nodes (N0), has not spread to distant parts of the body (M0), and is: The cancer is stage IIA. If the tumor size is 53 millimeters (T3), has spread to 4 to 9 axillary lymph nodes (N2), has not spread to other parts of the body (M0), and is: The tumor is stage IIIA. If the tumor size is 65 millimeters (T3), has spread to 3 axillary lymph nodes (N1a), has spread to the lungs (M1), and is: The cancer is stage IV (metastatic breast cancer). After surgery, your doctor will receive a pathology report that describes the size and location of the primary tumor, the spread of cancer to nearby lymph nodes, tumor grade, and whether certain biomarkers are present. The pathology report and other test results are used to determine your breast cancer stage. You are likely to have many questions. Ask your doctor to explain how staging is used to decide the best options to treat your cancer and whether there are clinical trials that might be right for you. Most pregnant women with breast cancer have surgery to remove the breast. Some of the lymph nodes under the arm may be removed so they can be checked under a microscope by a pathologist for signs of cancer. Types of surgery to remove the cancer include: After the doctor removes all of the cancer that can be seen at the time of surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. For pregnant women with early-stage breast cancer, radiation therapy and hormone therapy are given after the baby is born. Treatment given after surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be given to pregnant women with early stage (stage I or II) breast cancer after the baby is born. Women with late stage (stage III or IV) breast cancer may be given external radiation therapy after the first 3 months of pregnancy or, if possible, radiation therapy is delayed until after the baby is born. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy is usually not given during the first 3 months of pregnancy. Chemotherapy given after this time does not usually harm the fetus but may cause early labor or low birth weight. See Drugs Approved for Breast Cancer for more information. Because ending the pregnancy is not likely to improve the mother’s chance of survival, it is not usually a treatment option. For information about side effects caused by treatment for cancer, see our Side Effects page. For information about the treatments listed below, see the Treatment Option Overview section. Pregnant women with early-stage breast cancer (stage I and stage II) are usually treated in the same way as patients who are not pregnant, with some changes to protect the fetus. Treatment may include the following: Hormone therapy and trastuzumab should not be given during pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for patients with late-stage breast cancer (stage III or stage IV) during pregnancy. Treatment may include the following: Radiation therapy and chemotherapy should not be given during the first 3 months of pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. If surgery is planned, breast-feeding should be stopped to reduce blood flow in the breasts and make them smaller. Many chemotherapy drugs, especially cyclophosphamide and methotrexate, may occur in high levels in breast milk and may harm the nursing baby. Women receiving chemotherapy should not breast-feed. Stopping lactation does not improve the mother's prognosis. Breast cancer cells do not seem to pass from the mother to the fetus. For women who have had breast cancer, pregnancy does not seem to affect their survival. However, some doctors recommend that a woman wait 2 years after treatment for breast cancer before trying to have a baby, so that any early return of the cancer would be detected. This may affect a woman’s decision to become pregnant. For more information from the National Cancer Institute about breast cancer during pregnancy, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of breast cancer during pregnancy. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Breast Cancer Treatment During Pregnancy. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/breast/patient/pregnancy-breast-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389161] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Breast Cancer Treatment During Pregnancy (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Breast Cancer ?

Key Points - Breast cancer is a disease in which malignant (cancer) cells form in the tissues of the breast. - Sometimes breast cancer occurs in women who are pregnant or have just given birth. - Signs of breast cancer include a lump or change in the breast. - It may be difficult to detect (find) breast cancer early in pregnant or nursing women. - Breast exams should be part of prenatal and postnatal care. - Tests that examine the breasts are used to detect (find) and diagnose breast cancer. - If cancer is found, tests are done to study the cancer cells. - Certain factors affect prognosis (chance of recovery) and treatment options. Breast cancer is a disease in which malignant (cancer) cells form in the tissues of the breast. The breast is made up of lobes and ducts. Each breast has 15 to 20 sections called lobes. Each lobe has many smaller sections called lobules. Lobules end in dozens of tiny bulbs that can make milk. The lobes, lobules, and bulbs are linked by thin tubes called ducts. Each breast also has blood vessels and lymph vessels. The lymph vessels carry an almost colorless fluid called lymph. Lymph vessels carry lymph between lymph nodes. Lymph nodes are small bean-shaped structures that are found throughout the body. They filter substances in lymph and help fight infection and disease. Clusters of lymph nodes are found near the breast in the axilla (under the arm), above the collarbone, and in the chest. It may be difficult to detect (find) breast cancer early in pregnant or nursing women. The breasts usually get larger, tender, or lumpy in women who are pregnant, nursing, or have just given birth. This occurs because of normal hormone changes that take place during pregnancy. These changes can make small lumps difficult to detect. The breasts may also become denser. It is more difficult to detect breast cancer in women with dense breasts using mammography. Because these breast changes can delay diagnosis, breast cancer is often found at a later stage in these women. Other Information About Pregnancy and Breast Cancer Key Points - Lactation (breast milk production) and breast-feeding should be stopped if surgery or chemotherapy is planned. - Breast cancer does not appear to harm the unborn baby. - Pregnancy does not seem to affect the survival of women who have had breast cancer in the past. Lactation (breast milk production) and breast-feeding should be stopped if surgery or chemotherapy is planned. If surgery is planned, breast-feeding should be stopped to reduce blood flow in the breasts and make them smaller. Breast-feeding should also be stopped if chemotherapy is planned. Many anticancer drugs, especially cyclophosphamide and methotrexate, may occur in high levels in breast milk and may harm the nursing baby. Women receiving chemotherapy should not breast-feed. Stopping lactation does not improve the mother&apos;s prognosis. Breast cancer does not appear to harm the unborn baby. Breast cancer cells do not seem to pass from the mother to the unborn baby. Pregnancy does not seem to affect the survival of women who have had breast cancer in the past. For women who have had breast cancer, pregnancy does not seem to affect their survival. However, some doctors recommend that a woman wait 2 years after treatment for breast cancer before trying to have a baby, so that any early return of the cancer would be detected. This may affect a womans decision to become pregnant. The unborn baby does not seem to be affected if the mother has had breast cancer.

The breast is made up of lobes and ducts. Each breast has 15 to 20 sections called lobes. Each lobe has many smaller sections called lobules. Lobules end in dozens of tiny bulbs that can make milk. The lobes, lobules, and bulbs are linked by thin tubes called ducts. EnlargeAnatomy of the female breast. The nipple and areola are shown on the outside of the breast. The lymph nodes, lobes, lobules, ducts, and other parts of the inside of the breast are also shown. Each breast also has blood vessels and lymph vessels. The lymph vessels carry an almost colorless, watery fluid called lymph. Lymph vessels carry lymph between lymph nodes. Lymph nodes are small, bean-shaped structures found throughout the body. They filter lymph and store white blood cells that help fight infection and disease. Groups of lymph nodes are found near the breast in the axilla (under the arm), above the collarbone, and in the chest. Breast cancer occurs about once in every 3,000 pregnancies. It occurs most often in women aged 32 to 38 years. Because many women are choosing to delay having children, it is likely that the number of new cases of breast cancer during pregnancy will increase. These and other signs may be caused by breast cancer or by other conditions. Check with your doctor if you have any of the following: The breasts usually get larger, tender, or lumpy in women who are pregnant, nursing, or have just given birth. This occurs because of normal hormone changes that take place during pregnancy. These changes can make small lumps difficult to detect. The breasts may also become denser. It is more difficult to detect breast cancer in women with dense breasts using mammography. Because these breast changes can delay diagnosis, breast cancer is often found at a later stage in these women. To detect breast cancer, pregnant and nursing women should examine their breasts themselves. Women should also receive clinical breast exams during their regular prenatal and postnatal check-ups. Talk to your doctor if you notice any changes in your breasts that you do not expect or that worry you. The following tests and procedures may be used: There are three types of breast biopsies: Decisions about the best treatment are based on the results of these tests and the trimester of the pregnancy. The tests give information about: Tests may include the following: The prognosis and treatment options depend on the following: The process used to find out if the cancer has spread within the breast or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some procedures may expose the fetus to harmful radiation or dyes. These procedures are done only if absolutely necessary. Certain actions, such as using a lead-lined shield to cover the abdomen, are used to help protect the fetus from radiation as much as possible. The following tests and procedures may be used to stage breast cancer during pregnancy: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if breast cancer spreads to the bone, the cancer cells in the bone are actually breast cancer cells. The disease is metastatic breast cancer, not bone cancer. To plan the best treatment and understand your prognosis, it is important to know the breast cancer stage. There are 3 types of breast cancer stage groups: For breast cancer, the TNM system describes the tumor as follows: When the lymph nodes are removed by surgery and studied under a microscope by a pathologist, pathologic staging is used to describe the lymph nodes. The pathologic staging of lymph nodes is described below. or cancer has spread to 4 to 9 axillary lymph nodes and cancer in at least one of the lymph nodes is larger than 2 millimeters. Cancer has also spread to lymph nodes near the breastbone on the same side of the body as the primary tumor, and the cancer is larger than 0.2 millimeters and is found by sentinel lymph node biopsy. When the lymph nodes are checked using mammography or ultrasound, it is called clinical staging. The clinical staging of lymph nodes is not described here. The grading system describes a tumor based on how abnormal the cancer cells and tissue look under a microscope and how quickly the cancer cells are likely to grow and spread. Low-grade cancer cells look more like normal cells and tend to grow and spread more slowly than high-grade cancer cells. To describe how abnormal the cancer cells and tissue are, the pathologist will assess the following three features: For each feature, the pathologist assigns a score of 1 to 3; a score of “1” means the cells and tumor tissue look the most like normal cells and tissue, and a score of “3” means the cells and tissue look the most abnormal. The scores for each feature are added together to get a total score between 3 and 9. Three grades are possible: Healthy breast cells, and some breast cancer cells, have receptors (biomarkers) that attach to the hormones estrogen and progesterone. These hormones are needed for healthy cells, and some breast cancer cells, to grow and divide. To check for these biomarkers, samples of tissue containing breast cancer cells are removed during a biopsy or surgery. The samples are tested in a laboratory to see whether the breast cancer cells have estrogen or progesterone receptors. Another type of receptor (biomarker) that is found on the surface of all breast cancer cells is called HER2. HER2 receptors are needed for the breast cancer cells to grow and divide. For breast cancer, biomarker testing includes the following: Sometimes the breast cancer cells will be described as triple negative or triple positive. It is important to know the estrogen receptor, progesterone receptor, and HER2 receptor status to choose the best treatment. There are drugs that can stop the receptors from attaching to the hormones estrogen and progesterone and stop the cancer from growing. Other drugs may be used to block the HER2 receptors on the surface of the breast cancer cells and stop the cancer from growing. Here are 3 examples that combine the TNM system, the grading system, and the biomarker status to find out the Pathological Prognostic breast cancer stage for a woman whose first treatment was surgery: If the tumor size is 30 millimeters (T2), has not spread to nearby lymph nodes (N0), has not spread to distant parts of the body (M0), and is: The cancer is stage IIA. If the tumor size is 53 millimeters (T3), has spread to 4 to 9 axillary lymph nodes (N2), has not spread to other parts of the body (M0), and is: The tumor is stage IIIA. If the tumor size is 65 millimeters (T3), has spread to 3 axillary lymph nodes (N1a), has spread to the lungs (M1), and is: The cancer is stage IV (metastatic breast cancer). After surgery, your doctor will receive a pathology report that describes the size and location of the primary tumor, the spread of cancer to nearby lymph nodes, tumor grade, and whether certain biomarkers are present. The pathology report and other test results are used to determine your breast cancer stage. You are likely to have many questions. Ask your doctor to explain how staging is used to decide the best options to treat your cancer and whether there are clinical trials that might be right for you. Most pregnant women with breast cancer have surgery to remove the breast. Some of the lymph nodes under the arm may be removed so they can be checked under a microscope by a pathologist for signs of cancer. Types of surgery to remove the cancer include: After the doctor removes all of the cancer that can be seen at the time of surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. For pregnant women with early-stage breast cancer, radiation therapy and hormone therapy are given after the baby is born. Treatment given after surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be given to pregnant women with early stage (stage I or II) breast cancer after the baby is born. Women with late stage (stage III or IV) breast cancer may be given external radiation therapy after the first 3 months of pregnancy or, if possible, radiation therapy is delayed until after the baby is born. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy is usually not given during the first 3 months of pregnancy. Chemotherapy given after this time does not usually harm the fetus but may cause early labor or low birth weight. See Drugs Approved for Breast Cancer for more information. Because ending the pregnancy is not likely to improve the mother’s chance of survival, it is not usually a treatment option. For information about side effects caused by treatment for cancer, see our Side Effects page. For information about the treatments listed below, see the Treatment Option Overview section. Pregnant women with early-stage breast cancer (stage I and stage II) are usually treated in the same way as patients who are not pregnant, with some changes to protect the fetus. Treatment may include the following: Hormone therapy and trastuzumab should not be given during pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for patients with late-stage breast cancer (stage III or stage IV) during pregnancy. Treatment may include the following: Radiation therapy and chemotherapy should not be given during the first 3 months of pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. If surgery is planned, breast-feeding should be stopped to reduce blood flow in the breasts and make them smaller. Many chemotherapy drugs, especially cyclophosphamide and methotrexate, may occur in high levels in breast milk and may harm the nursing baby. Women receiving chemotherapy should not breast-feed. Stopping lactation does not improve the mother's prognosis. Breast cancer cells do not seem to pass from the mother to the fetus. For women who have had breast cancer, pregnancy does not seem to affect their survival. However, some doctors recommend that a woman wait 2 years after treatment for breast cancer before trying to have a baby, so that any early return of the cancer would be detected. This may affect a woman’s decision to become pregnant. For more information from the National Cancer Institute about breast cancer during pregnancy, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of breast cancer during pregnancy. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Breast Cancer Treatment During Pregnancy. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/breast/patient/pregnancy-breast-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389161] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Breast Cancer Treatment During Pregnancy (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Breast Cancer? ?

Sometimes breast cancer occurs in women who are pregnant or have just given birth. Breast cancer occurs about once in every 3,000 pregnancies. It occurs most often between the ages of 32 and 38.

The breast is made up of lobes and ducts. Each breast has 15 to 20 sections called lobes. Each lobe has many smaller sections called lobules. Lobules end in dozens of tiny bulbs that can make milk. The lobes, lobules, and bulbs are linked by thin tubes called ducts. EnlargeAnatomy of the female breast. The nipple and areola are shown on the outside of the breast. The lymph nodes, lobes, lobules, ducts, and other parts of the inside of the breast are also shown. Each breast also has blood vessels and lymph vessels. The lymph vessels carry an almost colorless, watery fluid called lymph. Lymph vessels carry lymph between lymph nodes. Lymph nodes are small, bean-shaped structures found throughout the body. They filter lymph and store white blood cells that help fight infection and disease. Groups of lymph nodes are found near the breast in the axilla (under the arm), above the collarbone, and in the chest. Breast cancer occurs about once in every 3,000 pregnancies. It occurs most often in women aged 32 to 38 years. Because many women are choosing to delay having children, it is likely that the number of new cases of breast cancer during pregnancy will increase. These and other signs may be caused by breast cancer or by other conditions. Check with your doctor if you have any of the following: The breasts usually get larger, tender, or lumpy in women who are pregnant, nursing, or have just given birth. This occurs because of normal hormone changes that take place during pregnancy. These changes can make small lumps difficult to detect. The breasts may also become denser. It is more difficult to detect breast cancer in women with dense breasts using mammography. Because these breast changes can delay diagnosis, breast cancer is often found at a later stage in these women. To detect breast cancer, pregnant and nursing women should examine their breasts themselves. Women should also receive clinical breast exams during their regular prenatal and postnatal check-ups. Talk to your doctor if you notice any changes in your breasts that you do not expect or that worry you. The following tests and procedures may be used: There are three types of breast biopsies: Decisions about the best treatment are based on the results of these tests and the trimester of the pregnancy. The tests give information about: Tests may include the following: The prognosis and treatment options depend on the following: The process used to find out if the cancer has spread within the breast or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some procedures may expose the fetus to harmful radiation or dyes. These procedures are done only if absolutely necessary. Certain actions, such as using a lead-lined shield to cover the abdomen, are used to help protect the fetus from radiation as much as possible. The following tests and procedures may be used to stage breast cancer during pregnancy: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if breast cancer spreads to the bone, the cancer cells in the bone are actually breast cancer cells. The disease is metastatic breast cancer, not bone cancer. To plan the best treatment and understand your prognosis, it is important to know the breast cancer stage. There are 3 types of breast cancer stage groups: For breast cancer, the TNM system describes the tumor as follows: When the lymph nodes are removed by surgery and studied under a microscope by a pathologist, pathologic staging is used to describe the lymph nodes. The pathologic staging of lymph nodes is described below. or cancer has spread to 4 to 9 axillary lymph nodes and cancer in at least one of the lymph nodes is larger than 2 millimeters. Cancer has also spread to lymph nodes near the breastbone on the same side of the body as the primary tumor, and the cancer is larger than 0.2 millimeters and is found by sentinel lymph node biopsy. When the lymph nodes are checked using mammography or ultrasound, it is called clinical staging. The clinical staging of lymph nodes is not described here. The grading system describes a tumor based on how abnormal the cancer cells and tissue look under a microscope and how quickly the cancer cells are likely to grow and spread. Low-grade cancer cells look more like normal cells and tend to grow and spread more slowly than high-grade cancer cells. To describe how abnormal the cancer cells and tissue are, the pathologist will assess the following three features: For each feature, the pathologist assigns a score of 1 to 3; a score of “1” means the cells and tumor tissue look the most like normal cells and tissue, and a score of “3” means the cells and tissue look the most abnormal. The scores for each feature are added together to get a total score between 3 and 9. Three grades are possible: Healthy breast cells, and some breast cancer cells, have receptors (biomarkers) that attach to the hormones estrogen and progesterone. These hormones are needed for healthy cells, and some breast cancer cells, to grow and divide. To check for these biomarkers, samples of tissue containing breast cancer cells are removed during a biopsy or surgery. The samples are tested in a laboratory to see whether the breast cancer cells have estrogen or progesterone receptors. Another type of receptor (biomarker) that is found on the surface of all breast cancer cells is called HER2. HER2 receptors are needed for the breast cancer cells to grow and divide. For breast cancer, biomarker testing includes the following: Sometimes the breast cancer cells will be described as triple negative or triple positive. It is important to know the estrogen receptor, progesterone receptor, and HER2 receptor status to choose the best treatment. There are drugs that can stop the receptors from attaching to the hormones estrogen and progesterone and stop the cancer from growing. Other drugs may be used to block the HER2 receptors on the surface of the breast cancer cells and stop the cancer from growing. Here are 3 examples that combine the TNM system, the grading system, and the biomarker status to find out the Pathological Prognostic breast cancer stage for a woman whose first treatment was surgery: If the tumor size is 30 millimeters (T2), has not spread to nearby lymph nodes (N0), has not spread to distant parts of the body (M0), and is: The cancer is stage IIA. If the tumor size is 53 millimeters (T3), has spread to 4 to 9 axillary lymph nodes (N2), has not spread to other parts of the body (M0), and is: The tumor is stage IIIA. If the tumor size is 65 millimeters (T3), has spread to 3 axillary lymph nodes (N1a), has spread to the lungs (M1), and is: The cancer is stage IV (metastatic breast cancer). After surgery, your doctor will receive a pathology report that describes the size and location of the primary tumor, the spread of cancer to nearby lymph nodes, tumor grade, and whether certain biomarkers are present. The pathology report and other test results are used to determine your breast cancer stage. You are likely to have many questions. Ask your doctor to explain how staging is used to decide the best options to treat your cancer and whether there are clinical trials that might be right for you. Most pregnant women with breast cancer have surgery to remove the breast. Some of the lymph nodes under the arm may be removed so they can be checked under a microscope by a pathologist for signs of cancer. Types of surgery to remove the cancer include: After the doctor removes all of the cancer that can be seen at the time of surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. For pregnant women with early-stage breast cancer, radiation therapy and hormone therapy are given after the baby is born. Treatment given after surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be given to pregnant women with early stage (stage I or II) breast cancer after the baby is born. Women with late stage (stage III or IV) breast cancer may be given external radiation therapy after the first 3 months of pregnancy or, if possible, radiation therapy is delayed until after the baby is born. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy is usually not given during the first 3 months of pregnancy. Chemotherapy given after this time does not usually harm the fetus but may cause early labor or low birth weight. See Drugs Approved for Breast Cancer for more information. Because ending the pregnancy is not likely to improve the mother’s chance of survival, it is not usually a treatment option. For information about side effects caused by treatment for cancer, see our Side Effects page. For information about the treatments listed below, see the Treatment Option Overview section. Pregnant women with early-stage breast cancer (stage I and stage II) are usually treated in the same way as patients who are not pregnant, with some changes to protect the fetus. Treatment may include the following: Hormone therapy and trastuzumab should not be given during pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for patients with late-stage breast cancer (stage III or stage IV) during pregnancy. Treatment may include the following: Radiation therapy and chemotherapy should not be given during the first 3 months of pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. If surgery is planned, breast-feeding should be stopped to reduce blood flow in the breasts and make them smaller. Many chemotherapy drugs, especially cyclophosphamide and methotrexate, may occur in high levels in breast milk and may harm the nursing baby. Women receiving chemotherapy should not breast-feed. Stopping lactation does not improve the mother's prognosis. Breast cancer cells do not seem to pass from the mother to the fetus. For women who have had breast cancer, pregnancy does not seem to affect their survival. However, some doctors recommend that a woman wait 2 years after treatment for breast cancer before trying to have a baby, so that any early return of the cancer would be detected. This may affect a woman’s decision to become pregnant. For more information from the National Cancer Institute about breast cancer during pregnancy, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of breast cancer during pregnancy. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Breast Cancer Treatment During Pregnancy. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/breast/patient/pregnancy-breast-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389161] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Breast Cancer Treatment During Pregnancy (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Breast Cancer ?

Signs of breast cancer include a lump or change in the breast. These and other signs may be caused by breast cancer or by other conditions. Check with your doctor if you have any of the following: - A lump or thickening in or near the breast or in the underarm area. - A change in the size or shape of the breast. - A dimple or puckering in the skin of the breast. - A nipple turned inward into the breast. - Fluid, other than breast milk, from the nipple, especially if it&apos;s bloody. - Scaly, red, or swollen skin on the breast, nipple, or areola (the dark area of skin around the nipple). - Dimples in the breast that look like the skin of an orange, called peau dorange. It may be difficult to detect (find) breast cancer early in pregnant or nursing women. The breasts usually get larger, tender, or lumpy in women who are pregnant, nursing, or have just given birth. This occurs because of normal hormone changes that take place during pregnancy. These changes can make small lumps difficult to detect. The breasts may also become denser. It is more difficult to detect breast cancer in women with dense breasts using mammography. Because these breast changes can delay diagnosis, breast cancer is often found at a later stage in these women.

The breast is made up of lobes and ducts. Each breast has 15 to 20 sections called lobes. Each lobe has many smaller sections called lobules. Lobules end in dozens of tiny bulbs that can make milk. The lobes, lobules, and bulbs are linked by thin tubes called ducts. EnlargeAnatomy of the female breast. The nipple and areola are shown on the outside of the breast. The lymph nodes, lobes, lobules, ducts, and other parts of the inside of the breast are also shown. Each breast also has blood vessels and lymph vessels. The lymph vessels carry an almost colorless, watery fluid called lymph. Lymph vessels carry lymph between lymph nodes. Lymph nodes are small, bean-shaped structures found throughout the body. They filter lymph and store white blood cells that help fight infection and disease. Groups of lymph nodes are found near the breast in the axilla (under the arm), above the collarbone, and in the chest. Breast cancer occurs about once in every 3,000 pregnancies. It occurs most often in women aged 32 to 38 years. Because many women are choosing to delay having children, it is likely that the number of new cases of breast cancer during pregnancy will increase. These and other signs may be caused by breast cancer or by other conditions. Check with your doctor if you have any of the following: The breasts usually get larger, tender, or lumpy in women who are pregnant, nursing, or have just given birth. This occurs because of normal hormone changes that take place during pregnancy. These changes can make small lumps difficult to detect. The breasts may also become denser. It is more difficult to detect breast cancer in women with dense breasts using mammography. Because these breast changes can delay diagnosis, breast cancer is often found at a later stage in these women. To detect breast cancer, pregnant and nursing women should examine their breasts themselves. Women should also receive clinical breast exams during their regular prenatal and postnatal check-ups. Talk to your doctor if you notice any changes in your breasts that you do not expect or that worry you. The following tests and procedures may be used: There are three types of breast biopsies: Decisions about the best treatment are based on the results of these tests and the trimester of the pregnancy. The tests give information about: Tests may include the following: The prognosis and treatment options depend on the following: The process used to find out if the cancer has spread within the breast or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some procedures may expose the fetus to harmful radiation or dyes. These procedures are done only if absolutely necessary. Certain actions, such as using a lead-lined shield to cover the abdomen, are used to help protect the fetus from radiation as much as possible. The following tests and procedures may be used to stage breast cancer during pregnancy: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if breast cancer spreads to the bone, the cancer cells in the bone are actually breast cancer cells. The disease is metastatic breast cancer, not bone cancer. To plan the best treatment and understand your prognosis, it is important to know the breast cancer stage. There are 3 types of breast cancer stage groups: For breast cancer, the TNM system describes the tumor as follows: When the lymph nodes are removed by surgery and studied under a microscope by a pathologist, pathologic staging is used to describe the lymph nodes. The pathologic staging of lymph nodes is described below. or cancer has spread to 4 to 9 axillary lymph nodes and cancer in at least one of the lymph nodes is larger than 2 millimeters. Cancer has also spread to lymph nodes near the breastbone on the same side of the body as the primary tumor, and the cancer is larger than 0.2 millimeters and is found by sentinel lymph node biopsy. When the lymph nodes are checked using mammography or ultrasound, it is called clinical staging. The clinical staging of lymph nodes is not described here. The grading system describes a tumor based on how abnormal the cancer cells and tissue look under a microscope and how quickly the cancer cells are likely to grow and spread. Low-grade cancer cells look more like normal cells and tend to grow and spread more slowly than high-grade cancer cells. To describe how abnormal the cancer cells and tissue are, the pathologist will assess the following three features: For each feature, the pathologist assigns a score of 1 to 3; a score of “1” means the cells and tumor tissue look the most like normal cells and tissue, and a score of “3” means the cells and tissue look the most abnormal. The scores for each feature are added together to get a total score between 3 and 9. Three grades are possible: Healthy breast cells, and some breast cancer cells, have receptors (biomarkers) that attach to the hormones estrogen and progesterone. These hormones are needed for healthy cells, and some breast cancer cells, to grow and divide. To check for these biomarkers, samples of tissue containing breast cancer cells are removed during a biopsy or surgery. The samples are tested in a laboratory to see whether the breast cancer cells have estrogen or progesterone receptors. Another type of receptor (biomarker) that is found on the surface of all breast cancer cells is called HER2. HER2 receptors are needed for the breast cancer cells to grow and divide. For breast cancer, biomarker testing includes the following: Sometimes the breast cancer cells will be described as triple negative or triple positive. It is important to know the estrogen receptor, progesterone receptor, and HER2 receptor status to choose the best treatment. There are drugs that can stop the receptors from attaching to the hormones estrogen and progesterone and stop the cancer from growing. Other drugs may be used to block the HER2 receptors on the surface of the breast cancer cells and stop the cancer from growing. Here are 3 examples that combine the TNM system, the grading system, and the biomarker status to find out the Pathological Prognostic breast cancer stage for a woman whose first treatment was surgery: If the tumor size is 30 millimeters (T2), has not spread to nearby lymph nodes (N0), has not spread to distant parts of the body (M0), and is: The cancer is stage IIA. If the tumor size is 53 millimeters (T3), has spread to 4 to 9 axillary lymph nodes (N2), has not spread to other parts of the body (M0), and is: The tumor is stage IIIA. If the tumor size is 65 millimeters (T3), has spread to 3 axillary lymph nodes (N1a), has spread to the lungs (M1), and is: The cancer is stage IV (metastatic breast cancer). After surgery, your doctor will receive a pathology report that describes the size and location of the primary tumor, the spread of cancer to nearby lymph nodes, tumor grade, and whether certain biomarkers are present. The pathology report and other test results are used to determine your breast cancer stage. You are likely to have many questions. Ask your doctor to explain how staging is used to decide the best options to treat your cancer and whether there are clinical trials that might be right for you. Most pregnant women with breast cancer have surgery to remove the breast. Some of the lymph nodes under the arm may be removed so they can be checked under a microscope by a pathologist for signs of cancer. Types of surgery to remove the cancer include: After the doctor removes all of the cancer that can be seen at the time of surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. For pregnant women with early-stage breast cancer, radiation therapy and hormone therapy are given after the baby is born. Treatment given after surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be given to pregnant women with early stage (stage I or II) breast cancer after the baby is born. Women with late stage (stage III or IV) breast cancer may be given external radiation therapy after the first 3 months of pregnancy or, if possible, radiation therapy is delayed until after the baby is born. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy is usually not given during the first 3 months of pregnancy. Chemotherapy given after this time does not usually harm the fetus but may cause early labor or low birth weight. See Drugs Approved for Breast Cancer for more information. Because ending the pregnancy is not likely to improve the mother’s chance of survival, it is not usually a treatment option. For information about side effects caused by treatment for cancer, see our Side Effects page. For information about the treatments listed below, see the Treatment Option Overview section. Pregnant women with early-stage breast cancer (stage I and stage II) are usually treated in the same way as patients who are not pregnant, with some changes to protect the fetus. Treatment may include the following: Hormone therapy and trastuzumab should not be given during pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for patients with late-stage breast cancer (stage III or stage IV) during pregnancy. Treatment may include the following: Radiation therapy and chemotherapy should not be given during the first 3 months of pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. If surgery is planned, breast-feeding should be stopped to reduce blood flow in the breasts and make them smaller. Many chemotherapy drugs, especially cyclophosphamide and methotrexate, may occur in high levels in breast milk and may harm the nursing baby. Women receiving chemotherapy should not breast-feed. Stopping lactation does not improve the mother's prognosis. Breast cancer cells do not seem to pass from the mother to the fetus. For women who have had breast cancer, pregnancy does not seem to affect their survival. However, some doctors recommend that a woman wait 2 years after treatment for breast cancer before trying to have a baby, so that any early return of the cancer would be detected. This may affect a woman’s decision to become pregnant. For more information from the National Cancer Institute about breast cancer during pregnancy, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of breast cancer during pregnancy. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Breast Cancer Treatment During Pregnancy. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/breast/patient/pregnancy-breast-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389161] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Breast Cancer Treatment During Pregnancy (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Breast Cancer ?

Breast exams should be part of prenatal and postnatal care. To detect breast cancer, pregnant and nursing women should examine their breasts themselves. Women should also receive clinical breast exams during their regular prenatal and postnatal check-ups. Talk to your doctor if you notice any changes in your breasts that you do not expect or that worry you. Tests that examine the breasts are used to detect (find) and diagnose breast cancer. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Clinical breast exam (CBE): An exam of the breast by a doctor or other health professional. The doctor will carefully feel the breasts and under the arms for lumps or anything else that seems unusual. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of both breasts. This procedure is also called nuclear magnetic resonance imaging (NMRI). - Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to look at later. - Mammogram : An x-ray of the breast. A mammogram can be done with little risk to the unborn baby. Mammograms in pregnant women may appear negative even though cancer is present. - Blood chemistry studies : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease. - Biopsy : The removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for signs of cancer. If a lump in the breast is found, a biopsy may be done. There are four types of breast biopsies: - Excisional biopsy : The removal of an entire lump of tissue. - Incisional biopsy : The removal of part of a lump or a sample of tissue. - Core biopsy : The removal of tissue using a wide needle. - Fine-needle aspiration (FNA) biopsy : The removal of tissue or fluid, using a thin needle. If cancer is found, tests are done to study the cancer cells. Decisions about the best treatment are based on the results of these tests and the age of the unborn baby. The tests give information about: - How quickly the cancer may grow. - How likely it is that the cancer will spread to other parts of the body. - How well certain treatments might work. - How likely the cancer is to recur (come back). Tests may include the following: - Estrogen and progesterone receptor test : A test to measure the amount of estrogen and progesterone (hormones) receptors in cancer tissue. If there are more estrogen and progesterone receptors than normal, the cancer is called estrogen and/or progesterone receptor positive. This type of breast cancer may grow more quickly. The test results show whether treatment to block estrogen and progesterone given after the baby is born may stop the cancer from growing. - Human epidermal growth factor type 2 receptor (HER2/neu) test : A laboratory test to measure how many HER2/neu genes there are and how much HER2/neu protein is made in a sample of tissue. If there are more HER2/neu genes or higher levels of HER2/neu protein than normal, the cancer is called HER2/neu positive. This type of breast cancer may grow more quickly and is more likely to spread to other parts of the body. The cancer may be treated with drugs that target the HER2/neu protein, such as trastuzumab and pertuzumab, after the baby is born. - Multigene tests: Tests in which samples of tissue are studied to look at the activity of many genes at the same time. These tests may help predict whether cancer will spread to other parts of the body or recur (come back). - Oncotype DX : This test helps predict whether stage I or stage II breast cancer that is estrogen receptor positive and node-negative will spread to other parts of the body. If the risk of the cancer spreading is high, chemotherapy may be given to lower the risk. - MammaPrint : This test helps predict whether stage I or stage II breast cancer that is node-negative will spread to other parts of the body. If the risk of the cancer spreading is high, chemotherapy may be given to lower the risk.

The breast is made up of lobes and ducts. Each breast has 15 to 20 sections called lobes. Each lobe has many smaller sections called lobules. Lobules end in dozens of tiny bulbs that can make milk. The lobes, lobules, and bulbs are linked by thin tubes called ducts. EnlargeAnatomy of the female breast. The nipple and areola are shown on the outside of the breast. The lymph nodes, lobes, lobules, ducts, and other parts of the inside of the breast are also shown. Each breast also has blood vessels and lymph vessels. The lymph vessels carry an almost colorless, watery fluid called lymph. Lymph vessels carry lymph between lymph nodes. Lymph nodes are small, bean-shaped structures found throughout the body. They filter lymph and store white blood cells that help fight infection and disease. Groups of lymph nodes are found near the breast in the axilla (under the arm), above the collarbone, and in the chest. Breast cancer occurs about once in every 3,000 pregnancies. It occurs most often in women aged 32 to 38 years. Because many women are choosing to delay having children, it is likely that the number of new cases of breast cancer during pregnancy will increase. These and other signs may be caused by breast cancer or by other conditions. Check with your doctor if you have any of the following: The breasts usually get larger, tender, or lumpy in women who are pregnant, nursing, or have just given birth. This occurs because of normal hormone changes that take place during pregnancy. These changes can make small lumps difficult to detect. The breasts may also become denser. It is more difficult to detect breast cancer in women with dense breasts using mammography. Because these breast changes can delay diagnosis, breast cancer is often found at a later stage in these women. To detect breast cancer, pregnant and nursing women should examine their breasts themselves. Women should also receive clinical breast exams during their regular prenatal and postnatal check-ups. Talk to your doctor if you notice any changes in your breasts that you do not expect or that worry you. The following tests and procedures may be used: There are three types of breast biopsies: Decisions about the best treatment are based on the results of these tests and the trimester of the pregnancy. The tests give information about: Tests may include the following: The prognosis and treatment options depend on the following: The process used to find out if the cancer has spread within the breast or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some procedures may expose the fetus to harmful radiation or dyes. These procedures are done only if absolutely necessary. Certain actions, such as using a lead-lined shield to cover the abdomen, are used to help protect the fetus from radiation as much as possible. The following tests and procedures may be used to stage breast cancer during pregnancy: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if breast cancer spreads to the bone, the cancer cells in the bone are actually breast cancer cells. The disease is metastatic breast cancer, not bone cancer. To plan the best treatment and understand your prognosis, it is important to know the breast cancer stage. There are 3 types of breast cancer stage groups: For breast cancer, the TNM system describes the tumor as follows: When the lymph nodes are removed by surgery and studied under a microscope by a pathologist, pathologic staging is used to describe the lymph nodes. The pathologic staging of lymph nodes is described below. or cancer has spread to 4 to 9 axillary lymph nodes and cancer in at least one of the lymph nodes is larger than 2 millimeters. Cancer has also spread to lymph nodes near the breastbone on the same side of the body as the primary tumor, and the cancer is larger than 0.2 millimeters and is found by sentinel lymph node biopsy. When the lymph nodes are checked using mammography or ultrasound, it is called clinical staging. The clinical staging of lymph nodes is not described here. The grading system describes a tumor based on how abnormal the cancer cells and tissue look under a microscope and how quickly the cancer cells are likely to grow and spread. Low-grade cancer cells look more like normal cells and tend to grow and spread more slowly than high-grade cancer cells. To describe how abnormal the cancer cells and tissue are, the pathologist will assess the following three features: For each feature, the pathologist assigns a score of 1 to 3; a score of “1” means the cells and tumor tissue look the most like normal cells and tissue, and a score of “3” means the cells and tissue look the most abnormal. The scores for each feature are added together to get a total score between 3 and 9. Three grades are possible: Healthy breast cells, and some breast cancer cells, have receptors (biomarkers) that attach to the hormones estrogen and progesterone. These hormones are needed for healthy cells, and some breast cancer cells, to grow and divide. To check for these biomarkers, samples of tissue containing breast cancer cells are removed during a biopsy or surgery. The samples are tested in a laboratory to see whether the breast cancer cells have estrogen or progesterone receptors. Another type of receptor (biomarker) that is found on the surface of all breast cancer cells is called HER2. HER2 receptors are needed for the breast cancer cells to grow and divide. For breast cancer, biomarker testing includes the following: Sometimes the breast cancer cells will be described as triple negative or triple positive. It is important to know the estrogen receptor, progesterone receptor, and HER2 receptor status to choose the best treatment. There are drugs that can stop the receptors from attaching to the hormones estrogen and progesterone and stop the cancer from growing. Other drugs may be used to block the HER2 receptors on the surface of the breast cancer cells and stop the cancer from growing. Here are 3 examples that combine the TNM system, the grading system, and the biomarker status to find out the Pathological Prognostic breast cancer stage for a woman whose first treatment was surgery: If the tumor size is 30 millimeters (T2), has not spread to nearby lymph nodes (N0), has not spread to distant parts of the body (M0), and is: The cancer is stage IIA. If the tumor size is 53 millimeters (T3), has spread to 4 to 9 axillary lymph nodes (N2), has not spread to other parts of the body (M0), and is: The tumor is stage IIIA. If the tumor size is 65 millimeters (T3), has spread to 3 axillary lymph nodes (N1a), has spread to the lungs (M1), and is: The cancer is stage IV (metastatic breast cancer). After surgery, your doctor will receive a pathology report that describes the size and location of the primary tumor, the spread of cancer to nearby lymph nodes, tumor grade, and whether certain biomarkers are present. The pathology report and other test results are used to determine your breast cancer stage. You are likely to have many questions. Ask your doctor to explain how staging is used to decide the best options to treat your cancer and whether there are clinical trials that might be right for you. Most pregnant women with breast cancer have surgery to remove the breast. Some of the lymph nodes under the arm may be removed so they can be checked under a microscope by a pathologist for signs of cancer. Types of surgery to remove the cancer include: After the doctor removes all of the cancer that can be seen at the time of surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. For pregnant women with early-stage breast cancer, radiation therapy and hormone therapy are given after the baby is born. Treatment given after surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be given to pregnant women with early stage (stage I or II) breast cancer after the baby is born. Women with late stage (stage III or IV) breast cancer may be given external radiation therapy after the first 3 months of pregnancy or, if possible, radiation therapy is delayed until after the baby is born. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy is usually not given during the first 3 months of pregnancy. Chemotherapy given after this time does not usually harm the fetus but may cause early labor or low birth weight. See Drugs Approved for Breast Cancer for more information. Because ending the pregnancy is not likely to improve the mother’s chance of survival, it is not usually a treatment option. For information about side effects caused by treatment for cancer, see our Side Effects page. For information about the treatments listed below, see the Treatment Option Overview section. Pregnant women with early-stage breast cancer (stage I and stage II) are usually treated in the same way as patients who are not pregnant, with some changes to protect the fetus. Treatment may include the following: Hormone therapy and trastuzumab should not be given during pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for patients with late-stage breast cancer (stage III or stage IV) during pregnancy. Treatment may include the following: Radiation therapy and chemotherapy should not be given during the first 3 months of pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. If surgery is planned, breast-feeding should be stopped to reduce blood flow in the breasts and make them smaller. Many chemotherapy drugs, especially cyclophosphamide and methotrexate, may occur in high levels in breast milk and may harm the nursing baby. Women receiving chemotherapy should not breast-feed. Stopping lactation does not improve the mother's prognosis. Breast cancer cells do not seem to pass from the mother to the fetus. For women who have had breast cancer, pregnancy does not seem to affect their survival. However, some doctors recommend that a woman wait 2 years after treatment for breast cancer before trying to have a baby, so that any early return of the cancer would be detected. This may affect a woman’s decision to become pregnant. For more information from the National Cancer Institute about breast cancer during pregnancy, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of breast cancer during pregnancy. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Breast Cancer Treatment During Pregnancy. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/breast/patient/pregnancy-breast-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389161] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Breast Cancer Treatment During Pregnancy (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? 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What is the outlook for Breast Cancer ?

Certain factors affect prognosis (chance of recovery) and treatment options.The prognosis (chance of recovery) and treatment options depend on the following: - The stage of the cancer (the size of the tumor and whether it is in the breast only or has spread to other parts of the body). - The type of breast cancer. - The age of the unborn baby. - Whether there are signs or symptoms. - The patients general health.

The breast is made up of lobes and ducts. Each breast has 15 to 20 sections called lobes. Each lobe has many smaller sections called lobules. Lobules end in dozens of tiny bulbs that can make milk. The lobes, lobules, and bulbs are linked by thin tubes called ducts. EnlargeAnatomy of the female breast. The nipple and areola are shown on the outside of the breast. The lymph nodes, lobes, lobules, ducts, and other parts of the inside of the breast are also shown. Each breast also has blood vessels and lymph vessels. The lymph vessels carry an almost colorless, watery fluid called lymph. Lymph vessels carry lymph between lymph nodes. Lymph nodes are small, bean-shaped structures found throughout the body. They filter lymph and store white blood cells that help fight infection and disease. Groups of lymph nodes are found near the breast in the axilla (under the arm), above the collarbone, and in the chest. Breast cancer occurs about once in every 3,000 pregnancies. It occurs most often in women aged 32 to 38 years. Because many women are choosing to delay having children, it is likely that the number of new cases of breast cancer during pregnancy will increase. These and other signs may be caused by breast cancer or by other conditions. Check with your doctor if you have any of the following: The breasts usually get larger, tender, or lumpy in women who are pregnant, nursing, or have just given birth. This occurs because of normal hormone changes that take place during pregnancy. These changes can make small lumps difficult to detect. The breasts may also become denser. It is more difficult to detect breast cancer in women with dense breasts using mammography. Because these breast changes can delay diagnosis, breast cancer is often found at a later stage in these women. To detect breast cancer, pregnant and nursing women should examine their breasts themselves. Women should also receive clinical breast exams during their regular prenatal and postnatal check-ups. Talk to your doctor if you notice any changes in your breasts that you do not expect or that worry you. The following tests and procedures may be used: There are three types of breast biopsies: Decisions about the best treatment are based on the results of these tests and the trimester of the pregnancy. The tests give information about: Tests may include the following: The prognosis and treatment options depend on the following: The process used to find out if the cancer has spread within the breast or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some procedures may expose the fetus to harmful radiation or dyes. These procedures are done only if absolutely necessary. Certain actions, such as using a lead-lined shield to cover the abdomen, are used to help protect the fetus from radiation as much as possible. The following tests and procedures may be used to stage breast cancer during pregnancy: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if breast cancer spreads to the bone, the cancer cells in the bone are actually breast cancer cells. The disease is metastatic breast cancer, not bone cancer. To plan the best treatment and understand your prognosis, it is important to know the breast cancer stage. There are 3 types of breast cancer stage groups: For breast cancer, the TNM system describes the tumor as follows: When the lymph nodes are removed by surgery and studied under a microscope by a pathologist, pathologic staging is used to describe the lymph nodes. The pathologic staging of lymph nodes is described below. or cancer has spread to 4 to 9 axillary lymph nodes and cancer in at least one of the lymph nodes is larger than 2 millimeters. Cancer has also spread to lymph nodes near the breastbone on the same side of the body as the primary tumor, and the cancer is larger than 0.2 millimeters and is found by sentinel lymph node biopsy. When the lymph nodes are checked using mammography or ultrasound, it is called clinical staging. The clinical staging of lymph nodes is not described here. The grading system describes a tumor based on how abnormal the cancer cells and tissue look under a microscope and how quickly the cancer cells are likely to grow and spread. Low-grade cancer cells look more like normal cells and tend to grow and spread more slowly than high-grade cancer cells. To describe how abnormal the cancer cells and tissue are, the pathologist will assess the following three features: For each feature, the pathologist assigns a score of 1 to 3; a score of “1” means the cells and tumor tissue look the most like normal cells and tissue, and a score of “3” means the cells and tissue look the most abnormal. The scores for each feature are added together to get a total score between 3 and 9. Three grades are possible: Healthy breast cells, and some breast cancer cells, have receptors (biomarkers) that attach to the hormones estrogen and progesterone. These hormones are needed for healthy cells, and some breast cancer cells, to grow and divide. To check for these biomarkers, samples of tissue containing breast cancer cells are removed during a biopsy or surgery. The samples are tested in a laboratory to see whether the breast cancer cells have estrogen or progesterone receptors. Another type of receptor (biomarker) that is found on the surface of all breast cancer cells is called HER2. HER2 receptors are needed for the breast cancer cells to grow and divide. For breast cancer, biomarker testing includes the following: Sometimes the breast cancer cells will be described as triple negative or triple positive. It is important to know the estrogen receptor, progesterone receptor, and HER2 receptor status to choose the best treatment. There are drugs that can stop the receptors from attaching to the hormones estrogen and progesterone and stop the cancer from growing. Other drugs may be used to block the HER2 receptors on the surface of the breast cancer cells and stop the cancer from growing. Here are 3 examples that combine the TNM system, the grading system, and the biomarker status to find out the Pathological Prognostic breast cancer stage for a woman whose first treatment was surgery: If the tumor size is 30 millimeters (T2), has not spread to nearby lymph nodes (N0), has not spread to distant parts of the body (M0), and is: The cancer is stage IIA. If the tumor size is 53 millimeters (T3), has spread to 4 to 9 axillary lymph nodes (N2), has not spread to other parts of the body (M0), and is: The tumor is stage IIIA. If the tumor size is 65 millimeters (T3), has spread to 3 axillary lymph nodes (N1a), has spread to the lungs (M1), and is: The cancer is stage IV (metastatic breast cancer). After surgery, your doctor will receive a pathology report that describes the size and location of the primary tumor, the spread of cancer to nearby lymph nodes, tumor grade, and whether certain biomarkers are present. The pathology report and other test results are used to determine your breast cancer stage. You are likely to have many questions. Ask your doctor to explain how staging is used to decide the best options to treat your cancer and whether there are clinical trials that might be right for you. Most pregnant women with breast cancer have surgery to remove the breast. Some of the lymph nodes under the arm may be removed so they can be checked under a microscope by a pathologist for signs of cancer. Types of surgery to remove the cancer include: After the doctor removes all of the cancer that can be seen at the time of surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. For pregnant women with early-stage breast cancer, radiation therapy and hormone therapy are given after the baby is born. Treatment given after surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be given to pregnant women with early stage (stage I or II) breast cancer after the baby is born. Women with late stage (stage III or IV) breast cancer may be given external radiation therapy after the first 3 months of pregnancy or, if possible, radiation therapy is delayed until after the baby is born. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy is usually not given during the first 3 months of pregnancy. Chemotherapy given after this time does not usually harm the fetus but may cause early labor or low birth weight. See Drugs Approved for Breast Cancer for more information. Because ending the pregnancy is not likely to improve the mother’s chance of survival, it is not usually a treatment option. For information about side effects caused by treatment for cancer, see our Side Effects page. For information about the treatments listed below, see the Treatment Option Overview section. Pregnant women with early-stage breast cancer (stage I and stage II) are usually treated in the same way as patients who are not pregnant, with some changes to protect the fetus. Treatment may include the following: Hormone therapy and trastuzumab should not be given during pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for patients with late-stage breast cancer (stage III or stage IV) during pregnancy. Treatment may include the following: Radiation therapy and chemotherapy should not be given during the first 3 months of pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. If surgery is planned, breast-feeding should be stopped to reduce blood flow in the breasts and make them smaller. Many chemotherapy drugs, especially cyclophosphamide and methotrexate, may occur in high levels in breast milk and may harm the nursing baby. Women receiving chemotherapy should not breast-feed. Stopping lactation does not improve the mother's prognosis. Breast cancer cells do not seem to pass from the mother to the fetus. For women who have had breast cancer, pregnancy does not seem to affect their survival. However, some doctors recommend that a woman wait 2 years after treatment for breast cancer before trying to have a baby, so that any early return of the cancer would be detected. This may affect a woman’s decision to become pregnant. For more information from the National Cancer Institute about breast cancer during pregnancy, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of breast cancer during pregnancy. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Breast Cancer Treatment During Pregnancy. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/breast/patient/pregnancy-breast-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389161] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Breast Cancer Treatment During Pregnancy (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Breast Cancer ?

Key Points - After breast cancer has been diagnosed, tests are done to find out if cancer cells have spread within the breast or to other parts of the body. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - The following stages are used for breast cancer: - Stage 0 (carcinoma in situ) - Stage I - Stage II - Stage IIIA - Stage IIIB - Stage IIIC - Stage IV After breast cancer has been diagnosed, tests are done to find out if cancer cells have spread within the breast or to other parts of the body. The process used to find out if the cancer has spread within the breast or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some procedures may expose the unborn baby to harmful radiation or dyes. These procedures are done only if absolutely necessary. Certain actions can be taken to expose the unborn baby to as little radiation as possible, such as the use of a lead-lined shield to cover the abdomen. The following tests and procedures may be used to stage breast cancer during pregnancy: - Sentinel lymph node biopsy : The removal of the sentinel lymph node during surgery. The sentinel lymph node is the first lymph node to receive lymphatic drainage from a tumor. It is the first lymph node the cancer is likely to spread to from the tumor. A radioactive substance and/or blue dye is injected near the tumor. The substance or dye flows through the lymph ducts to the lymph nodes. The first lymph node to receive the substance or dye is removed. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are not found, it may not be necessary to remove more lymph nodes. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - Bone scan : A procedure to check if there are rapidly dividing cells, such as cancer cells, in the bone. A very small amount of radioactive material is injected into a vein and travels through the bloodstream. The radioactive material collects in bones with cancer and is detected by a scanner. - PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body, such as the brain. This procedure is also called nuclear magnetic resonance imaging (NMRI). - Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs, such as the liver, and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to be looked at later. - Chest x-ray : An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if breast cancer spreads to the bone, the cancer cells in the bone are actually breast cancer cells. The disease is metastatic breast cancer, not bone cancer. The following stages are used for breast cancer: This section describes the stages of breast cancer. The breast cancer stage is based on the results of testing that is done on the tumor and lymph nodes removed during surgery and other tests. Stage 0 (carcinoma in situ) There are 3 types of breast carcinoma in situ: - Ductal carcinoma in situ (DCIS) is a noninvasive condition in which abnormal cells are found in the lining of a breast duct. The abnormal cells have not spread outside the duct to other tissues in the breast. In some cases, DCIS may become invasive cancer and spread to other tissues. At this time, there is no way to know which lesions could become invasive. - Lobular carcinoma in situ (LCIS) is a condition in which abnormal cells are found in the lobules of the breast. This condition seldom becomes invasive cancer. However, having LCIS in one breast increases the risk of developing breast cancer in either breast. - Paget disease of the nipple is a condition in which abnormal cells are found in the nipple only. Stage I In stage I, cancer has formed. Stage I is divided into stages IA and IB. - In stage IA, the tumor is 2 centimeters or smaller. Cancer has not spread outside the breast. - In stage IB, small clusters of breast cancer cells (larger than 0.2 millimeter but not larger than 2 millimeters) are found in the lymph nodes and either: - no tumor is found in the breast; or - the tumor is 2 centimeters or smaller. Stage II Stage II is divided into stages IIA and IIB. - In stage IIA: - no tumor is found in the breast or the tumor is 2 centimeters or smaller. Cancer (larger than 2 millimeters) is found in 1 to 3 axillary lymph nodes or in the lymph nodes near the breastbone (found during a sentinel lymph node biopsy); or - the tumor is larger than 2 centimeters but not larger than 5 centimeters. Cancer has not spread to the lymph nodes. - In stage IIB, the tumor is: - larger than 2 centimeters but not larger than 5 centimeters. Small clusters of breast cancer cells (larger than 0.2 millimeter but not larger than 2 millimeters) are found in the lymph nodes; or - larger than 2 centimeters but not larger than 5 centimeters. Cancer has spread to 1 to 3 axillary lymph nodes or to the lymph nodes near the breastbone (found during a sentinel lymph node biopsy); or - larger than 5 centimeters. Cancer has not spread to the lymph nodes. Stage IIIA In stage IIIA: - no tumor is found in the breast or the tumor may be any size. Cancer is found in 4 to 9 axillary lymph nodes or in the lymph nodes near the breastbone (found during imaging tests or a physical exam); or - the tumor is larger than 5 centimeters. Small clusters of breast cancer cells (larger than 0.2 millimeter but not larger than 2 millimeters) are found in the lymph nodes; or - the tumor is larger than 5 centimeters. Cancer has spread to 1 to 3 axillary lymph nodes or to the lymph nodes near the breastbone (found during a sentinel lymph node biopsy). Stage IIIB In stage IIIB, the tumor may be any size and cancer has spread to the chest wall and/or to the skin of the breast and caused swelling or an ulcer. Also, cancer may have spread to: - up to 9 axillary lymph nodes; or - the lymph nodes near the breastbone. Cancer that has spread to the skin of the breast may also be inflammatory breast cancer. See the section on Inflammatory Breast Cancer for more information. Stage IIIC In stage IIIC, no tumor is found in the breast or the tumor may be any size. Cancer may have spread to the skin of the breast and caused swelling or an ulcer and/or has spread to the chest wall. Also, cancer has spread to: - 10 or more axillary lymph nodes; or - lymph nodes above or below the collarbone; or - axillary lymph nodes and lymph nodes near the breastbone. Cancer that has spread to the skin of the breast may also be inflammatory breast cancer. See the section on Inflammatory Breast Cancer for more information. For treatment, stage IIIC breast cancer is divided into operable and inoperable stage IIIC. Stage IV In stage IV, cancer has spread to other organs of the body, most often the bones, lungs, liver, or brain.

The breast is made up of lobes and ducts. Each breast has 15 to 20 sections called lobes. Each lobe has many smaller sections called lobules. Lobules end in dozens of tiny bulbs that can make milk. The lobes, lobules, and bulbs are linked by thin tubes called ducts. EnlargeAnatomy of the female breast. The nipple and areola are shown on the outside of the breast. The lymph nodes, lobes, lobules, ducts, and other parts of the inside of the breast are also shown. Each breast also has blood vessels and lymph vessels. The lymph vessels carry an almost colorless, watery fluid called lymph. Lymph vessels carry lymph between lymph nodes. Lymph nodes are small, bean-shaped structures found throughout the body. They filter lymph and store white blood cells that help fight infection and disease. Groups of lymph nodes are found near the breast in the axilla (under the arm), above the collarbone, and in the chest. Breast cancer occurs about once in every 3,000 pregnancies. It occurs most often in women aged 32 to 38 years. Because many women are choosing to delay having children, it is likely that the number of new cases of breast cancer during pregnancy will increase. These and other signs may be caused by breast cancer or by other conditions. Check with your doctor if you have any of the following: The breasts usually get larger, tender, or lumpy in women who are pregnant, nursing, or have just given birth. This occurs because of normal hormone changes that take place during pregnancy. These changes can make small lumps difficult to detect. The breasts may also become denser. It is more difficult to detect breast cancer in women with dense breasts using mammography. Because these breast changes can delay diagnosis, breast cancer is often found at a later stage in these women. To detect breast cancer, pregnant and nursing women should examine their breasts themselves. Women should also receive clinical breast exams during their regular prenatal and postnatal check-ups. Talk to your doctor if you notice any changes in your breasts that you do not expect or that worry you. The following tests and procedures may be used: There are three types of breast biopsies: Decisions about the best treatment are based on the results of these tests and the trimester of the pregnancy. The tests give information about: Tests may include the following: The prognosis and treatment options depend on the following: The process used to find out if the cancer has spread within the breast or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some procedures may expose the fetus to harmful radiation or dyes. These procedures are done only if absolutely necessary. Certain actions, such as using a lead-lined shield to cover the abdomen, are used to help protect the fetus from radiation as much as possible. The following tests and procedures may be used to stage breast cancer during pregnancy: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if breast cancer spreads to the bone, the cancer cells in the bone are actually breast cancer cells. The disease is metastatic breast cancer, not bone cancer. To plan the best treatment and understand your prognosis, it is important to know the breast cancer stage. There are 3 types of breast cancer stage groups: For breast cancer, the TNM system describes the tumor as follows: When the lymph nodes are removed by surgery and studied under a microscope by a pathologist, pathologic staging is used to describe the lymph nodes. The pathologic staging of lymph nodes is described below. or cancer has spread to 4 to 9 axillary lymph nodes and cancer in at least one of the lymph nodes is larger than 2 millimeters. Cancer has also spread to lymph nodes near the breastbone on the same side of the body as the primary tumor, and the cancer is larger than 0.2 millimeters and is found by sentinel lymph node biopsy. When the lymph nodes are checked using mammography or ultrasound, it is called clinical staging. The clinical staging of lymph nodes is not described here. The grading system describes a tumor based on how abnormal the cancer cells and tissue look under a microscope and how quickly the cancer cells are likely to grow and spread. Low-grade cancer cells look more like normal cells and tend to grow and spread more slowly than high-grade cancer cells. To describe how abnormal the cancer cells and tissue are, the pathologist will assess the following three features: For each feature, the pathologist assigns a score of 1 to 3; a score of “1” means the cells and tumor tissue look the most like normal cells and tissue, and a score of “3” means the cells and tissue look the most abnormal. The scores for each feature are added together to get a total score between 3 and 9. Three grades are possible: Healthy breast cells, and some breast cancer cells, have receptors (biomarkers) that attach to the hormones estrogen and progesterone. These hormones are needed for healthy cells, and some breast cancer cells, to grow and divide. To check for these biomarkers, samples of tissue containing breast cancer cells are removed during a biopsy or surgery. The samples are tested in a laboratory to see whether the breast cancer cells have estrogen or progesterone receptors. Another type of receptor (biomarker) that is found on the surface of all breast cancer cells is called HER2. HER2 receptors are needed for the breast cancer cells to grow and divide. For breast cancer, biomarker testing includes the following: Sometimes the breast cancer cells will be described as triple negative or triple positive. It is important to know the estrogen receptor, progesterone receptor, and HER2 receptor status to choose the best treatment. There are drugs that can stop the receptors from attaching to the hormones estrogen and progesterone and stop the cancer from growing. Other drugs may be used to block the HER2 receptors on the surface of the breast cancer cells and stop the cancer from growing. Here are 3 examples that combine the TNM system, the grading system, and the biomarker status to find out the Pathological Prognostic breast cancer stage for a woman whose first treatment was surgery: If the tumor size is 30 millimeters (T2), has not spread to nearby lymph nodes (N0), has not spread to distant parts of the body (M0), and is: The cancer is stage IIA. If the tumor size is 53 millimeters (T3), has spread to 4 to 9 axillary lymph nodes (N2), has not spread to other parts of the body (M0), and is: The tumor is stage IIIA. If the tumor size is 65 millimeters (T3), has spread to 3 axillary lymph nodes (N1a), has spread to the lungs (M1), and is: The cancer is stage IV (metastatic breast cancer). After surgery, your doctor will receive a pathology report that describes the size and location of the primary tumor, the spread of cancer to nearby lymph nodes, tumor grade, and whether certain biomarkers are present. The pathology report and other test results are used to determine your breast cancer stage. You are likely to have many questions. Ask your doctor to explain how staging is used to decide the best options to treat your cancer and whether there are clinical trials that might be right for you. Most pregnant women with breast cancer have surgery to remove the breast. Some of the lymph nodes under the arm may be removed so they can be checked under a microscope by a pathologist for signs of cancer. Types of surgery to remove the cancer include: After the doctor removes all of the cancer that can be seen at the time of surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. For pregnant women with early-stage breast cancer, radiation therapy and hormone therapy are given after the baby is born. Treatment given after surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be given to pregnant women with early stage (stage I or II) breast cancer after the baby is born. Women with late stage (stage III or IV) breast cancer may be given external radiation therapy after the first 3 months of pregnancy or, if possible, radiation therapy is delayed until after the baby is born. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy is usually not given during the first 3 months of pregnancy. Chemotherapy given after this time does not usually harm the fetus but may cause early labor or low birth weight. See Drugs Approved for Breast Cancer for more information. Because ending the pregnancy is not likely to improve the mother’s chance of survival, it is not usually a treatment option. For information about side effects caused by treatment for cancer, see our Side Effects page. For information about the treatments listed below, see the Treatment Option Overview section. Pregnant women with early-stage breast cancer (stage I and stage II) are usually treated in the same way as patients who are not pregnant, with some changes to protect the fetus. Treatment may include the following: Hormone therapy and trastuzumab should not be given during pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for patients with late-stage breast cancer (stage III or stage IV) during pregnancy. Treatment may include the following: Radiation therapy and chemotherapy should not be given during the first 3 months of pregnancy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. If surgery is planned, breast-feeding should be stopped to reduce blood flow in the breasts and make them smaller. Many chemotherapy drugs, especially cyclophosphamide and methotrexate, may occur in high levels in breast milk and may harm the nursing baby. Women receiving chemotherapy should not breast-feed. Stopping lactation does not improve the mother's prognosis. Breast cancer cells do not seem to pass from the mother to the fetus. For women who have had breast cancer, pregnancy does not seem to affect their survival. However, some doctors recommend that a woman wait 2 years after treatment for breast cancer before trying to have a baby, so that any early return of the cancer would be detected. This may affect a woman’s decision to become pregnant. For more information from the National Cancer Institute about breast cancer during pregnancy, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of breast cancer during pregnancy. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. 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What are the treatments for Breast Cancer ?

Key Points - Treatment options for pregnant women depend on the stage of the disease and the age of the unborn baby. - Three types of standard treatment are used: - Surgery - Radiation therapy - Chemotherapy - Ending the pregnancy does not seem to improve the mothers chance of survival. - Treatment for breast cancer may cause side effects. Treatment options for pregnant women depend on the stage of the disease and the age of the unborn baby. Three types of standard treatment are used: Surgery Most pregnant women with breast cancer have surgery to remove the breast. Some of the lymph nodes under the arm may be removed and checked under a microscope for signs of cancer. Types of surgery to remove the cancer include: - Modified radical mastectomy: Surgery to remove the whole breast that has cancer, many of the lymph nodes under the arm, the lining over the chest muscles, and sometimes, part of the chest wall muscles. This type of surgery is most common in pregnant women. - Breast-conserving surgery: Surgery to remove the cancer and some normal tissue around it, but not the breast itself. Part of the chest wall lining may also be removed if the cancer is near it. This type of surgery may also be called lumpectomy, partial mastectomy, segmental mastectomy, quadrantectomy, or breast-sparing surgery. Even if the doctor removes all of the cancer that can be seen at the time of surgery, the patient may be given radiation therapy or chemotherapy after surgery to try to kill any cancer cells that may be left. For pregnant women with early-stage breast cancer, radiation therapy and hormone therapy are given after the baby is born. Treatment given after surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy is not given to pregnant women with early stage (stage I or II) breast cancer because it can harm the unborn baby. For women with late stage (stage III or IV) breast cancer, radiation therapy is not given during the first 3 months of pregnancy and is delayed until after the baby is born, if possible. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. Chemotherapy is usually not given during the first 3 months of pregnancy. Chemotherapy given after this time does not usually harm the unborn baby but may cause early labor and low birth weight. See Drugs Approved for Breast Cancer for more information. Ending the pregnancy does not seem to improve the mothers chance of survival. Because ending the pregnancy is not likely to improve the mothers chance of survival, it is not usually a treatment option. Treatment for breast cancer may cause side effects. For information about side effects caused by treatment for cancer, see our Side Effects page. Treatment Options by Stage Early Stage Breast Cancer (Stage I and Stage II) Treatment of early-stage breast cancer (stage I and stage II) may include the following: - Modified radical mastectomy. - Breast-conserving surgery followed by radiation therapy. In pregnant women, radiation therapy is delayed until after the baby is born. - Modified radical mastectomy or breast-conserving surgery during pregnancy followed by chemotherapy after the first 3 months of pregnancy. Late Stage Breast Cancer (Stage III and Stage IV) Treatment of late-stage breast cancer (stage III and stage IV) may include the following: - Radiation therapy. - Chemotherapy. Radiation therapy and chemotherapy should not be given during the first 3 months of pregnancy.

Screening is looking for cancer before a person has any symptoms. This can help find cancer at an early stage. When abnormal tissue or cancer is found early, it may be easier to treat. By the time symptoms appear, cancer may have begun to spread. Scientists are trying to better understand which people are more likely to get certain types of cancer. They also study the things we do and the things around us to see if they cause cancer. This information helps doctors recommend who should be screened for cancer, which screening tests should be used, and how often the tests should be done. It is important to remember that your doctor does not necessarily think you have cancer if he or she suggests a screening test. Screening tests are given when you have no cancer symptoms. If a screening test result is abnormal, you may need to have more tests done to find out if you have cancer. These are called diagnostic tests. Neuroblastoma often begins in the nerve tissue of the adrenal glands. There are two adrenal glands, one on top of each kidney, in the back of the upper abdomen. The adrenal glands make important hormones that help control heart rate, blood pressure, blood sugar, and the way the body reacts to stress. Neuroblastoma may also begin in the abdomen, chest, spinal cord, or in nerve tissue near the spine in the neck. Neuroblastoma most often begins during early childhood, usually in children younger than 5 years of age. See the PDQ summary on Neuroblastoma Treatment for more information about neuroblastoma. Neuroblastoma is the most common type of cancer in infants. The number of new cases of neuroblastoma is greatest among children under 1 year of age. As children get older, the number of new cases decreases. Neuroblastoma is slightly more common in males than females. Neuroblastoma sometimes forms before birth but is usually found later, when the tumor begins to grow and cause symptoms. In rare cases, neuroblastoma may be found before birth, by fetal ultrasound. Scientists study screening tests to find those with the fewest harms and most benefits. Cancer screening trials also are meant to show whether early detection (finding cancer before it causes symptoms) helps a person live longer or decreases a person's chance of dying from the disease. For some types of cancer, the chance of recovery is better if the disease is found and treated at an early stage. There is no standard or routine screening test used to find neuroblastoma. A urine test is sometimes used to check for neuroblastoma, usually when the child is 6 months old. This is a test in which urine is collected for 24 hours to measure the amounts of certain substances. An unusual (higher or lower than normal) amount of a substance can be a sign of disease in the organ or tissue that makes it. A higher than normal amount of homovanillic acid (HMA) and vanillyl mandelic acid (VMA) may be a sign of neuroblastoma. Studies have shown that screening for neuroblastoma does not decrease the chance of dying from the disease. Almost all neuroblastomas that are found by screening children at 6 months of age are the type that have a good prognosis (chance of recovery). Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Decisions about screening tests can be difficult. Not all screening tests are helpful and most have risks. Before having any screening test, you may want to discuss the test with your doctor. It is important to know the risks of the test and whether it has been proven to reduce the risk of dying from cancer. When a screening test result leads to the diagnosis and treatment of a disease that may never have caused symptoms or become life-threatening, it is called overdiagnosis. For example, when a urine test result shows a higher than normal amount of homovanillic acid (HMA) or vanillyl mandelic acid (VMA), tests and treatments for neuroblastoma are likely to be done, but may not be needed. At this time, it is not possible to know which neuroblastomas found by a screening test will cause symptoms and which neuroblastomas will not. Diagnostic tests (such as biopsies) and cancer treatments (such as surgery, radiation therapy, and chemotherapy) can have serious risks, including physical and emotional problems. Screening test results may appear to be normal even though neuroblastoma is present. A person who receives a false-negative test result (one that shows there is no cancer when there really is) may delay seeking medical care even if there are symptoms. Screening test results may appear to be abnormal even though no cancer is present. A false-positive test result (one that shows there is cancer when there really isn't) can cause anxiety and is usually followed by more tests and procedures, which also have risks. Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about neuroblastoma screening. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Screening and Prevention Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Screening and Prevention Editorial Board. PDQ Neuroblastoma Screening. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/neuroblastoma/patient/neuroblastoma-screening-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389302] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Neuroblastoma Screening (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Neuroblastoma ?

Key Points - Neuroblastoma is a disease in which malignant (cancer) cells form in nerve tissue. - Most cases of neuroblastoma are diagnosed before 1 year of age. - The risk factors for neuroblastoma are not known. Neuroblastoma is a disease in which malignant (cancer) cells form in nerve tissue. Neuroblastoma often begins in the nerve tissue of the adrenal glands. There are two adrenal glands, one on top of each kidney, in the back of the upper abdomen. The adrenal glands make important hormones that help control heart rate, blood pressure, blood sugar, and the way the body reacts to stress. Neuroblastoma may also begin in the abdomen, chest, spinal cord, or in nerve tissue near the spine in the neck. Neuroblastoma most often begins during early childhood, usually in children younger than 5 years of age. See the PDQ summary on Neuroblastoma Treatment for more information about neuroblastoma. Most cases of neuroblastoma are diagnosed before 1 year of age. Neuroblastoma is the most common type of cancer in infants. The number of new cases of neuroblastoma is greatest among children under 1 year of age. As children get older, the number of new cases decreases. Neuroblastoma is slightly more common in males than females. Neuroblastoma sometimes forms before birth but is usually found later, when the tumor begins to grow and cause symptoms. In rare cases, neuroblastoma may be found before birth, by fetal ultrasound.

Screening is looking for cancer before a person has any symptoms. This can help find cancer at an early stage. When abnormal tissue or cancer is found early, it may be easier to treat. By the time symptoms appear, cancer may have begun to spread. Scientists are trying to better understand which people are more likely to get certain types of cancer. They also study the things we do and the things around us to see if they cause cancer. This information helps doctors recommend who should be screened for cancer, which screening tests should be used, and how often the tests should be done. It is important to remember that your doctor does not necessarily think you have cancer if he or she suggests a screening test. Screening tests are given when you have no cancer symptoms. If a screening test result is abnormal, you may need to have more tests done to find out if you have cancer. These are called diagnostic tests. Neuroblastoma often begins in the nerve tissue of the adrenal glands. There are two adrenal glands, one on top of each kidney, in the back of the upper abdomen. The adrenal glands make important hormones that help control heart rate, blood pressure, blood sugar, and the way the body reacts to stress. Neuroblastoma may also begin in the abdomen, chest, spinal cord, or in nerve tissue near the spine in the neck. Neuroblastoma most often begins during early childhood, usually in children younger than 5 years of age. See the PDQ summary on Neuroblastoma Treatment for more information about neuroblastoma. Neuroblastoma is the most common type of cancer in infants. The number of new cases of neuroblastoma is greatest among children under 1 year of age. As children get older, the number of new cases decreases. Neuroblastoma is slightly more common in males than females. Neuroblastoma sometimes forms before birth but is usually found later, when the tumor begins to grow and cause symptoms. In rare cases, neuroblastoma may be found before birth, by fetal ultrasound. Scientists study screening tests to find those with the fewest harms and most benefits. Cancer screening trials also are meant to show whether early detection (finding cancer before it causes symptoms) helps a person live longer or decreases a person's chance of dying from the disease. For some types of cancer, the chance of recovery is better if the disease is found and treated at an early stage. There is no standard or routine screening test used to find neuroblastoma. A urine test is sometimes used to check for neuroblastoma, usually when the child is 6 months old. This is a test in which urine is collected for 24 hours to measure the amounts of certain substances. An unusual (higher or lower than normal) amount of a substance can be a sign of disease in the organ or tissue that makes it. A higher than normal amount of homovanillic acid (HMA) and vanillyl mandelic acid (VMA) may be a sign of neuroblastoma. Studies have shown that screening for neuroblastoma does not decrease the chance of dying from the disease. Almost all neuroblastomas that are found by screening children at 6 months of age are the type that have a good prognosis (chance of recovery). Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Decisions about screening tests can be difficult. Not all screening tests are helpful and most have risks. Before having any screening test, you may want to discuss the test with your doctor. It is important to know the risks of the test and whether it has been proven to reduce the risk of dying from cancer. When a screening test result leads to the diagnosis and treatment of a disease that may never have caused symptoms or become life-threatening, it is called overdiagnosis. For example, when a urine test result shows a higher than normal amount of homovanillic acid (HMA) or vanillyl mandelic acid (VMA), tests and treatments for neuroblastoma are likely to be done, but may not be needed. At this time, it is not possible to know which neuroblastomas found by a screening test will cause symptoms and which neuroblastomas will not. Diagnostic tests (such as biopsies) and cancer treatments (such as surgery, radiation therapy, and chemotherapy) can have serious risks, including physical and emotional problems. Screening test results may appear to be normal even though neuroblastoma is present. A person who receives a false-negative test result (one that shows there is no cancer when there really is) may delay seeking medical care even if there are symptoms. Screening test results may appear to be abnormal even though no cancer is present. A false-positive test result (one that shows there is cancer when there really isn't) can cause anxiety and is usually followed by more tests and procedures, which also have risks. Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about neuroblastoma screening. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Screening and Prevention Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Screening and Prevention Editorial Board. PDQ Neuroblastoma Screening. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/neuroblastoma/patient/neuroblastoma-screening-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389302] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Neuroblastoma Screening (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Neuroblastoma? ?

The risk factors for neuroblastoma are not known.

Screening is looking for cancer before a person has any symptoms. This can help find cancer at an early stage. When abnormal tissue or cancer is found early, it may be easier to treat. By the time symptoms appear, cancer may have begun to spread. Scientists are trying to better understand which people are more likely to get certain types of cancer. They also study the things we do and the things around us to see if they cause cancer. This information helps doctors recommend who should be screened for cancer, which screening tests should be used, and how often the tests should be done. It is important to remember that your doctor does not necessarily think you have cancer if he or she suggests a screening test. Screening tests are given when you have no cancer symptoms. If a screening test result is abnormal, you may need to have more tests done to find out if you have cancer. These are called diagnostic tests. Neuroblastoma often begins in the nerve tissue of the adrenal glands. There are two adrenal glands, one on top of each kidney, in the back of the upper abdomen. The adrenal glands make important hormones that help control heart rate, blood pressure, blood sugar, and the way the body reacts to stress. Neuroblastoma may also begin in the abdomen, chest, spinal cord, or in nerve tissue near the spine in the neck. Neuroblastoma most often begins during early childhood, usually in children younger than 5 years of age. See the PDQ summary on Neuroblastoma Treatment for more information about neuroblastoma. Neuroblastoma is the most common type of cancer in infants. The number of new cases of neuroblastoma is greatest among children under 1 year of age. As children get older, the number of new cases decreases. Neuroblastoma is slightly more common in males than females. Neuroblastoma sometimes forms before birth but is usually found later, when the tumor begins to grow and cause symptoms. In rare cases, neuroblastoma may be found before birth, by fetal ultrasound. Scientists study screening tests to find those with the fewest harms and most benefits. Cancer screening trials also are meant to show whether early detection (finding cancer before it causes symptoms) helps a person live longer or decreases a person's chance of dying from the disease. For some types of cancer, the chance of recovery is better if the disease is found and treated at an early stage. There is no standard or routine screening test used to find neuroblastoma. A urine test is sometimes used to check for neuroblastoma, usually when the child is 6 months old. This is a test in which urine is collected for 24 hours to measure the amounts of certain substances. An unusual (higher or lower than normal) amount of a substance can be a sign of disease in the organ or tissue that makes it. A higher than normal amount of homovanillic acid (HMA) and vanillyl mandelic acid (VMA) may be a sign of neuroblastoma. Studies have shown that screening for neuroblastoma does not decrease the chance of dying from the disease. Almost all neuroblastomas that are found by screening children at 6 months of age are the type that have a good prognosis (chance of recovery). Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Decisions about screening tests can be difficult. Not all screening tests are helpful and most have risks. Before having any screening test, you may want to discuss the test with your doctor. It is important to know the risks of the test and whether it has been proven to reduce the risk of dying from cancer. When a screening test result leads to the diagnosis and treatment of a disease that may never have caused symptoms or become life-threatening, it is called overdiagnosis. For example, when a urine test result shows a higher than normal amount of homovanillic acid (HMA) or vanillyl mandelic acid (VMA), tests and treatments for neuroblastoma are likely to be done, but may not be needed. At this time, it is not possible to know which neuroblastomas found by a screening test will cause symptoms and which neuroblastomas will not. Diagnostic tests (such as biopsies) and cancer treatments (such as surgery, radiation therapy, and chemotherapy) can have serious risks, including physical and emotional problems. Screening test results may appear to be normal even though neuroblastoma is present. A person who receives a false-negative test result (one that shows there is no cancer when there really is) may delay seeking medical care even if there are symptoms. Screening test results may appear to be abnormal even though no cancer is present. A false-positive test result (one that shows there is cancer when there really isn't) can cause anxiety and is usually followed by more tests and procedures, which also have risks. Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about neuroblastoma screening. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Screening and Prevention Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Screening and Prevention Editorial Board. PDQ Neuroblastoma Screening. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/neuroblastoma/patient/neuroblastoma-screening-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389302] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Neuroblastoma Screening (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Neuroblastoma? ?

Key Points - Screening tests have risks. - The risks of neuroblastoma screening include the following: - Neuroblastoma may be overdiagnosed. - False-negative test results can occur. - False-positive test results can occur. Screening tests have risks. Decisions about screening tests can be difficult. Not all screening tests are helpful and most have risks. Before having any screening test, you may want to discuss the test with your doctor. It is important to know the risks of the test and whether it has been proven to reduce the risk of dying from cancer. The risks of neuroblastoma screening include the following: Neuroblastoma may be overdiagnosed. When a screening test result leads to the diagnosis and treatment of a disease that may never have caused symptoms or become life-threatening, it is called overdiagnosis. For example, when a urine test result shows a higher than normal amount of homovanillic acid (HMA) or vanillyl mandelic acid (VMA), tests and treatments for neuroblastoma are likely to be done, but may not be needed. At this time, it is not possible to know which neuroblastomas found by a screening test will cause symptoms and which neuroblastomas will not. Diagnostic tests (such as biopsies) and cancer treatments (such as surgery, radiation therapy, and chemotherapy) can have serious risks, including physical and emotional problems. False-negative test results can occur. Screening test results may appear to be normal even though neuroblastoma is present. A person who receives a false-negative test result (one that shows there is no cancer when there really is) may delay seeking medical care even if there are symptoms. False-positive test results can occur. Screening test results may appear to be abnormal even though no cancer is present. A false-positive test result (one that shows there is cancer when there really isn&apos;t) can cause anxiety and is usually followed by more tests and procedures, which also have risks.

The lungs are a pair of cone-shaped breathing organs in the chest. The lungs bring oxygen into the body as you breathe in. They release carbon dioxide, a waste product of the body’s cells, as you breathe out. Each lung has sections called lobes. The left lung has two lobes. The right lung is slightly larger and has three lobes. Two tubes called bronchi lead from the trachea (windpipe) to the right and left lungs. The bronchi are sometimes also involved in lung cancer. Tiny air sacs called alveoli and small tubes called bronchioles make up the inside of the lungs.EnlargeAnatomy of the respiratory system showing the trachea, the right and left lungs and their lobes, and the bronchi. The lymph nodes and the diaphragm are also shown. Oxygen is inhaled into the lungs and passes through the alveoli (the tiny air sacs at the end of the bronchioles) and into the bloodstream (see inset), where it travels to the tissues throughout the body. A thin membrane called the pleura covers the outside of each lung and lines the inside wall of the chest cavity. This creates a sac called the pleural cavity. The pleural cavity normally contains a small amount of fluid that helps the lungs move smoothly in the chest when you breathe. There are two main types of lung cancer: non-small cell lung cancer and small cell lung cancer. See the following PDQ summaries for more information about lung cancer: Each type of non-small cell lung cancer has different kinds of cancer cells. The cancer cells of each type grow and spread in different ways. The types of non-small cell lung cancer are named for the kinds of cells found in the cancer and how the cells look under a microscope: Other less common types of non-small cell lung cancer are: adenosquamous carcinoma, sarcomatoid carcinoma, salivary gland carcinoma, carcinoid tumor, and unclassified carcinoma. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk to your doctor if you think you may be at risk for lung cancer. Risk factors for lung cancer include the following: Older age is the main risk factor for most cancers. The chance of getting cancer increases as you get older. When smoking is combined with other risk factors, the risk of lung cancer is increased. Sometimes lung cancer does not cause any signs or symptoms. It may be found during a chest x-ray done for another condition. Signs and symptoms may be caused by lung cancer or by other conditions. Check with your doctor if you have any of the following: Tests and procedures to diagnose and stage non-small cell lung cancer are often done at the same time. Some of the following tests and procedures may be used: One of the following types of biopsies is usually used: An endoscopic ultrasound (EUS) is a type of ultrasound that may be used to guide an FNA biopsy of the lung, lymph nodes, or other areas. EUS is a procedure in which an endoscope is inserted into the body. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. A probe at the end of the endoscope is used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. One or more of the following laboratory tests may be done to study the tissue samples: The prognosis and treatment options depend on the following: If lung cancer is found, taking part in one of the many clinical trials being done to improve treatment should be considered. Clinical trials are taking place in most parts of the country for patients with all stages of non-small cell lung cancer. Information about ongoing clinical trials is available from the NCI website. The process used to find out if cancer has spread within the lungs or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some of the tests used to diagnose non-small cell lung cancer are also used to stage the disease. For more information, see the General Information section. Other tests and procedures that may be used in the staging process include the following: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if non-small cell lung cancer spreads to the brain, the cancer cells in the brain are actually lung cancer cells. The disease is metastatic lung cancer, not brain cancer. In the occult (hidden) stage, cancer cannot be seen by imaging or bronchoscopy. Cancer cells are found in sputum or bronchial washings (a sample of cells taken from inside the airways that lead to the lungs). Cancer may have spread to other parts of the body. In stage 0, abnormal cells are found in the lining of the airways. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 may be adenocarcinoma in situ (AIS) or squamous cell carcinoma in situ (SCIS). In stage I, cancer has formed. Stage I is divided into stages IA and IB. The tumor is in the lung only and is 3 centimeters or smaller. Cancer has not spread to the lymph nodes. The tumor is larger than 3 centimeters but not larger than 4 centimeters. Cancer has not spread to the lymph nodes. or The tumor is 4 centimeters or smaller and one or more of the following is found: Cancer has not spread to the lymph nodes. Stage II is divided into stages IIA and IIB. The tumor is larger than 4 centimeters but not larger than 5 centimeters. Cancer has not spread to the lymph nodes and one or more of the following may be found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following may be found: or Cancer has not spread to the lymph nodes and one or more of the following is found: Stage III is divided into stages IIIA, IIIB, and IIIC. The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following may be found: or Cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: or Cancer may have spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following may be found: or The tumor may be any size and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following is found: The tumor may be any size and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following is found: Stage IV is divided into stages IVA and IVB. The tumor may be any size and cancer may have spread to the lymph nodes. One or more of the following is found: Cancer has spread to multiple places in one or more organs that are not near the lung. The cancer may come back in the brain, lung, or other parts of the body. Different types of treatments are available for patients with non-small cell lung cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Four types of surgery are used to treat lung cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Stereotactic body radiation therapy is a type of external radiation therapy. Special equipment is used to place the patient in the same position for each radiation treatment. Once a day for several days, a radiation machine aims a larger than usual dose of radiation directly at the tumor. By having the patient in the same position for each treatment, there is less damage to nearby healthy tissue. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. Stereotactic radiosurgery is a type of external radiation therapy used to treat lung cancer that has spread to the brain. A rigid head frame is attached to the skull to keep the head still during the radiation treatment. A machine aims a single large dose of radiation directly at the tumor in the brain. This procedure does not involve surgery. It is also called stereotaxic radiosurgery, radiosurgery, and radiation surgery. For tumors in the airways, radiation is given directly to the tumor through an endoscope. The way the radiation therapy is given depends on the type and stage of the cancer being treated. It also depends on where the cancer is found. External and internal radiation therapy are used to treat non-small cell lung cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Monoclonal antibodies, tyrosine kinase inhibitors, mammalian target of rapamycin (mTOR) inhibitors, and KRAS G12C inhibitors are four types of targeted therapy being used to treat advanced, metastatic, or recurrent non-small cell lung cancer. Monoclonal antibodies Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. There are different types of monoclonal antibody therapy: Tyrosine kinase inhibitors Tyrosine kinase inhibitors are small-molecule drugs that go through the cell membrane and work inside cancer cells to block signals that cancer cells need to grow and divide. Some tyrosine kinase inhibitors also have angiogenesis inhibitor effects. There are different types of tyrosine kinase inhibitors: Mammalian target of rapamycin (mTOR) inhibitors mTOR inhibitors block a protein called mTOR, which may keep cancer cells from growing and prevent the growth of new blood vessels that tumors need to grow. Everolimus is a type of mTOR inhibitor. KRAS G12C inhibitors KRAS G12C inhibitors block a protein called KRAS p.G12C, which may help keep cancer cells from growing and may kill them. Sotorasib and adagrasib are types of KRAS G12C inhibitors. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy used to treat some patients with advanced non-small-cell lung cancer. Types of immune checkpoint inhibitor therapy include: For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Laser therapy is a cancer treatment that uses a laser beam (a narrow beam of intense light) to kill cancer cells. Photodynamic therapy (PDT) is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. Fiberoptic tubes are then used to carry the laser light to the cancer cells, where the drug becomes active and kills the cells. Photodynamic therapy causes little damage to healthy tissue. It is used mainly to treat tumors on or just under the skin or in the lining of internal organs. When the tumor is in the airways, PDT is given directly to the tumor through an endoscope. Cryosurgery is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as carcinoma in situ. This type of treatment is also called cryotherapy. For tumors in the airways, cryosurgery is done through an endoscope. Electrocautery is a treatment that uses a probe or needle heated by an electric current to destroy abnormal tissue. For tumors in the airways, electrocautery is done through an endoscope. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are substances that make tumor cells easier to kill with radiation therapy. The combination of chemotherapy and radiation therapy given with a radiosensitizer is being studied in the treatment of non-small cell lung cancer. New combinations of treatments are being studied in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of occult non-small cell lung cancer depends on the stage of the disease. Occult tumors are often found at an early stage (the tumor is in the lung only) and sometimes can be cured by surgery. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IA non-small cell lung cancer and stage IB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIA non-small cell lung cancer and stage IIB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIA non-small cell lung cancer that can be removed with surgery may include the following: Treatment of stage IIIA non-small cell lung cancer that cannot be removed with surgery may include the following: For more information about supportive care for signs and symptoms including cough, shortness of breath, and chest pain, see Cardiopulmonary Syndromes. Non-small cell lung cancer of the superior sulcus, often called Pancoast tumor, begins in the upper part of the lung and spreads to nearby tissues such as the chest wall, large blood vessels, and spine. Treatment of Pancoast tumors may include the following: Some stage IIIA non-small cell lung tumors that have grown into the chest wall may be completely removed. Treatment of chest wall tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIB non-small cell lung cancer and stage IIIC non-small cell lung cancer may include the following: For more information about supportive care for signs and symptoms such as cough, shortness of breath, and chest pain, see the following PDQ summaries: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about non-small cell lung cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of non-small cell lung cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. 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What is (are) Non-Small Cell Lung Cancer ?

Key Points - Non-small cell lung cancer is a disease in which malignant (cancer) cells form in the tissues of the lung. - There are several types of non-small cell lung cancer. - Smoking is the major risk factor for non-small cell lung cancer. - Signs of non-small cell lung cancer include a cough that doesn&apos;t go away and shortness of breath. - Tests that examine the lungs are used to detect (find), diagnose, and stage non-small cell lung cancer. - Certain factors affect prognosis (chance of recovery) and treatment options. - For most patients with non-small cell lung cancer, current treatments do not cure the cancer. Non-small cell lung cancer is a disease in which malignant (cancer) cells form in the tissues of the lung. The lungs are a pair of cone-shaped breathing organs in the chest. The lungs bring oxygen into the body as you breathe in. They release carbon dioxide, a waste product of the bodys cells, as you breathe out. Each lung has sections called lobes. The left lung has two lobes. The right lung is slightly larger and has three lobes. Two tubes called bronchi lead from the trachea (windpipe) to the right and left lungs. The bronchi are sometimes also involved in lung cancer. Tiny air sacs called alveoli and small tubes called bronchioles make up the inside of the lungs. A thin membrane called the pleura covers the outside of each lung and lines the inside wall of the chest cavity. This creates a sac called the pleural cavity. The pleural cavity normally contains a small amount of fluid that helps the lungs move smoothly in the chest when you breathe. There are two main types of lung cancer: non-small cell lung cancer and small cell lung cancer. See the following PDQ summaries for more information about lung cancer: - Small Cell Lung Cancer Treatment - Unusual Cancers of Childhood Treatment - Lung Cancer Prevention - Lung Cancer Screening There are several types of non-small cell lung cancer. Each type of non-small cell lung cancer has different kinds of cancer cells. The cancer cells of each type grow and spread in different ways. The types of non-small cell lung cancer are named for the kinds of cells found in the cancer and how the cells look under a microscope: - Squamous cell carcinoma: Cancer that begins in squamous cells, which are thin, flat cells that look like fish scales. This is also called epidermoid carcinoma. - Large cell carcinoma: Cancer that may begin in several types of large cells. - Adenocarcinoma: Cancer that begins in the cells that line the alveoli and make substances such as mucus. Other less common types of non-small cell lung cancer are: pleomorphic, carcinoid tumor, salivary gland carcinoma, and unclassified carcinoma. For most patients with non-small cell lung cancer, current treatments do not cure the cancer. If lung cancer is found, taking part in one of the many clinical trials being done to improve treatment should be considered. Clinical trials are taking place in most parts of the country for patients with all stages of non-small cell lung cancer. Information about ongoing clinical trials is available from the NCI website.

The lungs are a pair of cone-shaped breathing organs in the chest. The lungs bring oxygen into the body as you breathe in. They release carbon dioxide, a waste product of the body’s cells, as you breathe out. Each lung has sections called lobes. The left lung has two lobes. The right lung is slightly larger and has three lobes. Two tubes called bronchi lead from the trachea (windpipe) to the right and left lungs. The bronchi are sometimes also involved in lung cancer. Tiny air sacs called alveoli and small tubes called bronchioles make up the inside of the lungs.EnlargeAnatomy of the respiratory system showing the trachea, the right and left lungs and their lobes, and the bronchi. The lymph nodes and the diaphragm are also shown. Oxygen is inhaled into the lungs and passes through the alveoli (the tiny air sacs at the end of the bronchioles) and into the bloodstream (see inset), where it travels to the tissues throughout the body. A thin membrane called the pleura covers the outside of each lung and lines the inside wall of the chest cavity. This creates a sac called the pleural cavity. The pleural cavity normally contains a small amount of fluid that helps the lungs move smoothly in the chest when you breathe. There are two main types of lung cancer: non-small cell lung cancer and small cell lung cancer. See the following PDQ summaries for more information about lung cancer: Each type of non-small cell lung cancer has different kinds of cancer cells. The cancer cells of each type grow and spread in different ways. The types of non-small cell lung cancer are named for the kinds of cells found in the cancer and how the cells look under a microscope: Other less common types of non-small cell lung cancer are: adenosquamous carcinoma, sarcomatoid carcinoma, salivary gland carcinoma, carcinoid tumor, and unclassified carcinoma. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk to your doctor if you think you may be at risk for lung cancer. Risk factors for lung cancer include the following: Older age is the main risk factor for most cancers. The chance of getting cancer increases as you get older. When smoking is combined with other risk factors, the risk of lung cancer is increased. Sometimes lung cancer does not cause any signs or symptoms. It may be found during a chest x-ray done for another condition. Signs and symptoms may be caused by lung cancer or by other conditions. Check with your doctor if you have any of the following: Tests and procedures to diagnose and stage non-small cell lung cancer are often done at the same time. Some of the following tests and procedures may be used: One of the following types of biopsies is usually used: An endoscopic ultrasound (EUS) is a type of ultrasound that may be used to guide an FNA biopsy of the lung, lymph nodes, or other areas. EUS is a procedure in which an endoscope is inserted into the body. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. A probe at the end of the endoscope is used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. One or more of the following laboratory tests may be done to study the tissue samples: The prognosis and treatment options depend on the following: If lung cancer is found, taking part in one of the many clinical trials being done to improve treatment should be considered. Clinical trials are taking place in most parts of the country for patients with all stages of non-small cell lung cancer. Information about ongoing clinical trials is available from the NCI website. The process used to find out if cancer has spread within the lungs or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some of the tests used to diagnose non-small cell lung cancer are also used to stage the disease. For more information, see the General Information section. Other tests and procedures that may be used in the staging process include the following: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if non-small cell lung cancer spreads to the brain, the cancer cells in the brain are actually lung cancer cells. The disease is metastatic lung cancer, not brain cancer. In the occult (hidden) stage, cancer cannot be seen by imaging or bronchoscopy. Cancer cells are found in sputum or bronchial washings (a sample of cells taken from inside the airways that lead to the lungs). Cancer may have spread to other parts of the body. In stage 0, abnormal cells are found in the lining of the airways. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 may be adenocarcinoma in situ (AIS) or squamous cell carcinoma in situ (SCIS). In stage I, cancer has formed. Stage I is divided into stages IA and IB. The tumor is in the lung only and is 3 centimeters or smaller. Cancer has not spread to the lymph nodes. The tumor is larger than 3 centimeters but not larger than 4 centimeters. Cancer has not spread to the lymph nodes. or The tumor is 4 centimeters or smaller and one or more of the following is found: Cancer has not spread to the lymph nodes. Stage II is divided into stages IIA and IIB. The tumor is larger than 4 centimeters but not larger than 5 centimeters. Cancer has not spread to the lymph nodes and one or more of the following may be found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following may be found: or Cancer has not spread to the lymph nodes and one or more of the following is found: Stage III is divided into stages IIIA, IIIB, and IIIC. The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following may be found: or Cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: or Cancer may have spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following may be found: or The tumor may be any size and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following is found: The tumor may be any size and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following is found: Stage IV is divided into stages IVA and IVB. The tumor may be any size and cancer may have spread to the lymph nodes. One or more of the following is found: Cancer has spread to multiple places in one or more organs that are not near the lung. The cancer may come back in the brain, lung, or other parts of the body. Different types of treatments are available for patients with non-small cell lung cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Four types of surgery are used to treat lung cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Stereotactic body radiation therapy is a type of external radiation therapy. Special equipment is used to place the patient in the same position for each radiation treatment. Once a day for several days, a radiation machine aims a larger than usual dose of radiation directly at the tumor. By having the patient in the same position for each treatment, there is less damage to nearby healthy tissue. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. Stereotactic radiosurgery is a type of external radiation therapy used to treat lung cancer that has spread to the brain. A rigid head frame is attached to the skull to keep the head still during the radiation treatment. A machine aims a single large dose of radiation directly at the tumor in the brain. This procedure does not involve surgery. It is also called stereotaxic radiosurgery, radiosurgery, and radiation surgery. For tumors in the airways, radiation is given directly to the tumor through an endoscope. The way the radiation therapy is given depends on the type and stage of the cancer being treated. It also depends on where the cancer is found. External and internal radiation therapy are used to treat non-small cell lung cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Monoclonal antibodies, tyrosine kinase inhibitors, mammalian target of rapamycin (mTOR) inhibitors, and KRAS G12C inhibitors are four types of targeted therapy being used to treat advanced, metastatic, or recurrent non-small cell lung cancer. Monoclonal antibodies Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. There are different types of monoclonal antibody therapy: Tyrosine kinase inhibitors Tyrosine kinase inhibitors are small-molecule drugs that go through the cell membrane and work inside cancer cells to block signals that cancer cells need to grow and divide. Some tyrosine kinase inhibitors also have angiogenesis inhibitor effects. There are different types of tyrosine kinase inhibitors: Mammalian target of rapamycin (mTOR) inhibitors mTOR inhibitors block a protein called mTOR, which may keep cancer cells from growing and prevent the growth of new blood vessels that tumors need to grow. Everolimus is a type of mTOR inhibitor. KRAS G12C inhibitors KRAS G12C inhibitors block a protein called KRAS p.G12C, which may help keep cancer cells from growing and may kill them. Sotorasib and adagrasib are types of KRAS G12C inhibitors. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy used to treat some patients with advanced non-small-cell lung cancer. Types of immune checkpoint inhibitor therapy include: For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Laser therapy is a cancer treatment that uses a laser beam (a narrow beam of intense light) to kill cancer cells. Photodynamic therapy (PDT) is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. Fiberoptic tubes are then used to carry the laser light to the cancer cells, where the drug becomes active and kills the cells. Photodynamic therapy causes little damage to healthy tissue. It is used mainly to treat tumors on or just under the skin or in the lining of internal organs. When the tumor is in the airways, PDT is given directly to the tumor through an endoscope. Cryosurgery is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as carcinoma in situ. This type of treatment is also called cryotherapy. For tumors in the airways, cryosurgery is done through an endoscope. Electrocautery is a treatment that uses a probe or needle heated by an electric current to destroy abnormal tissue. For tumors in the airways, electrocautery is done through an endoscope. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are substances that make tumor cells easier to kill with radiation therapy. The combination of chemotherapy and radiation therapy given with a radiosensitizer is being studied in the treatment of non-small cell lung cancer. New combinations of treatments are being studied in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of occult non-small cell lung cancer depends on the stage of the disease. Occult tumors are often found at an early stage (the tumor is in the lung only) and sometimes can be cured by surgery. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IA non-small cell lung cancer and stage IB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIA non-small cell lung cancer and stage IIB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIA non-small cell lung cancer that can be removed with surgery may include the following: Treatment of stage IIIA non-small cell lung cancer that cannot be removed with surgery may include the following: For more information about supportive care for signs and symptoms including cough, shortness of breath, and chest pain, see Cardiopulmonary Syndromes. Non-small cell lung cancer of the superior sulcus, often called Pancoast tumor, begins in the upper part of the lung and spreads to nearby tissues such as the chest wall, large blood vessels, and spine. Treatment of Pancoast tumors may include the following: Some stage IIIA non-small cell lung tumors that have grown into the chest wall may be completely removed. Treatment of chest wall tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIB non-small cell lung cancer and stage IIIC non-small cell lung cancer may include the following: For more information about supportive care for signs and symptoms such as cough, shortness of breath, and chest pain, see the following PDQ summaries: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about non-small cell lung cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of non-small cell lung cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Non-Small Cell Lung Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lung/patient/non-small-cell-lung-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389355] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Non-Small Cell Lung Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Non-Small Cell Lung Cancer? ?

Smoking is the major risk factor for non-small cell lung cancer. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn&apos;t mean that you will not get cancer. Talk to your doctor if you think you may be at risk for lung cancer. Risk factors for lung cancer include the following: - Smoking cigarettes, pipes, or cigars, now or in the past. This is the most important risk factor for lung cancer. The earlier in life a person starts smoking, the more often a person smokes, and the more years a person smokes, the greater the risk of lung cancer. - Being exposed to secondhand smoke. - Being exposed to radiation from any of the following: - Radiation therapy to the breast or chest. - Radon in the home or workplace. - Imaging tests such as CT scans. - Atomic bomb radiation. - Being exposed to asbestos, chromium, nickel, beryllium, arsenic, soot, or tar in the workplace. - Living where there is air pollution. - Having a family history of lung cancer. - Being infected with the human immunodeficiency virus (HIV). - Taking beta carotene supplements and being a heavy smoker. Older age is the main risk factor for most cancers. The chance of getting cancer increases as you get older. When smoking is combined with other risk factors, the risk of lung cancer is increased.

The lungs are a pair of cone-shaped breathing organs in the chest. The lungs bring oxygen into the body as you breathe in. They release carbon dioxide, a waste product of the body’s cells, as you breathe out. Each lung has sections called lobes. The left lung has two lobes. The right lung is slightly larger and has three lobes. Two tubes called bronchi lead from the trachea (windpipe) to the right and left lungs. The bronchi are sometimes also involved in lung cancer. Tiny air sacs called alveoli and small tubes called bronchioles make up the inside of the lungs.EnlargeAnatomy of the respiratory system showing the trachea, the right and left lungs and their lobes, and the bronchi. The lymph nodes and the diaphragm are also shown. Oxygen is inhaled into the lungs and passes through the alveoli (the tiny air sacs at the end of the bronchioles) and into the bloodstream (see inset), where it travels to the tissues throughout the body. A thin membrane called the pleura covers the outside of each lung and lines the inside wall of the chest cavity. This creates a sac called the pleural cavity. The pleural cavity normally contains a small amount of fluid that helps the lungs move smoothly in the chest when you breathe. There are two main types of lung cancer: non-small cell lung cancer and small cell lung cancer. See the following PDQ summaries for more information about lung cancer: Each type of non-small cell lung cancer has different kinds of cancer cells. The cancer cells of each type grow and spread in different ways. The types of non-small cell lung cancer are named for the kinds of cells found in the cancer and how the cells look under a microscope: Other less common types of non-small cell lung cancer are: adenosquamous carcinoma, sarcomatoid carcinoma, salivary gland carcinoma, carcinoid tumor, and unclassified carcinoma. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk to your doctor if you think you may be at risk for lung cancer. Risk factors for lung cancer include the following: Older age is the main risk factor for most cancers. The chance of getting cancer increases as you get older. When smoking is combined with other risk factors, the risk of lung cancer is increased. Sometimes lung cancer does not cause any signs or symptoms. It may be found during a chest x-ray done for another condition. Signs and symptoms may be caused by lung cancer or by other conditions. Check with your doctor if you have any of the following: Tests and procedures to diagnose and stage non-small cell lung cancer are often done at the same time. Some of the following tests and procedures may be used: One of the following types of biopsies is usually used: An endoscopic ultrasound (EUS) is a type of ultrasound that may be used to guide an FNA biopsy of the lung, lymph nodes, or other areas. EUS is a procedure in which an endoscope is inserted into the body. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. A probe at the end of the endoscope is used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. One or more of the following laboratory tests may be done to study the tissue samples: The prognosis and treatment options depend on the following: If lung cancer is found, taking part in one of the many clinical trials being done to improve treatment should be considered. Clinical trials are taking place in most parts of the country for patients with all stages of non-small cell lung cancer. Information about ongoing clinical trials is available from the NCI website. The process used to find out if cancer has spread within the lungs or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some of the tests used to diagnose non-small cell lung cancer are also used to stage the disease. For more information, see the General Information section. Other tests and procedures that may be used in the staging process include the following: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if non-small cell lung cancer spreads to the brain, the cancer cells in the brain are actually lung cancer cells. The disease is metastatic lung cancer, not brain cancer. In the occult (hidden) stage, cancer cannot be seen by imaging or bronchoscopy. Cancer cells are found in sputum or bronchial washings (a sample of cells taken from inside the airways that lead to the lungs). Cancer may have spread to other parts of the body. In stage 0, abnormal cells are found in the lining of the airways. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 may be adenocarcinoma in situ (AIS) or squamous cell carcinoma in situ (SCIS). In stage I, cancer has formed. Stage I is divided into stages IA and IB. The tumor is in the lung only and is 3 centimeters or smaller. Cancer has not spread to the lymph nodes. The tumor is larger than 3 centimeters but not larger than 4 centimeters. Cancer has not spread to the lymph nodes. or The tumor is 4 centimeters or smaller and one or more of the following is found: Cancer has not spread to the lymph nodes. Stage II is divided into stages IIA and IIB. The tumor is larger than 4 centimeters but not larger than 5 centimeters. Cancer has not spread to the lymph nodes and one or more of the following may be found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following may be found: or Cancer has not spread to the lymph nodes and one or more of the following is found: Stage III is divided into stages IIIA, IIIB, and IIIC. The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following may be found: or Cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: or Cancer may have spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following may be found: or The tumor may be any size and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following is found: The tumor may be any size and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following is found: Stage IV is divided into stages IVA and IVB. The tumor may be any size and cancer may have spread to the lymph nodes. One or more of the following is found: Cancer has spread to multiple places in one or more organs that are not near the lung. The cancer may come back in the brain, lung, or other parts of the body. Different types of treatments are available for patients with non-small cell lung cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Four types of surgery are used to treat lung cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Stereotactic body radiation therapy is a type of external radiation therapy. Special equipment is used to place the patient in the same position for each radiation treatment. Once a day for several days, a radiation machine aims a larger than usual dose of radiation directly at the tumor. By having the patient in the same position for each treatment, there is less damage to nearby healthy tissue. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. Stereotactic radiosurgery is a type of external radiation therapy used to treat lung cancer that has spread to the brain. A rigid head frame is attached to the skull to keep the head still during the radiation treatment. A machine aims a single large dose of radiation directly at the tumor in the brain. This procedure does not involve surgery. It is also called stereotaxic radiosurgery, radiosurgery, and radiation surgery. For tumors in the airways, radiation is given directly to the tumor through an endoscope. The way the radiation therapy is given depends on the type and stage of the cancer being treated. It also depends on where the cancer is found. External and internal radiation therapy are used to treat non-small cell lung cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Monoclonal antibodies, tyrosine kinase inhibitors, mammalian target of rapamycin (mTOR) inhibitors, and KRAS G12C inhibitors are four types of targeted therapy being used to treat advanced, metastatic, or recurrent non-small cell lung cancer. Monoclonal antibodies Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. There are different types of monoclonal antibody therapy: Tyrosine kinase inhibitors Tyrosine kinase inhibitors are small-molecule drugs that go through the cell membrane and work inside cancer cells to block signals that cancer cells need to grow and divide. Some tyrosine kinase inhibitors also have angiogenesis inhibitor effects. There are different types of tyrosine kinase inhibitors: Mammalian target of rapamycin (mTOR) inhibitors mTOR inhibitors block a protein called mTOR, which may keep cancer cells from growing and prevent the growth of new blood vessels that tumors need to grow. Everolimus is a type of mTOR inhibitor. KRAS G12C inhibitors KRAS G12C inhibitors block a protein called KRAS p.G12C, which may help keep cancer cells from growing and may kill them. Sotorasib and adagrasib are types of KRAS G12C inhibitors. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy used to treat some patients with advanced non-small-cell lung cancer. Types of immune checkpoint inhibitor therapy include: For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Laser therapy is a cancer treatment that uses a laser beam (a narrow beam of intense light) to kill cancer cells. Photodynamic therapy (PDT) is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. Fiberoptic tubes are then used to carry the laser light to the cancer cells, where the drug becomes active and kills the cells. Photodynamic therapy causes little damage to healthy tissue. It is used mainly to treat tumors on or just under the skin or in the lining of internal organs. When the tumor is in the airways, PDT is given directly to the tumor through an endoscope. Cryosurgery is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as carcinoma in situ. This type of treatment is also called cryotherapy. For tumors in the airways, cryosurgery is done through an endoscope. Electrocautery is a treatment that uses a probe or needle heated by an electric current to destroy abnormal tissue. For tumors in the airways, electrocautery is done through an endoscope. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are substances that make tumor cells easier to kill with radiation therapy. The combination of chemotherapy and radiation therapy given with a radiosensitizer is being studied in the treatment of non-small cell lung cancer. New combinations of treatments are being studied in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of occult non-small cell lung cancer depends on the stage of the disease. Occult tumors are often found at an early stage (the tumor is in the lung only) and sometimes can be cured by surgery. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IA non-small cell lung cancer and stage IB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIA non-small cell lung cancer and stage IIB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIA non-small cell lung cancer that can be removed with surgery may include the following: Treatment of stage IIIA non-small cell lung cancer that cannot be removed with surgery may include the following: For more information about supportive care for signs and symptoms including cough, shortness of breath, and chest pain, see Cardiopulmonary Syndromes. Non-small cell lung cancer of the superior sulcus, often called Pancoast tumor, begins in the upper part of the lung and spreads to nearby tissues such as the chest wall, large blood vessels, and spine. Treatment of Pancoast tumors may include the following: Some stage IIIA non-small cell lung tumors that have grown into the chest wall may be completely removed. Treatment of chest wall tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIB non-small cell lung cancer and stage IIIC non-small cell lung cancer may include the following: For more information about supportive care for signs and symptoms such as cough, shortness of breath, and chest pain, see the following PDQ summaries: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about non-small cell lung cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of non-small cell lung cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Non-Small Cell Lung Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lung/patient/non-small-cell-lung-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389355] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Non-Small Cell Lung Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Non-Small Cell Lung Cancer ?

Signs of non-small cell lung cancer include a cough that doesn&apos;t go away and shortness of breath. Sometimes lung cancer does not cause any signs or symptoms. It may be found during a chest x-ray done for another condition. Signs and symptoms may be caused by lung cancer or by other conditions. Check with your doctor if you have any of the following: - Chest discomfort or pain. - A cough that doesnt go away or gets worse over time. - Trouble breathing. - Wheezing. - Blood in sputum (mucus coughed up from the lungs). - Hoarseness. - Loss of appetite. - Weight loss for no known reason. - Feeling very tired. - Trouble swallowing. - Swelling in the face and/or veins in the neck.

The lungs are a pair of cone-shaped breathing organs in the chest. The lungs bring oxygen into the body as you breathe in. They release carbon dioxide, a waste product of the body’s cells, as you breathe out. Each lung has sections called lobes. The left lung has two lobes. The right lung is slightly larger and has three lobes. Two tubes called bronchi lead from the trachea (windpipe) to the right and left lungs. The bronchi are sometimes also involved in lung cancer. Tiny air sacs called alveoli and small tubes called bronchioles make up the inside of the lungs.EnlargeAnatomy of the respiratory system showing the trachea, the right and left lungs and their lobes, and the bronchi. The lymph nodes and the diaphragm are also shown. Oxygen is inhaled into the lungs and passes through the alveoli (the tiny air sacs at the end of the bronchioles) and into the bloodstream (see inset), where it travels to the tissues throughout the body. A thin membrane called the pleura covers the outside of each lung and lines the inside wall of the chest cavity. This creates a sac called the pleural cavity. The pleural cavity normally contains a small amount of fluid that helps the lungs move smoothly in the chest when you breathe. There are two main types of lung cancer: non-small cell lung cancer and small cell lung cancer. See the following PDQ summaries for more information about lung cancer: Each type of non-small cell lung cancer has different kinds of cancer cells. The cancer cells of each type grow and spread in different ways. The types of non-small cell lung cancer are named for the kinds of cells found in the cancer and how the cells look under a microscope: Other less common types of non-small cell lung cancer are: adenosquamous carcinoma, sarcomatoid carcinoma, salivary gland carcinoma, carcinoid tumor, and unclassified carcinoma. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk to your doctor if you think you may be at risk for lung cancer. Risk factors for lung cancer include the following: Older age is the main risk factor for most cancers. The chance of getting cancer increases as you get older. When smoking is combined with other risk factors, the risk of lung cancer is increased. Sometimes lung cancer does not cause any signs or symptoms. It may be found during a chest x-ray done for another condition. Signs and symptoms may be caused by lung cancer or by other conditions. Check with your doctor if you have any of the following: Tests and procedures to diagnose and stage non-small cell lung cancer are often done at the same time. Some of the following tests and procedures may be used: One of the following types of biopsies is usually used: An endoscopic ultrasound (EUS) is a type of ultrasound that may be used to guide an FNA biopsy of the lung, lymph nodes, or other areas. EUS is a procedure in which an endoscope is inserted into the body. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. A probe at the end of the endoscope is used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. One or more of the following laboratory tests may be done to study the tissue samples: The prognosis and treatment options depend on the following: If lung cancer is found, taking part in one of the many clinical trials being done to improve treatment should be considered. Clinical trials are taking place in most parts of the country for patients with all stages of non-small cell lung cancer. Information about ongoing clinical trials is available from the NCI website. The process used to find out if cancer has spread within the lungs or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some of the tests used to diagnose non-small cell lung cancer are also used to stage the disease. For more information, see the General Information section. Other tests and procedures that may be used in the staging process include the following: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if non-small cell lung cancer spreads to the brain, the cancer cells in the brain are actually lung cancer cells. The disease is metastatic lung cancer, not brain cancer. In the occult (hidden) stage, cancer cannot be seen by imaging or bronchoscopy. Cancer cells are found in sputum or bronchial washings (a sample of cells taken from inside the airways that lead to the lungs). Cancer may have spread to other parts of the body. In stage 0, abnormal cells are found in the lining of the airways. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 may be adenocarcinoma in situ (AIS) or squamous cell carcinoma in situ (SCIS). In stage I, cancer has formed. Stage I is divided into stages IA and IB. The tumor is in the lung only and is 3 centimeters or smaller. Cancer has not spread to the lymph nodes. The tumor is larger than 3 centimeters but not larger than 4 centimeters. Cancer has not spread to the lymph nodes. or The tumor is 4 centimeters or smaller and one or more of the following is found: Cancer has not spread to the lymph nodes. Stage II is divided into stages IIA and IIB. The tumor is larger than 4 centimeters but not larger than 5 centimeters. Cancer has not spread to the lymph nodes and one or more of the following may be found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following may be found: or Cancer has not spread to the lymph nodes and one or more of the following is found: Stage III is divided into stages IIIA, IIIB, and IIIC. The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following may be found: or Cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: or Cancer may have spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following may be found: or The tumor may be any size and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following is found: The tumor may be any size and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following is found: Stage IV is divided into stages IVA and IVB. The tumor may be any size and cancer may have spread to the lymph nodes. One or more of the following is found: Cancer has spread to multiple places in one or more organs that are not near the lung. The cancer may come back in the brain, lung, or other parts of the body. Different types of treatments are available for patients with non-small cell lung cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Four types of surgery are used to treat lung cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Stereotactic body radiation therapy is a type of external radiation therapy. Special equipment is used to place the patient in the same position for each radiation treatment. Once a day for several days, a radiation machine aims a larger than usual dose of radiation directly at the tumor. By having the patient in the same position for each treatment, there is less damage to nearby healthy tissue. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. Stereotactic radiosurgery is a type of external radiation therapy used to treat lung cancer that has spread to the brain. A rigid head frame is attached to the skull to keep the head still during the radiation treatment. A machine aims a single large dose of radiation directly at the tumor in the brain. This procedure does not involve surgery. It is also called stereotaxic radiosurgery, radiosurgery, and radiation surgery. For tumors in the airways, radiation is given directly to the tumor through an endoscope. The way the radiation therapy is given depends on the type and stage of the cancer being treated. It also depends on where the cancer is found. External and internal radiation therapy are used to treat non-small cell lung cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Monoclonal antibodies, tyrosine kinase inhibitors, mammalian target of rapamycin (mTOR) inhibitors, and KRAS G12C inhibitors are four types of targeted therapy being used to treat advanced, metastatic, or recurrent non-small cell lung cancer. Monoclonal antibodies Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. There are different types of monoclonal antibody therapy: Tyrosine kinase inhibitors Tyrosine kinase inhibitors are small-molecule drugs that go through the cell membrane and work inside cancer cells to block signals that cancer cells need to grow and divide. Some tyrosine kinase inhibitors also have angiogenesis inhibitor effects. There are different types of tyrosine kinase inhibitors: Mammalian target of rapamycin (mTOR) inhibitors mTOR inhibitors block a protein called mTOR, which may keep cancer cells from growing and prevent the growth of new blood vessels that tumors need to grow. Everolimus is a type of mTOR inhibitor. KRAS G12C inhibitors KRAS G12C inhibitors block a protein called KRAS p.G12C, which may help keep cancer cells from growing and may kill them. Sotorasib and adagrasib are types of KRAS G12C inhibitors. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy used to treat some patients with advanced non-small-cell lung cancer. Types of immune checkpoint inhibitor therapy include: For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Laser therapy is a cancer treatment that uses a laser beam (a narrow beam of intense light) to kill cancer cells. Photodynamic therapy (PDT) is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. Fiberoptic tubes are then used to carry the laser light to the cancer cells, where the drug becomes active and kills the cells. Photodynamic therapy causes little damage to healthy tissue. It is used mainly to treat tumors on or just under the skin or in the lining of internal organs. When the tumor is in the airways, PDT is given directly to the tumor through an endoscope. Cryosurgery is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as carcinoma in situ. This type of treatment is also called cryotherapy. For tumors in the airways, cryosurgery is done through an endoscope. Electrocautery is a treatment that uses a probe or needle heated by an electric current to destroy abnormal tissue. For tumors in the airways, electrocautery is done through an endoscope. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are substances that make tumor cells easier to kill with radiation therapy. The combination of chemotherapy and radiation therapy given with a radiosensitizer is being studied in the treatment of non-small cell lung cancer. New combinations of treatments are being studied in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of occult non-small cell lung cancer depends on the stage of the disease. Occult tumors are often found at an early stage (the tumor is in the lung only) and sometimes can be cured by surgery. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IA non-small cell lung cancer and stage IB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIA non-small cell lung cancer and stage IIB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIA non-small cell lung cancer that can be removed with surgery may include the following: Treatment of stage IIIA non-small cell lung cancer that cannot be removed with surgery may include the following: For more information about supportive care for signs and symptoms including cough, shortness of breath, and chest pain, see Cardiopulmonary Syndromes. Non-small cell lung cancer of the superior sulcus, often called Pancoast tumor, begins in the upper part of the lung and spreads to nearby tissues such as the chest wall, large blood vessels, and spine. Treatment of Pancoast tumors may include the following: Some stage IIIA non-small cell lung tumors that have grown into the chest wall may be completely removed. Treatment of chest wall tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIB non-small cell lung cancer and stage IIIC non-small cell lung cancer may include the following: For more information about supportive care for signs and symptoms such as cough, shortness of breath, and chest pain, see the following PDQ summaries: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about non-small cell lung cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of non-small cell lung cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. 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How to diagnose Non-Small Cell Lung Cancer ?

Tests that examine the lungs are used to detect (find), diagnose, and stage non-small cell lung cancer. Tests and procedures to detect, diagnose, and stage non-small cell lung cancer are often done at the same time. Some of the following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits, including smoking, and past jobs, illnesses, and treatments will also be taken. - Laboratory tests : Medical procedures that test samples of tissue, blood, urine, or other substances in the body. These tests help to diagnose disease, plan and check treatment, or monitor the disease over time. - Chest x-ray: An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the chest, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - Sputum cytology : A procedure in which a pathologist views a sample of sputum (mucus coughed up from the lungs) under a microscope, to check for cancer cells. - Fine-needle aspiration (FNA) biopsy of the lung: The removal of tissue or fluid from the lung using a thin needle. A CT scan, ultrasound, or other imaging procedure is used to locate the abnormal tissue or fluid in the lung. A small incision may be made in the skin where the biopsy needle is inserted into the abnormal tissue or fluid. A sample is removed with the needle and sent to the laboratory. A pathologist then views the sample under a microscope to look for cancer cells. A chest x-ray is done after the procedure to make sure no air is leaking from the lung into the chest. - Bronchoscopy : A procedure to look inside the trachea and large airways in the lung for abnormal areas. A bronchoscope is inserted through the nose or mouth into the trachea and lungs. A bronchoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove tissue samples, which are checked under a microscope for signs of cancer. - Thoracoscopy : A surgical procedure to look at the organs inside the chest to check for abnormal areas. An incision (cut) is made between two ribs, and a thoracoscope is inserted into the chest. A thoracoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove tissue or lymph node samples, which are checked under a microscope for signs of cancer. In some cases, this procedure is used to remove part of the esophagus or lung. If certain tissues, organs, or lymph nodes cant be reached, a thoracotomy may be done. In this procedure, a larger incision is made between the ribs and the chest is opened. - Thoracentesis : The removal of fluid from the space between the lining of the chest and the lung, using a needle. A pathologist views the fluid under a microscope to look for cancer cells. - Light and electron microscopy : A laboratory test in which cells in a sample of tissue are viewed under regular and high-powered microscopes to look for certain changes in the cells. - Immunohistochemistry : A test that uses antibodies to check for certain antigens in a sample of tissue. The antibody is usually linked to a radioactive substance or a dye that causes the tissue to light up under a microscope. This type of test may be used to tell the difference between different types of cancer.

The lungs are a pair of cone-shaped breathing organs in the chest. The lungs bring oxygen into the body as you breathe in. They release carbon dioxide, a waste product of the body’s cells, as you breathe out. Each lung has sections called lobes. The left lung has two lobes. The right lung is slightly larger and has three lobes. Two tubes called bronchi lead from the trachea (windpipe) to the right and left lungs. The bronchi are sometimes also involved in lung cancer. Tiny air sacs called alveoli and small tubes called bronchioles make up the inside of the lungs.EnlargeAnatomy of the respiratory system showing the trachea, the right and left lungs and their lobes, and the bronchi. The lymph nodes and the diaphragm are also shown. Oxygen is inhaled into the lungs and passes through the alveoli (the tiny air sacs at the end of the bronchioles) and into the bloodstream (see inset), where it travels to the tissues throughout the body. A thin membrane called the pleura covers the outside of each lung and lines the inside wall of the chest cavity. This creates a sac called the pleural cavity. The pleural cavity normally contains a small amount of fluid that helps the lungs move smoothly in the chest when you breathe. There are two main types of lung cancer: non-small cell lung cancer and small cell lung cancer. See the following PDQ summaries for more information about lung cancer: Each type of non-small cell lung cancer has different kinds of cancer cells. The cancer cells of each type grow and spread in different ways. The types of non-small cell lung cancer are named for the kinds of cells found in the cancer and how the cells look under a microscope: Other less common types of non-small cell lung cancer are: adenosquamous carcinoma, sarcomatoid carcinoma, salivary gland carcinoma, carcinoid tumor, and unclassified carcinoma. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk to your doctor if you think you may be at risk for lung cancer. Risk factors for lung cancer include the following: Older age is the main risk factor for most cancers. The chance of getting cancer increases as you get older. When smoking is combined with other risk factors, the risk of lung cancer is increased. Sometimes lung cancer does not cause any signs or symptoms. It may be found during a chest x-ray done for another condition. Signs and symptoms may be caused by lung cancer or by other conditions. Check with your doctor if you have any of the following: Tests and procedures to diagnose and stage non-small cell lung cancer are often done at the same time. Some of the following tests and procedures may be used: One of the following types of biopsies is usually used: An endoscopic ultrasound (EUS) is a type of ultrasound that may be used to guide an FNA biopsy of the lung, lymph nodes, or other areas. EUS is a procedure in which an endoscope is inserted into the body. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. A probe at the end of the endoscope is used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. One or more of the following laboratory tests may be done to study the tissue samples: The prognosis and treatment options depend on the following: If lung cancer is found, taking part in one of the many clinical trials being done to improve treatment should be considered. Clinical trials are taking place in most parts of the country for patients with all stages of non-small cell lung cancer. Information about ongoing clinical trials is available from the NCI website. The process used to find out if cancer has spread within the lungs or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some of the tests used to diagnose non-small cell lung cancer are also used to stage the disease. For more information, see the General Information section. Other tests and procedures that may be used in the staging process include the following: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if non-small cell lung cancer spreads to the brain, the cancer cells in the brain are actually lung cancer cells. The disease is metastatic lung cancer, not brain cancer. In the occult (hidden) stage, cancer cannot be seen by imaging or bronchoscopy. Cancer cells are found in sputum or bronchial washings (a sample of cells taken from inside the airways that lead to the lungs). Cancer may have spread to other parts of the body. In stage 0, abnormal cells are found in the lining of the airways. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 may be adenocarcinoma in situ (AIS) or squamous cell carcinoma in situ (SCIS). In stage I, cancer has formed. Stage I is divided into stages IA and IB. The tumor is in the lung only and is 3 centimeters or smaller. Cancer has not spread to the lymph nodes. The tumor is larger than 3 centimeters but not larger than 4 centimeters. Cancer has not spread to the lymph nodes. or The tumor is 4 centimeters or smaller and one or more of the following is found: Cancer has not spread to the lymph nodes. Stage II is divided into stages IIA and IIB. The tumor is larger than 4 centimeters but not larger than 5 centimeters. Cancer has not spread to the lymph nodes and one or more of the following may be found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following may be found: or Cancer has not spread to the lymph nodes and one or more of the following is found: Stage III is divided into stages IIIA, IIIB, and IIIC. The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following may be found: or Cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: or Cancer may have spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following may be found: or The tumor may be any size and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following is found: The tumor may be any size and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following is found: Stage IV is divided into stages IVA and IVB. The tumor may be any size and cancer may have spread to the lymph nodes. One or more of the following is found: Cancer has spread to multiple places in one or more organs that are not near the lung. The cancer may come back in the brain, lung, or other parts of the body. Different types of treatments are available for patients with non-small cell lung cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Four types of surgery are used to treat lung cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Stereotactic body radiation therapy is a type of external radiation therapy. Special equipment is used to place the patient in the same position for each radiation treatment. Once a day for several days, a radiation machine aims a larger than usual dose of radiation directly at the tumor. By having the patient in the same position for each treatment, there is less damage to nearby healthy tissue. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. Stereotactic radiosurgery is a type of external radiation therapy used to treat lung cancer that has spread to the brain. A rigid head frame is attached to the skull to keep the head still during the radiation treatment. A machine aims a single large dose of radiation directly at the tumor in the brain. This procedure does not involve surgery. It is also called stereotaxic radiosurgery, radiosurgery, and radiation surgery. For tumors in the airways, radiation is given directly to the tumor through an endoscope. The way the radiation therapy is given depends on the type and stage of the cancer being treated. It also depends on where the cancer is found. External and internal radiation therapy are used to treat non-small cell lung cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Monoclonal antibodies, tyrosine kinase inhibitors, mammalian target of rapamycin (mTOR) inhibitors, and KRAS G12C inhibitors are four types of targeted therapy being used to treat advanced, metastatic, or recurrent non-small cell lung cancer. Monoclonal antibodies Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. There are different types of monoclonal antibody therapy: Tyrosine kinase inhibitors Tyrosine kinase inhibitors are small-molecule drugs that go through the cell membrane and work inside cancer cells to block signals that cancer cells need to grow and divide. Some tyrosine kinase inhibitors also have angiogenesis inhibitor effects. There are different types of tyrosine kinase inhibitors: Mammalian target of rapamycin (mTOR) inhibitors mTOR inhibitors block a protein called mTOR, which may keep cancer cells from growing and prevent the growth of new blood vessels that tumors need to grow. Everolimus is a type of mTOR inhibitor. KRAS G12C inhibitors KRAS G12C inhibitors block a protein called KRAS p.G12C, which may help keep cancer cells from growing and may kill them. Sotorasib and adagrasib are types of KRAS G12C inhibitors. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy used to treat some patients with advanced non-small-cell lung cancer. Types of immune checkpoint inhibitor therapy include: For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Laser therapy is a cancer treatment that uses a laser beam (a narrow beam of intense light) to kill cancer cells. Photodynamic therapy (PDT) is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. Fiberoptic tubes are then used to carry the laser light to the cancer cells, where the drug becomes active and kills the cells. Photodynamic therapy causes little damage to healthy tissue. It is used mainly to treat tumors on or just under the skin or in the lining of internal organs. When the tumor is in the airways, PDT is given directly to the tumor through an endoscope. Cryosurgery is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as carcinoma in situ. This type of treatment is also called cryotherapy. For tumors in the airways, cryosurgery is done through an endoscope. Electrocautery is a treatment that uses a probe or needle heated by an electric current to destroy abnormal tissue. For tumors in the airways, electrocautery is done through an endoscope. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are substances that make tumor cells easier to kill with radiation therapy. The combination of chemotherapy and radiation therapy given with a radiosensitizer is being studied in the treatment of non-small cell lung cancer. New combinations of treatments are being studied in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of occult non-small cell lung cancer depends on the stage of the disease. Occult tumors are often found at an early stage (the tumor is in the lung only) and sometimes can be cured by surgery. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IA non-small cell lung cancer and stage IB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIA non-small cell lung cancer and stage IIB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIA non-small cell lung cancer that can be removed with surgery may include the following: Treatment of stage IIIA non-small cell lung cancer that cannot be removed with surgery may include the following: For more information about supportive care for signs and symptoms including cough, shortness of breath, and chest pain, see Cardiopulmonary Syndromes. Non-small cell lung cancer of the superior sulcus, often called Pancoast tumor, begins in the upper part of the lung and spreads to nearby tissues such as the chest wall, large blood vessels, and spine. Treatment of Pancoast tumors may include the following: Some stage IIIA non-small cell lung tumors that have grown into the chest wall may be completely removed. Treatment of chest wall tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIB non-small cell lung cancer and stage IIIC non-small cell lung cancer may include the following: For more information about supportive care for signs and symptoms such as cough, shortness of breath, and chest pain, see the following PDQ summaries: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about non-small cell lung cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of non-small cell lung cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Non-Small Cell Lung Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lung/patient/non-small-cell-lung-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389355] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. 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What is the outlook for Non-Small Cell Lung Cancer ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options depend on the following: - The stage of the cancer (the size of the tumor and whether it is in the lung only or has spread to other places in the body). - The type of lung cancer. - Whether the cancer has mutations (changes) in certain genes, such as the epidermal growth factor receptor (EGFR) gene or the anaplastic lymphoma kinase (ALK) gene. - Whether there are signs and symptoms such as coughing or trouble breathing. - The patients general health.

The lungs are a pair of cone-shaped breathing organs in the chest. The lungs bring oxygen into the body as you breathe in. They release carbon dioxide, a waste product of the body’s cells, as you breathe out. Each lung has sections called lobes. The left lung has two lobes. The right lung is slightly larger and has three lobes. Two tubes called bronchi lead from the trachea (windpipe) to the right and left lungs. The bronchi are sometimes also involved in lung cancer. Tiny air sacs called alveoli and small tubes called bronchioles make up the inside of the lungs.EnlargeAnatomy of the respiratory system showing the trachea, the right and left lungs and their lobes, and the bronchi. The lymph nodes and the diaphragm are also shown. Oxygen is inhaled into the lungs and passes through the alveoli (the tiny air sacs at the end of the bronchioles) and into the bloodstream (see inset), where it travels to the tissues throughout the body. A thin membrane called the pleura covers the outside of each lung and lines the inside wall of the chest cavity. This creates a sac called the pleural cavity. The pleural cavity normally contains a small amount of fluid that helps the lungs move smoothly in the chest when you breathe. There are two main types of lung cancer: non-small cell lung cancer and small cell lung cancer. See the following PDQ summaries for more information about lung cancer: Each type of non-small cell lung cancer has different kinds of cancer cells. The cancer cells of each type grow and spread in different ways. The types of non-small cell lung cancer are named for the kinds of cells found in the cancer and how the cells look under a microscope: Other less common types of non-small cell lung cancer are: adenosquamous carcinoma, sarcomatoid carcinoma, salivary gland carcinoma, carcinoid tumor, and unclassified carcinoma. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk to your doctor if you think you may be at risk for lung cancer. Risk factors for lung cancer include the following: Older age is the main risk factor for most cancers. The chance of getting cancer increases as you get older. When smoking is combined with other risk factors, the risk of lung cancer is increased. Sometimes lung cancer does not cause any signs or symptoms. It may be found during a chest x-ray done for another condition. Signs and symptoms may be caused by lung cancer or by other conditions. Check with your doctor if you have any of the following: Tests and procedures to diagnose and stage non-small cell lung cancer are often done at the same time. Some of the following tests and procedures may be used: One of the following types of biopsies is usually used: An endoscopic ultrasound (EUS) is a type of ultrasound that may be used to guide an FNA biopsy of the lung, lymph nodes, or other areas. EUS is a procedure in which an endoscope is inserted into the body. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. A probe at the end of the endoscope is used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. One or more of the following laboratory tests may be done to study the tissue samples: The prognosis and treatment options depend on the following: If lung cancer is found, taking part in one of the many clinical trials being done to improve treatment should be considered. Clinical trials are taking place in most parts of the country for patients with all stages of non-small cell lung cancer. Information about ongoing clinical trials is available from the NCI website. The process used to find out if cancer has spread within the lungs or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some of the tests used to diagnose non-small cell lung cancer are also used to stage the disease. For more information, see the General Information section. Other tests and procedures that may be used in the staging process include the following: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if non-small cell lung cancer spreads to the brain, the cancer cells in the brain are actually lung cancer cells. The disease is metastatic lung cancer, not brain cancer. In the occult (hidden) stage, cancer cannot be seen by imaging or bronchoscopy. Cancer cells are found in sputum or bronchial washings (a sample of cells taken from inside the airways that lead to the lungs). Cancer may have spread to other parts of the body. In stage 0, abnormal cells are found in the lining of the airways. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 may be adenocarcinoma in situ (AIS) or squamous cell carcinoma in situ (SCIS). In stage I, cancer has formed. Stage I is divided into stages IA and IB. The tumor is in the lung only and is 3 centimeters or smaller. Cancer has not spread to the lymph nodes. The tumor is larger than 3 centimeters but not larger than 4 centimeters. Cancer has not spread to the lymph nodes. or The tumor is 4 centimeters or smaller and one or more of the following is found: Cancer has not spread to the lymph nodes. Stage II is divided into stages IIA and IIB. The tumor is larger than 4 centimeters but not larger than 5 centimeters. Cancer has not spread to the lymph nodes and one or more of the following may be found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following may be found: or Cancer has not spread to the lymph nodes and one or more of the following is found: Stage III is divided into stages IIIA, IIIB, and IIIC. The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following may be found: or Cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: or Cancer may have spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following may be found: or The tumor may be any size and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following is found: The tumor may be any size and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following is found: Stage IV is divided into stages IVA and IVB. The tumor may be any size and cancer may have spread to the lymph nodes. One or more of the following is found: Cancer has spread to multiple places in one or more organs that are not near the lung. The cancer may come back in the brain, lung, or other parts of the body. Different types of treatments are available for patients with non-small cell lung cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Four types of surgery are used to treat lung cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Stereotactic body radiation therapy is a type of external radiation therapy. Special equipment is used to place the patient in the same position for each radiation treatment. Once a day for several days, a radiation machine aims a larger than usual dose of radiation directly at the tumor. By having the patient in the same position for each treatment, there is less damage to nearby healthy tissue. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. Stereotactic radiosurgery is a type of external radiation therapy used to treat lung cancer that has spread to the brain. A rigid head frame is attached to the skull to keep the head still during the radiation treatment. A machine aims a single large dose of radiation directly at the tumor in the brain. This procedure does not involve surgery. It is also called stereotaxic radiosurgery, radiosurgery, and radiation surgery. For tumors in the airways, radiation is given directly to the tumor through an endoscope. The way the radiation therapy is given depends on the type and stage of the cancer being treated. It also depends on where the cancer is found. External and internal radiation therapy are used to treat non-small cell lung cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Monoclonal antibodies, tyrosine kinase inhibitors, mammalian target of rapamycin (mTOR) inhibitors, and KRAS G12C inhibitors are four types of targeted therapy being used to treat advanced, metastatic, or recurrent non-small cell lung cancer. Monoclonal antibodies Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. There are different types of monoclonal antibody therapy: Tyrosine kinase inhibitors Tyrosine kinase inhibitors are small-molecule drugs that go through the cell membrane and work inside cancer cells to block signals that cancer cells need to grow and divide. Some tyrosine kinase inhibitors also have angiogenesis inhibitor effects. There are different types of tyrosine kinase inhibitors: Mammalian target of rapamycin (mTOR) inhibitors mTOR inhibitors block a protein called mTOR, which may keep cancer cells from growing and prevent the growth of new blood vessels that tumors need to grow. Everolimus is a type of mTOR inhibitor. KRAS G12C inhibitors KRAS G12C inhibitors block a protein called KRAS p.G12C, which may help keep cancer cells from growing and may kill them. Sotorasib and adagrasib are types of KRAS G12C inhibitors. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy used to treat some patients with advanced non-small-cell lung cancer. Types of immune checkpoint inhibitor therapy include: For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Laser therapy is a cancer treatment that uses a laser beam (a narrow beam of intense light) to kill cancer cells. Photodynamic therapy (PDT) is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. Fiberoptic tubes are then used to carry the laser light to the cancer cells, where the drug becomes active and kills the cells. Photodynamic therapy causes little damage to healthy tissue. It is used mainly to treat tumors on or just under the skin or in the lining of internal organs. When the tumor is in the airways, PDT is given directly to the tumor through an endoscope. Cryosurgery is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as carcinoma in situ. This type of treatment is also called cryotherapy. For tumors in the airways, cryosurgery is done through an endoscope. Electrocautery is a treatment that uses a probe or needle heated by an electric current to destroy abnormal tissue. For tumors in the airways, electrocautery is done through an endoscope. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are substances that make tumor cells easier to kill with radiation therapy. The combination of chemotherapy and radiation therapy given with a radiosensitizer is being studied in the treatment of non-small cell lung cancer. New combinations of treatments are being studied in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of occult non-small cell lung cancer depends on the stage of the disease. Occult tumors are often found at an early stage (the tumor is in the lung only) and sometimes can be cured by surgery. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IA non-small cell lung cancer and stage IB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIA non-small cell lung cancer and stage IIB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIA non-small cell lung cancer that can be removed with surgery may include the following: Treatment of stage IIIA non-small cell lung cancer that cannot be removed with surgery may include the following: For more information about supportive care for signs and symptoms including cough, shortness of breath, and chest pain, see Cardiopulmonary Syndromes. Non-small cell lung cancer of the superior sulcus, often called Pancoast tumor, begins in the upper part of the lung and spreads to nearby tissues such as the chest wall, large blood vessels, and spine. Treatment of Pancoast tumors may include the following: Some stage IIIA non-small cell lung tumors that have grown into the chest wall may be completely removed. Treatment of chest wall tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIB non-small cell lung cancer and stage IIIC non-small cell lung cancer may include the following: For more information about supportive care for signs and symptoms such as cough, shortness of breath, and chest pain, see the following PDQ summaries: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about non-small cell lung cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of non-small cell lung cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. 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What are the stages of Non-Small Cell Lung Cancer ?

Key Points - After lung cancer has been diagnosed, tests are done to find out if cancer cells have spread within the lungs or to other parts of the body. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - The following stages are used for non-small cell lung cancer: - Occult (hidden) stage - Stage 0 (carcinoma in situ) - Stage I - Stage II - Stage IIIA - Stage IIIB - Stage IV After lung cancer has been diagnosed, tests are done to find out if cancer cells have spread within the lungs or to other parts of the body. The process used to find out if cancer has spread within the lungs or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some of the tests used to diagnose non-small cell lung cancer are also used to stage the disease. (See the General Information section.) Other tests and procedures that may be used in the staging process include the following: - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body, such as the brain. This procedure is also called nuclear magnetic resonance imaging (NMRI). - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the brain and abdomen, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. - Radionuclide bone scan : A procedure to check if there are rapidly dividing cells, such as cancer cells, in the bone. A very small amount of radioactive material is injected into a vein and travels through the bloodstream. The radioactive material collects in the bones and is detected by a scanner. - Pulmonary function test (PFT): A test to see how well the lungs are working. It measures how much air the lungs can hold and how quickly air moves into and out of the lungs. It also measures how much oxygen is used and how much carbon dioxide is given off during breathing. This is also called lung function test. - Endoscopic ultrasound (EUS): A procedure in which an endoscope is inserted into the body. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. A probe at the end of the endoscope is used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. This procedure is also called endosonography. EUS may be used to guide fine needle aspiration (FNA) biopsy of the lung, lymph nodes, or other areas. - Mediastinoscopy : A surgical procedure to look at the organs, tissues, and lymph nodes between the lungs for abnormal areas. An incision (cut) is made at the top of the breastbone and a mediastinoscope is inserted into the chest. A mediastinoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove tissue or lymph node samples, which are checked under a microscope for signs of cancer. - Anterior mediastinotomy : A surgical procedure to look at the organs and tissues between the lungs and between the breastbone and heart for abnormal areas. An incision (cut) is made next to the breastbone and a mediastinoscope is inserted into the chest. A mediastinoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove tissue or lymph node samples, which are checked under a microscope for signs of cancer. This is also called the Chamberlain procedure. - Lymph node biopsy : The removal of all or part of a lymph node. A pathologist views the tissue under a microscope to look for cancer cells. - Bone marrow aspiration and biopsy : The removal of bone marrow, blood, and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for signs of cancer. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if non-small cell lung cancer spreads to the brain, the cancer cells in the brain are actually lung cancer cells. The disease is metastatic lung cancer, not brain cancer. The following stages are used for non-small cell lung cancer: Occult (hidden) stage In the occult (hidden) stage, cancer cannot be seen by imaging or bronchoscopy. Cancer cells are found in sputum (mucus coughed up from the lungs) or bronchial washing (a sample of cells taken from inside the airways that lead to the lung). Cancer may have spread to other parts of the body. Stage 0 (carcinoma in situ) In stage 0, abnormal cells are found in the lining of the airways. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. Stage I In stage I, cancer has formed. Stage I is divided into stages IA and IB: - Stage IA: The tumor is in the lung only and is 3 centimeters or smaller. - Stage IB: Cancer has not spread to the lymph nodes and one or more of the following is true: - The tumor is larger than 3 centimeters but not larger than 5 centimeters. - Cancer has spread to the main bronchus and is at least 2 centimeters below where the trachea joins the bronchus. - Cancer has spread to the innermost layer of the membrane that covers the lung. - Part of the lung has collapsed or developed pneumonitis (inflammation of the lung) in the area where the trachea joins the bronchus. Stage II Stage II is divided into stages IIA and IIB. Stage IIA and IIB are each divided into two sections depending on the size of the tumor, where the tumor is found, and whether there is cancer in the lymph nodes. - Stage IIA: (1) Cancer has spread to lymph nodes on the same side of the chest as the tumor. The lymph nodes with cancer are within the lung or near the bronchus. Also, one or more of the following is true: - The tumor is not larger than 5 centimeters. - Cancer has spread to the main bronchus and is at least 2 centimeters below where the trachea joins the bronchus. - Cancer has spread to the innermost layer of the membrane that covers the lung. - Part of the lung has collapsed or developed pneumonitis (inflammation of the lung) in the area where the trachea joins the bronchus. or (2) Cancer has not spread to lymph nodes and one or more of the following is true: - The tumor is larger than 5 centimeters but not larger than 7 centimeters. - Cancer has spread to the main bronchus and is at least 2 centimeters below where the trachea joins the bronchus. - Cancer has spread to the innermost layer of the membrane that covers the lung. - Part of the lung has collapsed or developed pneumonitis (inflammation of the lung) in the area where the trachea joins the bronchus. - Stage IIB: (1) Cancer has spread to nearby lymph nodes on the same side of the chest as the tumor. The lymph nodes with cancer are within the lung or near the bronchus. Also, one or more of the following is true: - The tumor is larger than 5 centimeters but not larger than 7 centimeters. - Cancer has spread to the main bronchus and is at least 2 centimeters below where the trachea joins the bronchus. - Cancer has spread to the innermost layer of the membrane that covers the lung. - Part of the lung has collapsed or developed pneumonitis (inflammation of the lung) in the area where the trachea joins the bronchus. or (2) Cancer has not spread to lymph nodes and one or more of the following is true: - The tumor is larger than 7 centimeters. - Cancer has spread to the main bronchus (and is less than 2 centimeters below where the trachea joins the bronchus), the chest wall, the diaphragm, or the nerve that controls the diaphragm. - Cancer has spread to the membrane around the heart or lining the chest wall. - The whole lung has collapsed or developed pneumonitis (inflammation of the lung). - There are one or more separate tumors in the same lobe of the lung. Stage IIIA Stage IIIA is divided into three sections depending on the size of the tumor, where the tumor is found, and which lymph nodes have cancer (if any). (1) Cancer has spread to lymph nodes on the same side of the chest as the tumor. The lymph nodes with cancer are near the sternum (chest bone) or where the bronchus enters the lung. Also: - The tumor may be any size. - Part of the lung (where the trachea joins the bronchus) or the whole lung may have collapsed or developed pneumonitis (inflammation of the lung). - There may be one or more separate tumors in the same lobe of the lung. - Cancer may have spread to any of the following: - Main bronchus, but not the area where the trachea joins the bronchus. - Chest wall. - Diaphragm and the nerve that controls it. - Membrane around the lung or lining the chest wall. - Membrane around the heart. or (2) Cancer has spread to lymph nodes on the same side of the chest as the tumor. The lymph nodes with cancer are within the lung or near the bronchus. Also: - The tumor may be any size. - The whole lung may have collapsed or developed pneumonitis (inflammation of the lung). - There may be one or more separate tumors in any of the lobes of the lung with cancer. - Cancer may have spread to any of the following: - Main bronchus, but not the area where the trachea joins the bronchus. - Chest wall. - Diaphragm and the nerve that controls it. - Membrane around the lung or lining the chest wall. - Heart or the membrane around it. - Major blood vessels that lead to or from the heart. - Trachea. - Esophagus. - Nerve that controls the larynx (voice box). - Sternum (chest bone) or backbone. - Carina (where the trachea joins the bronchi). or (3) Cancer has not spread to the lymph nodes and the tumor may be any size. Cancer has spread to any of the following: - Heart. - Major blood vessels that lead to or from the heart. - Trachea. - Esophagus. - Nerve that controls the larynx (voice box). - Sternum (chest bone) or backbone. - Carina (where the trachea joins the bronchi). Stage IIIB Stage IIIB is divided into two sections depending on the size of the tumor, where the tumor is found, and which lymph nodes have cancer. (1) Cancer has spread to lymph nodes above the collarbone or to lymph nodes on the opposite side of the chest as the tumor. Also: - The tumor may be any size. - Part of the lung (where the trachea joins the bronchus) or the whole lung may have collapsed or developed pneumonitis (inflammation of the lung). - There may be one or more separate tumors in any of the lobes of the lung with cancer. - Cancer may have spread to any of the following: - Main bronchus. - Chest wall. - Diaphragm and the nerve that controls it. - Membrane around the lung or lining the chest wall. - Heart or the membrane around it. - Major blood vessels that lead to or from the heart. - Trachea. - Esophagus. - Nerve that controls the larynx (voice box). - Sternum (chest bone) or backbone. - Carina (where the trachea joins the bronchi). or (2) Cancer has spread to lymph nodes on the same side of the chest as the tumor. The lymph nodes with cancer are near the sternum (chest bone) or where the bronchus enters the lung. Also: - The tumor may be any size. - There may be separate tumors in different lobes of the same lung. - Cancer has spread to any of the following: - Heart. - Major blood vessels that lead to or from the heart. - Trachea. - Esophagus. - Nerve that controls the larynx (voice box). - Sternum (chest bone) or backbone. - Carina (where the trachea joins the bronchi). Stage IV In stage IV, the tumor may be any size and cancer may have spread to lymph nodes. One or more of the following is true: - There are one or more tumors in both lungs. - Cancer is found in fluid around the lungs or the heart. - Cancer has spread to other parts of the body, such as the brain, liver, adrenal glands, kidneys, or bone.

The lungs are a pair of cone-shaped breathing organs in the chest. The lungs bring oxygen into the body as you breathe in. They release carbon dioxide, a waste product of the body’s cells, as you breathe out. Each lung has sections called lobes. The left lung has two lobes. The right lung is slightly larger and has three lobes. Two tubes called bronchi lead from the trachea (windpipe) to the right and left lungs. The bronchi are sometimes also involved in lung cancer. Tiny air sacs called alveoli and small tubes called bronchioles make up the inside of the lungs.EnlargeAnatomy of the respiratory system showing the trachea, the right and left lungs and their lobes, and the bronchi. The lymph nodes and the diaphragm are also shown. Oxygen is inhaled into the lungs and passes through the alveoli (the tiny air sacs at the end of the bronchioles) and into the bloodstream (see inset), where it travels to the tissues throughout the body. A thin membrane called the pleura covers the outside of each lung and lines the inside wall of the chest cavity. This creates a sac called the pleural cavity. The pleural cavity normally contains a small amount of fluid that helps the lungs move smoothly in the chest when you breathe. There are two main types of lung cancer: non-small cell lung cancer and small cell lung cancer. See the following PDQ summaries for more information about lung cancer: Each type of non-small cell lung cancer has different kinds of cancer cells. The cancer cells of each type grow and spread in different ways. The types of non-small cell lung cancer are named for the kinds of cells found in the cancer and how the cells look under a microscope: Other less common types of non-small cell lung cancer are: adenosquamous carcinoma, sarcomatoid carcinoma, salivary gland carcinoma, carcinoid tumor, and unclassified carcinoma. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk to your doctor if you think you may be at risk for lung cancer. Risk factors for lung cancer include the following: Older age is the main risk factor for most cancers. The chance of getting cancer increases as you get older. When smoking is combined with other risk factors, the risk of lung cancer is increased. Sometimes lung cancer does not cause any signs or symptoms. It may be found during a chest x-ray done for another condition. Signs and symptoms may be caused by lung cancer or by other conditions. Check with your doctor if you have any of the following: Tests and procedures to diagnose and stage non-small cell lung cancer are often done at the same time. Some of the following tests and procedures may be used: One of the following types of biopsies is usually used: An endoscopic ultrasound (EUS) is a type of ultrasound that may be used to guide an FNA biopsy of the lung, lymph nodes, or other areas. EUS is a procedure in which an endoscope is inserted into the body. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. A probe at the end of the endoscope is used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. One or more of the following laboratory tests may be done to study the tissue samples: The prognosis and treatment options depend on the following: If lung cancer is found, taking part in one of the many clinical trials being done to improve treatment should be considered. Clinical trials are taking place in most parts of the country for patients with all stages of non-small cell lung cancer. Information about ongoing clinical trials is available from the NCI website. The process used to find out if cancer has spread within the lungs or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some of the tests used to diagnose non-small cell lung cancer are also used to stage the disease. For more information, see the General Information section. Other tests and procedures that may be used in the staging process include the following: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if non-small cell lung cancer spreads to the brain, the cancer cells in the brain are actually lung cancer cells. The disease is metastatic lung cancer, not brain cancer. In the occult (hidden) stage, cancer cannot be seen by imaging or bronchoscopy. Cancer cells are found in sputum or bronchial washings (a sample of cells taken from inside the airways that lead to the lungs). Cancer may have spread to other parts of the body. In stage 0, abnormal cells are found in the lining of the airways. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 may be adenocarcinoma in situ (AIS) or squamous cell carcinoma in situ (SCIS). In stage I, cancer has formed. Stage I is divided into stages IA and IB. The tumor is in the lung only and is 3 centimeters or smaller. Cancer has not spread to the lymph nodes. The tumor is larger than 3 centimeters but not larger than 4 centimeters. Cancer has not spread to the lymph nodes. or The tumor is 4 centimeters or smaller and one or more of the following is found: Cancer has not spread to the lymph nodes. Stage II is divided into stages IIA and IIB. The tumor is larger than 4 centimeters but not larger than 5 centimeters. Cancer has not spread to the lymph nodes and one or more of the following may be found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following may be found: or Cancer has not spread to the lymph nodes and one or more of the following is found: Stage III is divided into stages IIIA, IIIB, and IIIC. The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following may be found: or Cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: or Cancer may have spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following may be found: or The tumor may be any size and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following is found: The tumor may be any size and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following is found: Stage IV is divided into stages IVA and IVB. The tumor may be any size and cancer may have spread to the lymph nodes. One or more of the following is found: Cancer has spread to multiple places in one or more organs that are not near the lung. The cancer may come back in the brain, lung, or other parts of the body. Different types of treatments are available for patients with non-small cell lung cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Four types of surgery are used to treat lung cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Stereotactic body radiation therapy is a type of external radiation therapy. Special equipment is used to place the patient in the same position for each radiation treatment. Once a day for several days, a radiation machine aims a larger than usual dose of radiation directly at the tumor. By having the patient in the same position for each treatment, there is less damage to nearby healthy tissue. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. Stereotactic radiosurgery is a type of external radiation therapy used to treat lung cancer that has spread to the brain. A rigid head frame is attached to the skull to keep the head still during the radiation treatment. A machine aims a single large dose of radiation directly at the tumor in the brain. This procedure does not involve surgery. It is also called stereotaxic radiosurgery, radiosurgery, and radiation surgery. For tumors in the airways, radiation is given directly to the tumor through an endoscope. The way the radiation therapy is given depends on the type and stage of the cancer being treated. It also depends on where the cancer is found. External and internal radiation therapy are used to treat non-small cell lung cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Monoclonal antibodies, tyrosine kinase inhibitors, mammalian target of rapamycin (mTOR) inhibitors, and KRAS G12C inhibitors are four types of targeted therapy being used to treat advanced, metastatic, or recurrent non-small cell lung cancer. Monoclonal antibodies Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. There are different types of monoclonal antibody therapy: Tyrosine kinase inhibitors Tyrosine kinase inhibitors are small-molecule drugs that go through the cell membrane and work inside cancer cells to block signals that cancer cells need to grow and divide. Some tyrosine kinase inhibitors also have angiogenesis inhibitor effects. There are different types of tyrosine kinase inhibitors: Mammalian target of rapamycin (mTOR) inhibitors mTOR inhibitors block a protein called mTOR, which may keep cancer cells from growing and prevent the growth of new blood vessels that tumors need to grow. Everolimus is a type of mTOR inhibitor. KRAS G12C inhibitors KRAS G12C inhibitors block a protein called KRAS p.G12C, which may help keep cancer cells from growing and may kill them. Sotorasib and adagrasib are types of KRAS G12C inhibitors. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy used to treat some patients with advanced non-small-cell lung cancer. Types of immune checkpoint inhibitor therapy include: For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Laser therapy is a cancer treatment that uses a laser beam (a narrow beam of intense light) to kill cancer cells. Photodynamic therapy (PDT) is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. Fiberoptic tubes are then used to carry the laser light to the cancer cells, where the drug becomes active and kills the cells. Photodynamic therapy causes little damage to healthy tissue. It is used mainly to treat tumors on or just under the skin or in the lining of internal organs. When the tumor is in the airways, PDT is given directly to the tumor through an endoscope. Cryosurgery is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as carcinoma in situ. This type of treatment is also called cryotherapy. For tumors in the airways, cryosurgery is done through an endoscope. Electrocautery is a treatment that uses a probe or needle heated by an electric current to destroy abnormal tissue. For tumors in the airways, electrocautery is done through an endoscope. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are substances that make tumor cells easier to kill with radiation therapy. The combination of chemotherapy and radiation therapy given with a radiosensitizer is being studied in the treatment of non-small cell lung cancer. New combinations of treatments are being studied in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of occult non-small cell lung cancer depends on the stage of the disease. Occult tumors are often found at an early stage (the tumor is in the lung only) and sometimes can be cured by surgery. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IA non-small cell lung cancer and stage IB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIA non-small cell lung cancer and stage IIB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIA non-small cell lung cancer that can be removed with surgery may include the following: Treatment of stage IIIA non-small cell lung cancer that cannot be removed with surgery may include the following: For more information about supportive care for signs and symptoms including cough, shortness of breath, and chest pain, see Cardiopulmonary Syndromes. Non-small cell lung cancer of the superior sulcus, often called Pancoast tumor, begins in the upper part of the lung and spreads to nearby tissues such as the chest wall, large blood vessels, and spine. Treatment of Pancoast tumors may include the following: Some stage IIIA non-small cell lung tumors that have grown into the chest wall may be completely removed. Treatment of chest wall tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIB non-small cell lung cancer and stage IIIC non-small cell lung cancer may include the following: For more information about supportive care for signs and symptoms such as cough, shortness of breath, and chest pain, see the following PDQ summaries: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about non-small cell lung cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of non-small cell lung cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. 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What are the treatments for Non-Small Cell Lung Cancer ?

Key Points - There are different types of treatment for patients with non-small cell lung cancer. - Nine types of standard treatment are used: - Surgery - Radiation therapy - Chemotherapy - Targeted therapy - Laser therapy - Photodynamic therapy (PDT) - Cryosurgery - Electrocautery - Watchful waiting - New types of treatment are being tested in clinical trials. - Chemoprevention - Radiosensitizers - New combinations - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with non-small cell lung cancer. Different types of treatments are available for patients with non-small cell lung cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Nine types of standard treatment are used: Surgery Four types of surgery are used to treat lung cancer: - Wedge resection: Surgery to remove a tumor and some of the normal tissue around it. When a slightly larger amount of tissue is taken, it is called a segmental resection. - Lobectomy: Surgery to remove a whole lobe (section) of the lung. - Pneumonectomy: Surgery to remove one whole lung. - Sleeve resection: Surgery to remove part of the bronchus. Even if the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. Stereotactic body radiation therapy is a type of external radiation therapy. Special equipment is used to place the patient in the same position for each radiation treatment. Once a day for several days, a radiation machine aims a larger than usual dose of radiation directly at the tumor. By having the patient in the same position for each treatment, there is less damage to nearby healthy tissue. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. Stereotactic radiosurgery is a type of external radiation therapy used to treat lung cancer that has spread to the brain. A rigid head frame is attached to the skull to keep the head still during the radiation treatment. A machine aims a single large dose of radiation directly at the tumor in the brain. This procedure does not involve surgery. It is also called stereotaxic radiosurgery, radiosurgery, and radiation surgery. For tumors in the airways, radiation is given directly to the tumor through an endoscope. The way the radiation therapy is given depends on the type and stage of the cancer being treated. It also depends on where the cancer is found. External and internal radiation therapy are used to treat non-small cell lung cancer. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Non-Small Cell Lung Cancer for more information. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Monoclonal antibodies and tyrosine kinase inhibitors are the two main types of targeted therapy being used to treat advanced, metastatic, or recurrent non-small cell lung cancer. Monoclonal antibodies Monoclonal antibody therapy is a cancer treatment that uses antibodies made in the laboratory from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances in the blood or tissues that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. There are different types of monoclonal antibody therapy: - Vascular endothelial growth factor (VEGF) inhibitor therapy: Cancer cells make a substance called VEGF, which causes new blood vessels to form (angiogenesis) and helps the cancer grow. VEGF inhibitors block VEGF and stop new blood vessels from forming. This may kill cancer cells because they need new blood vessels to grow. Bevacizumab and ramucirumab are VEGF inhibitors and angiogenesis inhibitors. - Epidermal growth factor receptor (EGFR) inhibitor therapy: EGFRs are proteins found on the surface of certain cells, including cancer cells. Epidermal growth factor attaches to the EGFR on the surface of the cell and causes the cells to grow and divide. EGFR inhibitors block the receptor and stop the epidermal growth factor from attaching to the cancer cell. This stops the cancer cell from growing and dividing. Cetuximab and necitumumab are EGFR inhibitors. - Immune checkpoint inhibitor therapy: PD-1 is a protein on the surface of T cells that helps keep the bodys immune responses in check. When PD-1 attaches to another protein called PDL-1 on a cancer cell, it stops the T cell from killing the cancer cell. PD-1 inhibitors attach to PDL-1 and allow the T cells to kill cancer cells. Nivolumab, pembrolizumab, and atezolizumab are types of immune checkpoint inhibitors. Tyrosine kinase inhibitors Tyrosine kinase inhibitors are small-molecule drugs that go through the cell membrane and work inside cancer cells to block signals that cancer cells need to grow and divide. Some tyrosine kinase inhibitors also have angiogenesis inhibitor effects. There are different types of tyrosine kinase inhibitors: - Epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors: EGFRs are proteins found on the surface and inside certain cells, including cancer cells. Epidermal growth factor attaches to the EGFR inside the cell and sends signals to the tyrosine kinase area of the cell, which tells the cell to grow and divide. EGFR tyrosine kinase inhibitors stop these signals and stop the cancer cell from growing and dividing. Erlotinib, gefitinib, and afatinib are types of EGFR tyrosine kinase inhibitors. Some of these drugs work better when there is also a mutation (change) in the EGFR gene. - Kinase inhibitors that affect cells with certain gene changes: Certain changes in the ALK and ROS1 genes cause too much protein to be made. Blocking these proteins may stop the cancer from growing and spreading. Crizotinib is used to stop proteins from being made by the ALK and ROS1 gene. Ceritinib is used to stop proteins from being made by the ALK gene. See Drugs Approved for Non-Small Cell Lung Cancer for more information. Laser therapy Laser therapy is a cancer treatment that uses a laser beam (a narrow beam of intense light) to kill cancer cells. Photodynamic therapy (PDT) Photodynamic therapy (PDT) is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. Fiberoptic tubes are then used to carry the laser light to the cancer cells, where the drug becomes active and kills the cells. Photodynamic therapy causes little damage to healthy tissue. It is used mainly to treat tumors on or just under the skin or in the lining of internal organs. When the tumor is in the airways, PDT is given directly to the tumor through an endoscope. Cryosurgery Cryosurgery is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as carcinoma in situ. This type of treatment is also called cryotherapy. For tumors in the airways, cryosurgery is done through an endoscope. Electrocautery Electrocautery is a treatment that uses a probe or needle heated by an electric current to destroy abnormal tissue. For tumors in the airways, electrocautery is done through an endoscope. Watchful waiting Watchful waiting is closely monitoring a patients condition without giving any treatment until signs or symptoms appear or change. This may be done in certain rare cases of non-small cell lung cancer. New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Chemoprevention Chemoprevention is the use of drugs, vitamins, or other substances to reduce the risk of cancer or to reduce the risk cancer will recur (come back). For lung cancer, chemoprevention is used to lessen the chance that a new tumor will form in the lung. Radiosensitizers Radiosensitizers are substances that make tumor cells easier to kill with radiation therapy. The combination of chemotherapy and radiation therapy given with a radiosensitizer is being studied in the treatment of non-small cell lung cancer. New combinations New combinations of treatments are being studied in clinical trials. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options by Stage Occult Non-Small Cell Lung Cancer Treatment of occult non-small cell lung cancer depends on the stage of the disease. Occult tumors are often found at an early stage (the tumor is in the lung only) and sometimes can be cured by surgery. Check the list of NCI-supported cancer clinical trials that are now accepting patients with occult non-small cell lung cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage 0 (Carcinoma in Situ) Treatment of stage 0 may include the following: - Surgery (wedge resection or segmental resection). - Photodynamic therapy for tumors in or near the bronchus. - Electrocautery, cryosurgery, or laser surgery for tumors in or near the bronchus. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage 0 non-small cell lung cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage I Non-Small Cell Lung Cancer Treatment of stage I non-small cell lung cancer may include the following: - Surgery (wedge resection, segmental resection, sleeve resection, or lobectomy). - External radiation therapy, including stereotactic body radiation therapy for patients who cannot have surgery or choose not to have surgery. - A clinical trial of chemotherapy or radiation therapy following surgery. - A clinical trial of treatment given through an endoscope, such as photodynamic therapy (PDT). - A clinical trial of surgery followed by chemoprevention. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I non-small cell lung cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage II Non-Small Cell Lung Cancer Treatment of stage II non-small cell lung cancer may include the following: - Surgery (wedge resection, segmental resection, sleeve resection, lobectomy, or pneumonectomy). - Chemotherapy followed by surgery. - Surgery followed by chemotherapy. - External radiation therapy for patients who cannot have surgery. - A clinical trial of radiation therapy following surgery. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage II non-small cell lung cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IIIA Non-Small Cell Lung Cancer Treatment of stage IIIA non-small cell lung cancer that can be removed with surgery may include the following: - Surgery followed by chemotherapy. - Surgery followed by radiation therapy. - Chemotherapy followed by surgery. - Surgery followed by chemotherapy combined with radiation therapy. - Chemotherapy and radiation therapy followed by surgery. - A clinical trial of new combinations of treatments. Treatment of stage IIIA non-small cell lung cancer that cannot be removed with surgery may include the following: - Chemotherapy and radiation therapy given over the same period of time or one followed by the other. - External radiation therapy alone for patients who cannot be treated with combined therapy, or as palliative treatment to relieve symptoms and improve the quality of life. - Internal radiation therapy or laser surgery, as palliative treatment to relieve symptoms and improve the quality of life. - A clinical trial of new combinations of treatments. For more information about supportive care for signs and symptoms including cough, shortness of breath, and chest pain, see the PDQ summary on Cardiopulmonary Syndromes. Non-small cell lung cancer of the superior sulcus, often called Pancoast tumor, begins in the upper part of the lung and spreads to nearby tissues such as the chest wall, large blood vessels, and spine. Treatment of Pancoast tumors may include the following: - Radiation therapy alone. - Radiation therapy followed by surgery. - Chemotherapy and radiation therapy given as separate treatments over the same period of time. Surgery may also be done after chemotherapy and radiation therapy. - Surgery alone. - A clinical trial of new combinations of treatments. Some stage IIIA non-small cell lung tumors that have grown into the chest wall may be completely removed. Treatment of chest wall tumors may include the following: - Surgery. - Surgery and radiation therapy. - Radiation therapy alone. - Chemotherapy combined with radiation therapy and/or surgery. - A clinical trial of new combinations of treatments. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage III non-small cell lung cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IIIB Non-Small Cell Lung Cancer Treatment of stage IIIB non-small cell lung cancer may include the following: - Chemotherapy followed by external radiation therapy. - Chemotherapy and radiation therapy given as separate treatments over the same period of time. - Chemotherapy followed by surgery. - External radiation therapy alone for patients who cannot be treated with chemotherapy. - External radiation therapy as palliative therapy, to relieve symptoms and improve the quality of life. - Laser therapy and/or internal radiation therapy to relieve symptoms and improve the quality of life. - Clinical trials of new external radiation therapy schedules and new types of treatment. - A clinical trial of chemotherapy and radiation therapy combined with a radiosensitizer. - Clinical trials of targeted therapy combined with chemotherapy and radiation therapy. For more information about supportive care for signs and symptoms such as cough, shortness of breath, and chest pain, see the following PDQ summaries: - Cardiopulmonary Syndromes - Cancer Pain Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage III non-small cell lung cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IV Non-Small Cell Lung Cancer Treatment of stage IV non-small cell lung cancer may include the following: - Chemotherapy. - Chemotherapy followed by more chemotherapy as maintenance therapy to help keep cancer from progressing. - Combination chemotherapy and targeted therapy with a monoclonal antibody, such as bevacizumab, cetuximab, or necitumumab. - Targeted therapy with a monoclonal antibody, such as nivolumab, pembrolizumab, or atezolizumab. - Targeted therapy with a tyrosine kinase inhibitor, such as erlotinib, gefitinib, afatinib, crizotinib, or ceritinib. - External radiation therapy as palliative therapy, to relieve symptoms and improve the quality of life. - Laser therapy and/or internal radiation therapy for tumors that are blocking the airways. - A clinical trial of new drugs and combinations of treatments. For more information about supportive care for signs and symptoms including cough, shortness of breath, and chest pain, see the following PDQ summaries: - Cardiopulmonary Syndromes - Cancer Pain - Last Days of Life Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage IV non-small cell lung cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

The lungs are a pair of cone-shaped breathing organs in the chest. The lungs bring oxygen into the body as you breathe in. They release carbon dioxide, a waste product of the body’s cells, as you breathe out. Each lung has sections called lobes. The left lung has two lobes. The right lung is slightly larger and has three lobes. Two tubes called bronchi lead from the trachea (windpipe) to the right and left lungs. The bronchi are sometimes also involved in lung cancer. Tiny air sacs called alveoli and small tubes called bronchioles make up the inside of the lungs.EnlargeAnatomy of the respiratory system showing the trachea, the right and left lungs and their lobes, and the bronchi. The lymph nodes and the diaphragm are also shown. Oxygen is inhaled into the lungs and passes through the alveoli (the tiny air sacs at the end of the bronchioles) and into the bloodstream (see inset), where it travels to the tissues throughout the body. A thin membrane called the pleura covers the outside of each lung and lines the inside wall of the chest cavity. This creates a sac called the pleural cavity. The pleural cavity normally contains a small amount of fluid that helps the lungs move smoothly in the chest when you breathe. There are two main types of lung cancer: non-small cell lung cancer and small cell lung cancer. See the following PDQ summaries for more information about lung cancer: Each type of non-small cell lung cancer has different kinds of cancer cells. The cancer cells of each type grow and spread in different ways. The types of non-small cell lung cancer are named for the kinds of cells found in the cancer and how the cells look under a microscope: Other less common types of non-small cell lung cancer are: adenosquamous carcinoma, sarcomatoid carcinoma, salivary gland carcinoma, carcinoid tumor, and unclassified carcinoma. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk to your doctor if you think you may be at risk for lung cancer. Risk factors for lung cancer include the following: Older age is the main risk factor for most cancers. The chance of getting cancer increases as you get older. When smoking is combined with other risk factors, the risk of lung cancer is increased. Sometimes lung cancer does not cause any signs or symptoms. It may be found during a chest x-ray done for another condition. Signs and symptoms may be caused by lung cancer or by other conditions. Check with your doctor if you have any of the following: Tests and procedures to diagnose and stage non-small cell lung cancer are often done at the same time. Some of the following tests and procedures may be used: One of the following types of biopsies is usually used: An endoscopic ultrasound (EUS) is a type of ultrasound that may be used to guide an FNA biopsy of the lung, lymph nodes, or other areas. EUS is a procedure in which an endoscope is inserted into the body. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. A probe at the end of the endoscope is used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. One or more of the following laboratory tests may be done to study the tissue samples: The prognosis and treatment options depend on the following: If lung cancer is found, taking part in one of the many clinical trials being done to improve treatment should be considered. Clinical trials are taking place in most parts of the country for patients with all stages of non-small cell lung cancer. Information about ongoing clinical trials is available from the NCI website. The process used to find out if cancer has spread within the lungs or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. Some of the tests used to diagnose non-small cell lung cancer are also used to stage the disease. For more information, see the General Information section. Other tests and procedures that may be used in the staging process include the following: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if non-small cell lung cancer spreads to the brain, the cancer cells in the brain are actually lung cancer cells. The disease is metastatic lung cancer, not brain cancer. In the occult (hidden) stage, cancer cannot be seen by imaging or bronchoscopy. Cancer cells are found in sputum or bronchial washings (a sample of cells taken from inside the airways that lead to the lungs). Cancer may have spread to other parts of the body. In stage 0, abnormal cells are found in the lining of the airways. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 may be adenocarcinoma in situ (AIS) or squamous cell carcinoma in situ (SCIS). In stage I, cancer has formed. Stage I is divided into stages IA and IB. The tumor is in the lung only and is 3 centimeters or smaller. Cancer has not spread to the lymph nodes. The tumor is larger than 3 centimeters but not larger than 4 centimeters. Cancer has not spread to the lymph nodes. or The tumor is 4 centimeters or smaller and one or more of the following is found: Cancer has not spread to the lymph nodes. Stage II is divided into stages IIA and IIB. The tumor is larger than 4 centimeters but not larger than 5 centimeters. Cancer has not spread to the lymph nodes and one or more of the following may be found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following may be found: or Cancer has not spread to the lymph nodes and one or more of the following is found: Stage III is divided into stages IIIA, IIIB, and IIIC. The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following may be found: or Cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: or Cancer may have spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are in the lung or near the bronchus. Also, one or more of the following is found: The tumor is 5 centimeters or smaller and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following may be found: or The tumor may be any size and cancer has spread to lymph nodes on the same side of the chest as the primary tumor. The lymph nodes with cancer are around the trachea or aorta, or where the trachea divides into the bronchi. Also, one or more of the following is found: The tumor may be any size and cancer has spread to lymph nodes above the collarbone on the same side of the chest as the primary tumor or to any lymph nodes on the opposite side of the chest as the primary tumor. Also, one or more of the following is found: Stage IV is divided into stages IVA and IVB. The tumor may be any size and cancer may have spread to the lymph nodes. One or more of the following is found: Cancer has spread to multiple places in one or more organs that are not near the lung. The cancer may come back in the brain, lung, or other parts of the body. Different types of treatments are available for patients with non-small cell lung cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Four types of surgery are used to treat lung cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: Stereotactic body radiation therapy is a type of external radiation therapy. Special equipment is used to place the patient in the same position for each radiation treatment. Once a day for several days, a radiation machine aims a larger than usual dose of radiation directly at the tumor. By having the patient in the same position for each treatment, there is less damage to nearby healthy tissue. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. Stereotactic radiosurgery is a type of external radiation therapy used to treat lung cancer that has spread to the brain. A rigid head frame is attached to the skull to keep the head still during the radiation treatment. A machine aims a single large dose of radiation directly at the tumor in the brain. This procedure does not involve surgery. It is also called stereotaxic radiosurgery, radiosurgery, and radiation surgery. For tumors in the airways, radiation is given directly to the tumor through an endoscope. The way the radiation therapy is given depends on the type and stage of the cancer being treated. It also depends on where the cancer is found. External and internal radiation therapy are used to treat non-small cell lung cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Monoclonal antibodies, tyrosine kinase inhibitors, mammalian target of rapamycin (mTOR) inhibitors, and KRAS G12C inhibitors are four types of targeted therapy being used to treat advanced, metastatic, or recurrent non-small cell lung cancer. Monoclonal antibodies Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. There are different types of monoclonal antibody therapy: Tyrosine kinase inhibitors Tyrosine kinase inhibitors are small-molecule drugs that go through the cell membrane and work inside cancer cells to block signals that cancer cells need to grow and divide. Some tyrosine kinase inhibitors also have angiogenesis inhibitor effects. There are different types of tyrosine kinase inhibitors: Mammalian target of rapamycin (mTOR) inhibitors mTOR inhibitors block a protein called mTOR, which may keep cancer cells from growing and prevent the growth of new blood vessels that tumors need to grow. Everolimus is a type of mTOR inhibitor. KRAS G12C inhibitors KRAS G12C inhibitors block a protein called KRAS p.G12C, which may help keep cancer cells from growing and may kill them. Sotorasib and adagrasib are types of KRAS G12C inhibitors. For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy used to treat some patients with advanced non-small-cell lung cancer. Types of immune checkpoint inhibitor therapy include: For more information, see Drugs Approved for Non-Small Cell Lung Cancer. Laser therapy is a cancer treatment that uses a laser beam (a narrow beam of intense light) to kill cancer cells. Photodynamic therapy (PDT) is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. Fiberoptic tubes are then used to carry the laser light to the cancer cells, where the drug becomes active and kills the cells. Photodynamic therapy causes little damage to healthy tissue. It is used mainly to treat tumors on or just under the skin or in the lining of internal organs. When the tumor is in the airways, PDT is given directly to the tumor through an endoscope. Cryosurgery is a treatment that uses an instrument to freeze and destroy abnormal tissue, such as carcinoma in situ. This type of treatment is also called cryotherapy. For tumors in the airways, cryosurgery is done through an endoscope. Electrocautery is a treatment that uses a probe or needle heated by an electric current to destroy abnormal tissue. For tumors in the airways, electrocautery is done through an endoscope. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are substances that make tumor cells easier to kill with radiation therapy. The combination of chemotherapy and radiation therapy given with a radiosensitizer is being studied in the treatment of non-small cell lung cancer. New combinations of treatments are being studied in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of occult non-small cell lung cancer depends on the stage of the disease. Occult tumors are often found at an early stage (the tumor is in the lung only) and sometimes can be cured by surgery. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IA non-small cell lung cancer and stage IB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIA non-small cell lung cancer and stage IIB non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIA non-small cell lung cancer that can be removed with surgery may include the following: Treatment of stage IIIA non-small cell lung cancer that cannot be removed with surgery may include the following: For more information about supportive care for signs and symptoms including cough, shortness of breath, and chest pain, see Cardiopulmonary Syndromes. Non-small cell lung cancer of the superior sulcus, often called Pancoast tumor, begins in the upper part of the lung and spreads to nearby tissues such as the chest wall, large blood vessels, and spine. Treatment of Pancoast tumors may include the following: Some stage IIIA non-small cell lung tumors that have grown into the chest wall may be completely removed. Treatment of chest wall tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IIIB non-small cell lung cancer and stage IIIC non-small cell lung cancer may include the following: For more information about supportive care for signs and symptoms such as cough, shortness of breath, and chest pain, see the following PDQ summaries: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive stage IV, relapsed, and recurrent non-small cell lung cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about non-small cell lung cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of non-small cell lung cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Non-Small Cell Lung Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lung/patient/non-small-cell-lung-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389355] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. 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what research (or clinical trials) is being done for Non-Small Cell Lung Cancer ?

New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Chemoprevention Chemoprevention is the use of drugs, vitamins, or other substances to reduce the risk of cancer or to reduce the risk cancer will recur (come back). For lung cancer, chemoprevention is used to lessen the chance that a new tumor will form in the lung. Radiosensitizers Radiosensitizers are substances that make tumor cells easier to kill with radiation therapy. The combination of chemotherapy and radiation therapy given with a radiosensitizer is being studied in the treatment of non-small cell lung cancer. New combinations New combinations of treatments are being studied in clinical trials. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Myelodysplastic/myeloproliferative neoplasms are diseases of the blood and bone marrow. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myelodysplastic diseases, the blood stem cells do not mature into healthy red blood cells, white blood cells, or platelets. The immature blood cells, called blasts, do not work the way they should and die in the bone marrow or soon after they enter the blood. As a result, there are fewer healthy red blood cells, white blood cells, and platelets. In myeloproliferative diseases, a greater than normal number of blood stem cells become one or more types of blood cells and the total number of blood cells slowly increases. This summary is about neoplasms that have features of both myelodysplastic and myeloproliferative diseases. See the following PDQ summaries for more information about related diseases: The 3 main types of myelodysplastic/myeloproliferative neoplasms include the following: When a myelodysplastic/myeloproliferative neoplasm does not match any of these types, it is called myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC). Myelodysplastic/myeloproliferative neoplasms may progress to acute leukemia. The following tests and procedures may be used: The following tests may be done on the sample of tissue that is removed: In chronic myelomonocytic leukemia (CMML), the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. Anything that increases your chance of getting a disease is called a risk factor. Possible risk factors for CMML include the following: These and other signs and symptoms may be caused by CMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for CMML depend on the following: Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children younger than 2 years. Children who have neurofibromatosis type 1 and males have an increased risk of juvenile myelomonocytic leukemia. In JMML, the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. These and other signs and symptoms may be caused by JMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for JMML depend on the following: In atypical chronic myelogenous leukemia (CML), the body tells too many blood stem cells to become a type of white blood cell called granulocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. The leukemia cells in atypical CML and CML look alike under a microscope. However, in atypical CML a certain chromosome change, called the "Philadelphia chromosome" is not there. These and other signs and symptoms may be caused by atypical CML or by other conditions. Check with your doctor if you have any of the following: The prognosis for atypical CML depends on the number of red blood cells and platelets in the blood. In myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPD-UC), the body tells too many blood stem cells to become red blood cells, white blood cells, or platelets. Some of these blood stem cells never become mature blood cells. These immature blood cells are called blasts. Over time, the abnormal blood cells and blasts in the bone marrow crowd out the healthy red blood cells, white blood cells, and platelets. MDS/MPN-UC is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known. These and other signs and symptoms may be caused by MDS/MPN-UC or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread is called staging. There is no standard staging system for myelodysplastic/myeloproliferative neoplasms. It is important to know the type of myelodysplastic/myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with myelodysplastic/myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. See Drugs Approved for Myeloproliferative Neoplasms for more information. 13-cis retinoic acid is a vitamin-like drug that slows the cancer's ability to make more cancer cells and changes the way these cells look and act. Chemotherapy is given to kill abnormal cells or cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include transfusion therapy or drug therapy, such as antibiotics to fight infection. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Myeloproliferative Neoplasms for more information. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic myelomonocytic leukemia (CMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of juvenile myelomonocytic leukemia (JMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of atypical chronic myelogenous leukemia (CML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Because myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) is a rare disease, little is known about its treatment. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about myelodysplastic/myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of myelodysplastic/ myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic/ Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/mds-mpd-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389360] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Myelodysplastic/ Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Atypical Chronic Myelogenous Leukemia ?

Key Points - Atypical chronic myelogenous leukemia is a disease in which too many granulocytes (immature white blood cells) are made in the bone marrow. - Signs and symptoms of atypical chronic myelogenous leukemia include easy bruising or bleeding and feeling tired and weak. - Certain factors affect prognosis (chance of recovery). Atypical chronic myelogenous leukemia is a disease in which too many granulocytes (immature white blood cells) are made in the bone marrow. In atypical chronic myelogenous leukemia (CML), the body tells too many blood stem cells to become a type of white blood cell called granulocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. The leukemia cells in atypical CML and CML look alike under a microscope. However, in atypical CML a certain chromosome change, called the &quot;Philadelphia chromosome&quot; is not there.

Myelodysplastic/myeloproliferative neoplasms are diseases of the blood and bone marrow. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myelodysplastic diseases, the blood stem cells do not mature into healthy red blood cells, white blood cells, or platelets. The immature blood cells, called blasts, do not work the way they should and die in the bone marrow or soon after they enter the blood. As a result, there are fewer healthy red blood cells, white blood cells, and platelets. In myeloproliferative diseases, a greater than normal number of blood stem cells become one or more types of blood cells and the total number of blood cells slowly increases. This summary is about neoplasms that have features of both myelodysplastic and myeloproliferative diseases. See the following PDQ summaries for more information about related diseases: The 3 main types of myelodysplastic/myeloproliferative neoplasms include the following: When a myelodysplastic/myeloproliferative neoplasm does not match any of these types, it is called myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC). Myelodysplastic/myeloproliferative neoplasms may progress to acute leukemia. The following tests and procedures may be used: The following tests may be done on the sample of tissue that is removed: In chronic myelomonocytic leukemia (CMML), the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. Anything that increases your chance of getting a disease is called a risk factor. Possible risk factors for CMML include the following: These and other signs and symptoms may be caused by CMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for CMML depend on the following: Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children younger than 2 years. Children who have neurofibromatosis type 1 and males have an increased risk of juvenile myelomonocytic leukemia. In JMML, the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. These and other signs and symptoms may be caused by JMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for JMML depend on the following: In atypical chronic myelogenous leukemia (CML), the body tells too many blood stem cells to become a type of white blood cell called granulocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. The leukemia cells in atypical CML and CML look alike under a microscope. However, in atypical CML a certain chromosome change, called the "Philadelphia chromosome" is not there. These and other signs and symptoms may be caused by atypical CML or by other conditions. Check with your doctor if you have any of the following: The prognosis for atypical CML depends on the number of red blood cells and platelets in the blood. In myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPD-UC), the body tells too many blood stem cells to become red blood cells, white blood cells, or platelets. Some of these blood stem cells never become mature blood cells. These immature blood cells are called blasts. Over time, the abnormal blood cells and blasts in the bone marrow crowd out the healthy red blood cells, white blood cells, and platelets. MDS/MPN-UC is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known. These and other signs and symptoms may be caused by MDS/MPN-UC or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread is called staging. There is no standard staging system for myelodysplastic/myeloproliferative neoplasms. It is important to know the type of myelodysplastic/myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with myelodysplastic/myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. See Drugs Approved for Myeloproliferative Neoplasms for more information. 13-cis retinoic acid is a vitamin-like drug that slows the cancer's ability to make more cancer cells and changes the way these cells look and act. Chemotherapy is given to kill abnormal cells or cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include transfusion therapy or drug therapy, such as antibiotics to fight infection. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Myeloproliferative Neoplasms for more information. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic myelomonocytic leukemia (CMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of juvenile myelomonocytic leukemia (JMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of atypical chronic myelogenous leukemia (CML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Because myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) is a rare disease, little is known about its treatment. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about myelodysplastic/myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of myelodysplastic/ myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic/ Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/mds-mpd-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389360] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Myelodysplastic/ Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Atypical Chronic Myelogenous Leukemia ?

Signs and symptoms of atypical chronic myelogenous leukemia include easy bruising or bleeding and feeling tired and weak. These and other signs and symptoms may be caused by atypical CML or by other conditions. Check with your doctor if you have any of the following: - Shortness of breath. - Pale skin. - Feeling very tired and weak. - Easy bruising or bleeding. - Petechiae (flat, pinpoint spots under the skin caused by bleeding). - Pain or a feeling of fullness below the ribs on the left side.

Myelodysplastic/myeloproliferative neoplasms are diseases of the blood and bone marrow. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myelodysplastic diseases, the blood stem cells do not mature into healthy red blood cells, white blood cells, or platelets. The immature blood cells, called blasts, do not work the way they should and die in the bone marrow or soon after they enter the blood. As a result, there are fewer healthy red blood cells, white blood cells, and platelets. In myeloproliferative diseases, a greater than normal number of blood stem cells become one or more types of blood cells and the total number of blood cells slowly increases. This summary is about neoplasms that have features of both myelodysplastic and myeloproliferative diseases. See the following PDQ summaries for more information about related diseases: The 3 main types of myelodysplastic/myeloproliferative neoplasms include the following: When a myelodysplastic/myeloproliferative neoplasm does not match any of these types, it is called myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC). Myelodysplastic/myeloproliferative neoplasms may progress to acute leukemia. The following tests and procedures may be used: The following tests may be done on the sample of tissue that is removed: In chronic myelomonocytic leukemia (CMML), the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. Anything that increases your chance of getting a disease is called a risk factor. Possible risk factors for CMML include the following: These and other signs and symptoms may be caused by CMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for CMML depend on the following: Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children younger than 2 years. Children who have neurofibromatosis type 1 and males have an increased risk of juvenile myelomonocytic leukemia. In JMML, the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. These and other signs and symptoms may be caused by JMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for JMML depend on the following: In atypical chronic myelogenous leukemia (CML), the body tells too many blood stem cells to become a type of white blood cell called granulocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. The leukemia cells in atypical CML and CML look alike under a microscope. However, in atypical CML a certain chromosome change, called the "Philadelphia chromosome" is not there. These and other signs and symptoms may be caused by atypical CML or by other conditions. Check with your doctor if you have any of the following: The prognosis for atypical CML depends on the number of red blood cells and platelets in the blood. In myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPD-UC), the body tells too many blood stem cells to become red blood cells, white blood cells, or platelets. Some of these blood stem cells never become mature blood cells. These immature blood cells are called blasts. Over time, the abnormal blood cells and blasts in the bone marrow crowd out the healthy red blood cells, white blood cells, and platelets. MDS/MPN-UC is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known. These and other signs and symptoms may be caused by MDS/MPN-UC or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread is called staging. There is no standard staging system for myelodysplastic/myeloproliferative neoplasms. It is important to know the type of myelodysplastic/myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with myelodysplastic/myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. See Drugs Approved for Myeloproliferative Neoplasms for more information. 13-cis retinoic acid is a vitamin-like drug that slows the cancer's ability to make more cancer cells and changes the way these cells look and act. Chemotherapy is given to kill abnormal cells or cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include transfusion therapy or drug therapy, such as antibiotics to fight infection. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Myeloproliferative Neoplasms for more information. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic myelomonocytic leukemia (CMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of juvenile myelomonocytic leukemia (JMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of atypical chronic myelogenous leukemia (CML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Because myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) is a rare disease, little is known about its treatment. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about myelodysplastic/myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of myelodysplastic/ myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic/ Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/mds-mpd-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389360] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Myelodysplastic/ Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Atypical Chronic Myelogenous Leukemia ?

Certain factors affect prognosis (chance of recovery). The prognosis (chance of recovery) for atypical CML depends on the number of red blood cells and platelets in the blood.

Myelodysplastic/myeloproliferative neoplasms are diseases of the blood and bone marrow. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myelodysplastic diseases, the blood stem cells do not mature into healthy red blood cells, white blood cells, or platelets. The immature blood cells, called blasts, do not work the way they should and die in the bone marrow or soon after they enter the blood. As a result, there are fewer healthy red blood cells, white blood cells, and platelets. In myeloproliferative diseases, a greater than normal number of blood stem cells become one or more types of blood cells and the total number of blood cells slowly increases. This summary is about neoplasms that have features of both myelodysplastic and myeloproliferative diseases. See the following PDQ summaries for more information about related diseases: The 3 main types of myelodysplastic/myeloproliferative neoplasms include the following: When a myelodysplastic/myeloproliferative neoplasm does not match any of these types, it is called myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC). Myelodysplastic/myeloproliferative neoplasms may progress to acute leukemia. The following tests and procedures may be used: The following tests may be done on the sample of tissue that is removed: In chronic myelomonocytic leukemia (CMML), the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. Anything that increases your chance of getting a disease is called a risk factor. Possible risk factors for CMML include the following: These and other signs and symptoms may be caused by CMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for CMML depend on the following: Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children younger than 2 years. Children who have neurofibromatosis type 1 and males have an increased risk of juvenile myelomonocytic leukemia. In JMML, the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. These and other signs and symptoms may be caused by JMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for JMML depend on the following: In atypical chronic myelogenous leukemia (CML), the body tells too many blood stem cells to become a type of white blood cell called granulocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. The leukemia cells in atypical CML and CML look alike under a microscope. However, in atypical CML a certain chromosome change, called the "Philadelphia chromosome" is not there. These and other signs and symptoms may be caused by atypical CML or by other conditions. Check with your doctor if you have any of the following: The prognosis for atypical CML depends on the number of red blood cells and platelets in the blood. In myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPD-UC), the body tells too many blood stem cells to become red blood cells, white blood cells, or platelets. Some of these blood stem cells never become mature blood cells. These immature blood cells are called blasts. Over time, the abnormal blood cells and blasts in the bone marrow crowd out the healthy red blood cells, white blood cells, and platelets. MDS/MPN-UC is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known. These and other signs and symptoms may be caused by MDS/MPN-UC or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread is called staging. There is no standard staging system for myelodysplastic/myeloproliferative neoplasms. It is important to know the type of myelodysplastic/myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with myelodysplastic/myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. See Drugs Approved for Myeloproliferative Neoplasms for more information. 13-cis retinoic acid is a vitamin-like drug that slows the cancer's ability to make more cancer cells and changes the way these cells look and act. Chemotherapy is given to kill abnormal cells or cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include transfusion therapy or drug therapy, such as antibiotics to fight infection. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Myeloproliferative Neoplasms for more information. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic myelomonocytic leukemia (CMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of juvenile myelomonocytic leukemia (JMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of atypical chronic myelogenous leukemia (CML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Because myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) is a rare disease, little is known about its treatment. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about myelodysplastic/myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of myelodysplastic/ myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic/ Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/mds-mpd-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389360] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Myelodysplastic/ Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Atypical Chronic Myelogenous Leukemia ?

Treatment of atypical chronic myelogenous leukemia (CML) may include chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with atypical chronic myeloid leukemia, BCR-ABL1 negative. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

The anus is the end of the large intestine, below the rectum, through which stool (solid waste) leaves the body. The anus is formed partly from the outer skin layers of the body and partly from the intestine. Two ring-like muscles, called sphincter muscles, open and close the anal opening and let stool pass out of the body. The anal canal, the part of the anus between the rectum and the anal opening, is about 1-1½ inches long. EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. The skin around the outside of the anus is called the perianal area. Tumors of the perianal skin that do not involve the anal sphincter are usually treated the same as anal cancers, although local therapy (treatment directed to a limited area of skin) may be used for some. Risk factors for anal cancer include the following: These and other signs and symptoms may be caused by anal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis depends on the following: The treatment options depend on the following: The process used to find out if cancer has spread within the anus or to other parts of the body is called staging. The information gathered from this staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if anal cancer spreads to the lung, the cancer cells in the lung are actually anal cancer cells. The disease is metastatic anal cancer, not lung cancer. In stage 0, abnormal cells are found in the mucosa (innermost layer) of the anus. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called high-grade intraepithelial lesion (HSIL). In stage I, cancer has formed and the tumor is 2 centimeters or smaller. Stage II anal cancer is divided into stages IIA and IIB. Stage III anal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor is any size. Cancer may have spread to lymph nodes or nearby organs and has spread to other parts of the body, such as the liver or lungs. The cancer may come back in the anus or other parts of the body, such as the liver or lungs. Different types of treatments are available for patients with anal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat anal cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 is usually local resection. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I, stage II, and stage III anal cancer may include the following: Patients who have had treatment that saves the sphincter muscles may receive follow-up exams every 3 months for the first 2 years, including rectal exams with endoscopy and biopsy, as needed to check for recurrence. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. In general, treatment for patients who have anal cancer and the human immunodeficiency virus (HIV) is similar to treatment for other patients, and these patients have similar outcomes. However, this treatment can further damage the weakened immune systems of patients who have HIV. Treatment in patients with a history of AIDS-related complications may require lower doses of anticancer drugs and radiation therapy than doses used for patients who do not have HIV. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about anal cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of anal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Anal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/anal/patient/anal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389368] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Anal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Anal Cancer ?

Key Points - Anal cancer is a disease in which malignant (cancer) cells form in the tissues of the anus. - Squamous cell carcinoma is the most common type of anal cancer. - In the United States, the number of new cases of anal cancer has increased in recent years. Anal cancer is a disease in which malignant (cancer) cells form in the tissues of the anus. The anus is the end of the large intestine, below the rectum, through which stool (solid waste) leaves the body. The anus is formed partly from the outer skin layers of the body and partly from the intestine. Two ring-like muscles, called sphincter muscles, open and close the anal opening and let stool pass out of the body. The anal canal, the part of the anus between the rectum and the anal opening, is about 1-1 inches long. The skin around the outside of the anus is called the perianal area. Tumors in this area are skin tumors, not anal cancer. See the following PDQ summary for more information about anal cancer: - Anal Cancer Treatment Squamous cell carcinoma is the most common type of anal cancer. In the United States, the most common type of anal cancer is squamous cell carcinoma. Studies show that human papillomavirus (HPV) infection is the main cause of this type of anal cancer. Another type of anal cancer, called anal adenocarcinoma, is very rare and is not discussed in this summary. In the United States, the number of new cases of anal cancer has increased in recent years. From 2004 to 2013, new cases of anal cancer and deaths from anal cancer increased each year. The increase in new cases was slightly higher in women and the increase in deaths from anal cancer was slightly higher in men.

The anus is the end of the large intestine, below the rectum, through which stool (solid waste) leaves the body. The anus is formed partly from the outer skin layers of the body and partly from the intestine. Two ring-like muscles, called sphincter muscles, open and close the anal opening and let stool pass out of the body. The anal canal, the part of the anus between the rectum and the anal opening, is about 1-1½ inches long. EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. The skin around the outside of the anus is called the perianal area. Tumors of the perianal skin that do not involve the anal sphincter are usually treated the same as anal cancers, although local therapy (treatment directed to a limited area of skin) may be used for some. Risk factors for anal cancer include the following: These and other signs and symptoms may be caused by anal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis depends on the following: The treatment options depend on the following: The process used to find out if cancer has spread within the anus or to other parts of the body is called staging. The information gathered from this staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if anal cancer spreads to the lung, the cancer cells in the lung are actually anal cancer cells. The disease is metastatic anal cancer, not lung cancer. In stage 0, abnormal cells are found in the mucosa (innermost layer) of the anus. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called high-grade intraepithelial lesion (HSIL). In stage I, cancer has formed and the tumor is 2 centimeters or smaller. Stage II anal cancer is divided into stages IIA and IIB. Stage III anal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor is any size. Cancer may have spread to lymph nodes or nearby organs and has spread to other parts of the body, such as the liver or lungs. The cancer may come back in the anus or other parts of the body, such as the liver or lungs. Different types of treatments are available for patients with anal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat anal cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 is usually local resection. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I, stage II, and stage III anal cancer may include the following: Patients who have had treatment that saves the sphincter muscles may receive follow-up exams every 3 months for the first 2 years, including rectal exams with endoscopy and biopsy, as needed to check for recurrence. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. In general, treatment for patients who have anal cancer and the human immunodeficiency virus (HIV) is similar to treatment for other patients, and these patients have similar outcomes. However, this treatment can further damage the weakened immune systems of patients who have HIV. Treatment in patients with a history of AIDS-related complications may require lower doses of anticancer drugs and radiation therapy than doses used for patients who do not have HIV. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about anal cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of anal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Anal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/anal/patient/anal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389368] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Anal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Anal Cancer? ?

Being infected with the human papillomavirus (HPV) increases the risk of developing anal cancer. Risk factors include the following: - Being infected with human papillomavirus (HPV). - Having many sexual partners. - Having receptive anal intercourse (anal sex). - Being older than 50 years. - Frequent anal redness, swelling, and soreness. - Having anal fistulas (abnormal openings). - Smoking cigarettes.

The anus is the end of the large intestine, below the rectum, through which stool (solid waste) leaves the body. The anus is formed partly from the outer skin layers of the body and partly from the intestine. Two ring-like muscles, called sphincter muscles, open and close the anal opening and let stool pass out of the body. The anal canal, the part of the anus between the rectum and the anal opening, is about 1-1½ inches long. EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. The skin around the outside of the anus is called the perianal area. Tumors of the perianal skin that do not involve the anal sphincter are usually treated the same as anal cancers, although local therapy (treatment directed to a limited area of skin) may be used for some. Risk factors for anal cancer include the following: These and other signs and symptoms may be caused by anal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis depends on the following: The treatment options depend on the following: The process used to find out if cancer has spread within the anus or to other parts of the body is called staging. The information gathered from this staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if anal cancer spreads to the lung, the cancer cells in the lung are actually anal cancer cells. The disease is metastatic anal cancer, not lung cancer. In stage 0, abnormal cells are found in the mucosa (innermost layer) of the anus. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called high-grade intraepithelial lesion (HSIL). In stage I, cancer has formed and the tumor is 2 centimeters or smaller. Stage II anal cancer is divided into stages IIA and IIB. Stage III anal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor is any size. Cancer may have spread to lymph nodes or nearby organs and has spread to other parts of the body, such as the liver or lungs. The cancer may come back in the anus or other parts of the body, such as the liver or lungs. Different types of treatments are available for patients with anal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat anal cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 is usually local resection. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I, stage II, and stage III anal cancer may include the following: Patients who have had treatment that saves the sphincter muscles may receive follow-up exams every 3 months for the first 2 years, including rectal exams with endoscopy and biopsy, as needed to check for recurrence. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. In general, treatment for patients who have anal cancer and the human immunodeficiency virus (HIV) is similar to treatment for other patients, and these patients have similar outcomes. However, this treatment can further damage the weakened immune systems of patients who have HIV. Treatment in patients with a history of AIDS-related complications may require lower doses of anticancer drugs and radiation therapy than doses used for patients who do not have HIV. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about anal cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of anal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Anal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/anal/patient/anal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389368] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Anal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Anal Cancer ?

Signs of anal cancer include bleeding from the anus or rectum or a lump near the anus. These and other signs and symptoms may be caused by anal cancer or by other conditions. Check with your doctor if you have any of the following: - Bleeding from the anus or rectum. - Pain or pressure in the area around the anus. - Itching or discharge from the anus. - A lump near the anus. - A change in bowel habits.

The anus is the end of the large intestine, below the rectum, through which stool (solid waste) leaves the body. The anus is formed partly from the outer skin layers of the body and partly from the intestine. Two ring-like muscles, called sphincter muscles, open and close the anal opening and let stool pass out of the body. The anal canal, the part of the anus between the rectum and the anal opening, is about 1-1½ inches long. EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. The skin around the outside of the anus is called the perianal area. Tumors of the perianal skin that do not involve the anal sphincter are usually treated the same as anal cancers, although local therapy (treatment directed to a limited area of skin) may be used for some. Risk factors for anal cancer include the following: These and other signs and symptoms may be caused by anal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis depends on the following: The treatment options depend on the following: The process used to find out if cancer has spread within the anus or to other parts of the body is called staging. The information gathered from this staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if anal cancer spreads to the lung, the cancer cells in the lung are actually anal cancer cells. The disease is metastatic anal cancer, not lung cancer. In stage 0, abnormal cells are found in the mucosa (innermost layer) of the anus. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called high-grade intraepithelial lesion (HSIL). In stage I, cancer has formed and the tumor is 2 centimeters or smaller. Stage II anal cancer is divided into stages IIA and IIB. Stage III anal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor is any size. Cancer may have spread to lymph nodes or nearby organs and has spread to other parts of the body, such as the liver or lungs. The cancer may come back in the anus or other parts of the body, such as the liver or lungs. Different types of treatments are available for patients with anal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat anal cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 is usually local resection. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I, stage II, and stage III anal cancer may include the following: Patients who have had treatment that saves the sphincter muscles may receive follow-up exams every 3 months for the first 2 years, including rectal exams with endoscopy and biopsy, as needed to check for recurrence. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. In general, treatment for patients who have anal cancer and the human immunodeficiency virus (HIV) is similar to treatment for other patients, and these patients have similar outcomes. However, this treatment can further damage the weakened immune systems of patients who have HIV. Treatment in patients with a history of AIDS-related complications may require lower doses of anticancer drugs and radiation therapy than doses used for patients who do not have HIV. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about anal cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of anal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Anal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/anal/patient/anal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389368] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Anal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Anal Cancer ?

Tests that examine the rectum and anus are used to detect (find) and diagnose anal cancer. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Digital rectal examination (DRE): An exam of the anus and rectum. The doctor or nurse inserts a lubricated, gloved finger into the lower part of the rectum to feel for lumps or anything else that seems unusual. - Anoscopy: An exam of the anus and lower rectum using a short, lighted tube called an anoscope. - Proctoscopy : An exam of the rectum using a short, lighted tube called a proctoscope. - Endo-anal or endorectal ultrasound : A procedure in which an ultrasound transducer (probe) is inserted into the anus or rectum and used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. - Biopsy : The removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for signs of cancer. If an abnormal area is seen during the anoscopy, a biopsy may be done at that time.

The anus is the end of the large intestine, below the rectum, through which stool (solid waste) leaves the body. The anus is formed partly from the outer skin layers of the body and partly from the intestine. Two ring-like muscles, called sphincter muscles, open and close the anal opening and let stool pass out of the body. The anal canal, the part of the anus between the rectum and the anal opening, is about 1-1½ inches long. EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. The skin around the outside of the anus is called the perianal area. Tumors of the perianal skin that do not involve the anal sphincter are usually treated the same as anal cancers, although local therapy (treatment directed to a limited area of skin) may be used for some. Risk factors for anal cancer include the following: These and other signs and symptoms may be caused by anal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis depends on the following: The treatment options depend on the following: The process used to find out if cancer has spread within the anus or to other parts of the body is called staging. The information gathered from this staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if anal cancer spreads to the lung, the cancer cells in the lung are actually anal cancer cells. The disease is metastatic anal cancer, not lung cancer. In stage 0, abnormal cells are found in the mucosa (innermost layer) of the anus. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called high-grade intraepithelial lesion (HSIL). In stage I, cancer has formed and the tumor is 2 centimeters or smaller. Stage II anal cancer is divided into stages IIA and IIB. Stage III anal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor is any size. Cancer may have spread to lymph nodes or nearby organs and has spread to other parts of the body, such as the liver or lungs. The cancer may come back in the anus or other parts of the body, such as the liver or lungs. Different types of treatments are available for patients with anal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat anal cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 is usually local resection. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I, stage II, and stage III anal cancer may include the following: Patients who have had treatment that saves the sphincter muscles may receive follow-up exams every 3 months for the first 2 years, including rectal exams with endoscopy and biopsy, as needed to check for recurrence. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. In general, treatment for patients who have anal cancer and the human immunodeficiency virus (HIV) is similar to treatment for other patients, and these patients have similar outcomes. However, this treatment can further damage the weakened immune systems of patients who have HIV. Treatment in patients with a history of AIDS-related complications may require lower doses of anticancer drugs and radiation therapy than doses used for patients who do not have HIV. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about anal cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of anal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Anal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/anal/patient/anal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389368] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Anal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Anal Cancer ?

Certain factors affect the prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) depends on the following: - The size of the tumor. - Where the tumor is in the anus. - Whether the cancer has spread to the lymph nodes. The treatment options depend on the following: - The stage of the cancer. - Where the tumor is in the anus. - Whether the patient has human immunodeficiency virus (HIV). - Whether cancer remains after initial treatment or has recurred.

The anus is the end of the large intestine, below the rectum, through which stool (solid waste) leaves the body. The anus is formed partly from the outer skin layers of the body and partly from the intestine. Two ring-like muscles, called sphincter muscles, open and close the anal opening and let stool pass out of the body. The anal canal, the part of the anus between the rectum and the anal opening, is about 1-1½ inches long. EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. The skin around the outside of the anus is called the perianal area. Tumors of the perianal skin that do not involve the anal sphincter are usually treated the same as anal cancers, although local therapy (treatment directed to a limited area of skin) may be used for some. Risk factors for anal cancer include the following: These and other signs and symptoms may be caused by anal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis depends on the following: The treatment options depend on the following: The process used to find out if cancer has spread within the anus or to other parts of the body is called staging. The information gathered from this staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if anal cancer spreads to the lung, the cancer cells in the lung are actually anal cancer cells. The disease is metastatic anal cancer, not lung cancer. In stage 0, abnormal cells are found in the mucosa (innermost layer) of the anus. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called high-grade intraepithelial lesion (HSIL). In stage I, cancer has formed and the tumor is 2 centimeters or smaller. Stage II anal cancer is divided into stages IIA and IIB. Stage III anal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor is any size. Cancer may have spread to lymph nodes or nearby organs and has spread to other parts of the body, such as the liver or lungs. The cancer may come back in the anus or other parts of the body, such as the liver or lungs. Different types of treatments are available for patients with anal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat anal cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 is usually local resection. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I, stage II, and stage III anal cancer may include the following: Patients who have had treatment that saves the sphincter muscles may receive follow-up exams every 3 months for the first 2 years, including rectal exams with endoscopy and biopsy, as needed to check for recurrence. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. In general, treatment for patients who have anal cancer and the human immunodeficiency virus (HIV) is similar to treatment for other patients, and these patients have similar outcomes. However, this treatment can further damage the weakened immune systems of patients who have HIV. Treatment in patients with a history of AIDS-related complications may require lower doses of anticancer drugs and radiation therapy than doses used for patients who do not have HIV. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about anal cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of anal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Anal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/anal/patient/anal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389368] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Anal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Anal Cancer ?

Key Points - After anal cancer has been diagnosed, tests are done to find out if cancer cells have spread within the anus or to other parts of the body. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - The following stages are used for anal cancer: - Stage 0 (Carcinoma in Situ) - Stage I - Stage II - Stage IIIA - Stage IIIB - Stage IV After anal cancer has been diagnosed, tests are done to find out if cancer cells have spread within the anus or to other parts of the body. The process used to find out if cancer has spread within the anus or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests may be used in the staging process: - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the abdomen or chest, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. For anal cancer, a CT scan of the pelvis and abdomen may be done. - Chest x-ray : An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). - PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if anal cancer spreads to the lung, the cancer cells in the lung are actually anal cancer cells. The disease is metastatic anal cancer, not lung cancer. The following stages are used for anal cancer: Stage 0 (Carcinoma in Situ) In stage 0, abnormal cells are found in the innermost lining of the anus. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. Stage I In stage I, cancer has formed and the tumor is 2 centimeters or smaller. Stage II In stage II, the tumor is larger than 2 centimeters. Stage IIIA In stage IIIA, the tumor may be any size and has spread to either: - lymph nodes near the rectum; or - nearby organs, such as the vagina, urethra, and bladder. Stage IIIB In stage IIIB, the tumor may be any size and has spread: - to nearby organs and to lymph nodes near the rectum; or - to lymph nodes on one side of the pelvis and/or groin, and may have spread to nearby organs; or - to lymph nodes near the rectum and in the groin, and/or to lymph nodes on both sides of the pelvis and/or groin, and may have spread to nearby organs. Stage IV In stage IV, the tumor may be any size and cancer may have spread to lymph nodes or nearby organs and has spread to distant parts of the body.

The anus is the end of the large intestine, below the rectum, through which stool (solid waste) leaves the body. The anus is formed partly from the outer skin layers of the body and partly from the intestine. Two ring-like muscles, called sphincter muscles, open and close the anal opening and let stool pass out of the body. The anal canal, the part of the anus between the rectum and the anal opening, is about 1-1½ inches long. EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. The skin around the outside of the anus is called the perianal area. Tumors of the perianal skin that do not involve the anal sphincter are usually treated the same as anal cancers, although local therapy (treatment directed to a limited area of skin) may be used for some. Risk factors for anal cancer include the following: These and other signs and symptoms may be caused by anal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis depends on the following: The treatment options depend on the following: The process used to find out if cancer has spread within the anus or to other parts of the body is called staging. The information gathered from this staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if anal cancer spreads to the lung, the cancer cells in the lung are actually anal cancer cells. The disease is metastatic anal cancer, not lung cancer. In stage 0, abnormal cells are found in the mucosa (innermost layer) of the anus. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called high-grade intraepithelial lesion (HSIL). In stage I, cancer has formed and the tumor is 2 centimeters or smaller. Stage II anal cancer is divided into stages IIA and IIB. Stage III anal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor is any size. Cancer may have spread to lymph nodes or nearby organs and has spread to other parts of the body, such as the liver or lungs. The cancer may come back in the anus or other parts of the body, such as the liver or lungs. Different types of treatments are available for patients with anal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat anal cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 is usually local resection. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I, stage II, and stage III anal cancer may include the following: Patients who have had treatment that saves the sphincter muscles may receive follow-up exams every 3 months for the first 2 years, including rectal exams with endoscopy and biopsy, as needed to check for recurrence. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. In general, treatment for patients who have anal cancer and the human immunodeficiency virus (HIV) is similar to treatment for other patients, and these patients have similar outcomes. However, this treatment can further damage the weakened immune systems of patients who have HIV. Treatment in patients with a history of AIDS-related complications may require lower doses of anticancer drugs and radiation therapy than doses used for patients who do not have HIV. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about anal cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of anal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Anal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/anal/patient/anal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389368] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Anal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Anal Cancer ?

New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

The anus is the end of the large intestine, below the rectum, through which stool (solid waste) leaves the body. The anus is formed partly from the outer skin layers of the body and partly from the intestine. Two ring-like muscles, called sphincter muscles, open and close the anal opening and let stool pass out of the body. The anal canal, the part of the anus between the rectum and the anal opening, is about 1-1½ inches long. EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. The skin around the outside of the anus is called the perianal area. Tumors of the perianal skin that do not involve the anal sphincter are usually treated the same as anal cancers, although local therapy (treatment directed to a limited area of skin) may be used for some. Risk factors for anal cancer include the following: These and other signs and symptoms may be caused by anal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis depends on the following: The treatment options depend on the following: The process used to find out if cancer has spread within the anus or to other parts of the body is called staging. The information gathered from this staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if anal cancer spreads to the lung, the cancer cells in the lung are actually anal cancer cells. The disease is metastatic anal cancer, not lung cancer. In stage 0, abnormal cells are found in the mucosa (innermost layer) of the anus. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called high-grade intraepithelial lesion (HSIL). In stage I, cancer has formed and the tumor is 2 centimeters or smaller. Stage II anal cancer is divided into stages IIA and IIB. Stage III anal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor is any size. Cancer may have spread to lymph nodes or nearby organs and has spread to other parts of the body, such as the liver or lungs. The cancer may come back in the anus or other parts of the body, such as the liver or lungs. Different types of treatments are available for patients with anal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat anal cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 is usually local resection. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I, stage II, and stage III anal cancer may include the following: Patients who have had treatment that saves the sphincter muscles may receive follow-up exams every 3 months for the first 2 years, including rectal exams with endoscopy and biopsy, as needed to check for recurrence. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. In general, treatment for patients who have anal cancer and the human immunodeficiency virus (HIV) is similar to treatment for other patients, and these patients have similar outcomes. However, this treatment can further damage the weakened immune systems of patients who have HIV. Treatment in patients with a history of AIDS-related complications may require lower doses of anticancer drugs and radiation therapy than doses used for patients who do not have HIV. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent anal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about anal cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of anal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Anal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/anal/patient/anal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389368] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Anal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Anal Cancer ?

Key Points - There are different types of treatment for patients with anal cancer. - Three types of standard treatment are used: - Radiation therapy - Chemotherapy - Surgery - Having the human immunodeficiency virus can affect treatment of anal cancer. - New types of treatment are being tested in clinical trials. - Radiosensitizers - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with anal cancer. Different types of treatments are available for patients with anal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Three types of standard treatment are used: Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat anal cancer. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. Surgery - Local resection: A surgical procedure in which the tumor is cut from the anus along with some of the healthy tissue around it. Local resection may be used if the cancer is small and has not spread. This procedure may save the sphincter muscles so the patient can still control bowel movements. Tumors that form in the lower part of the anus can often be removed with local resection. - Abdominoperineal resection: A surgical procedure in which the anus, the rectum, and part of the sigmoid colon are removed through an incision made in the abdomen. The doctor sews the end of the intestine to an opening, called a stoma, made in the surface of the abdomen so body waste can be collected in a disposable bag outside of the body. This is called a colostomy. Lymph nodes that contain cancer may also be removed during this operation. Having the human immunodeficiency virus can affect treatment of anal cancer. Cancer therapy can further damage the already weakened immune systems of patients who have the human immunodeficiency virus (HIV). For this reason, patients who have anal cancer and HIV are usually treated with lower doses of anticancer drugs and radiation than patients who do not have HIV. New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Radiosensitizers Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options by Stage Stage 0 (Carcinoma in Situ) Treatment of stage 0 is usually local resection. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage 0 anal cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage I Anal Cancer Treatment of stage I anal cancer may include the following: - Local resection. - External-beam radiation therapy with or without chemotherapy. If cancer remains after treatment, more chemotherapy and radiation therapy may be given to avoid the need for a permanent colostomy. - Internal radiation therapy. - Abdominoperineal resection, if cancer remains or comes back after treatment with radiation therapy and chemotherapy. - Internal radiation therapy for cancer that remains after treatment with external-beam radiation therapy. Patients who have had treatment that saves the sphincter muscles may receive follow-up exams every 3 months for the first 2 years, including rectal exams with endoscopy and biopsy, as needed. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I anal cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage II Anal Cancer Treatment of stage II anal cancer may include the following: - Local resection. - External-beam radiation therapy with chemotherapy. If cancer remains after treatment, more chemotherapy and radiation therapy may be given to avoid the need for a permanent colostomy. - Internal radiation therapy. - Abdominoperineal resection, if cancer remains or comes back after treatment with radiation therapy and chemotherapy. - A clinical trial of new treatment options. Patients who have had treatment that saves the sphincter muscles may receive follow-up exams every 3 months for the first 2 years, including rectal exams with endoscopy and biopsy, as needed. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage II anal cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IIIA Anal Cancer Treatment of stage IIIA anal cancer may include the following: - External-beam radiation therapy with chemotherapy. If cancer remains after treatment, more chemotherapy and radiation therapy may be given to avoid the need for a permanent colostomy. - Internal radiation therapy. - Abdominoperineal resection, if cancer remains or comes back after treatment with chemotherapy and radiation therapy. - A clinical trial of new treatment options. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage IIIA anal cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IIIB Anal Cancer Treatment of stage IIIB anal cancer may include the following: - External-beam radiation therapy with chemotherapy. - Local resection or abdominoperineal resection, if cancer remains or comes back after treatment with chemotherapy and radiation therapy. Lymph nodes may also be removed. - A clinical trial of new treatment options. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage IIIB anal cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IV Anal Cancer Treatment of stage IV anal cancer may include the following: - Surgery as palliative therapy to relieve symptoms and improve the quality of life. - Radiation therapy as palliative therapy. - Chemotherapy with radiation therapy as palliative therapy. - A clinical trial of new treatment options. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage IV anal cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

Intraocular melanoma begins in the middle of three layers of the wall of the eye. The outer layer includes the white sclera (the "white of the eye") and the clear cornea at the front of the eye. The inner layer has a lining of nerve tissue, called the retina, which senses light and sends images along the optic nerve to the brain. The middle layer, where intraocular melanoma forms, is called the uvea or uveal tract, and has three main parts: The iris is the colored area at the front of the eye (the "eye color"). It can be seen through the clear cornea. The pupil is in the center of the iris and it changes size to let more or less light into the eye. Intraocular melanoma of the iris is usually a small tumor that grows slowly and rarely spreads to other parts of the body. The ciliary body is a ring of tissue with muscle fibers that change the shape of the lens. It is found behind the iris. Changes in the shape of the lens help the eye focus. The ciliary body also makes the clear fluid that fills the space between the cornea and the iris. Intraocular melanoma of the ciliary body is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. The choroid is a layer of blood vessels that bring oxygen and nutrients to the eye. Most intraocular melanomas begin in the choroid. Intraocular melanoma of the choroid is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. Intraocular melanoma is a rare cancer that forms from cells that make melanin in the iris, ciliary body, and choroid. It is the most common eye cancer in adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for intraocular melanoma include the following: Intraocular melanoma may not cause early signs or symptoms. It is sometimes found during a regular eye exam when the doctor dilates the pupil and looks into the eye. Signs and symptoms may be caused by intraocular melanoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: A biopsy is the removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. Rarely, a biopsy of the tumor is needed to diagnose intraocular melanoma. Tissue that is removed during a biopsy or surgery to remove the tumor may be tested to get more information about prognosis and which treatment options are best. The following tests may be done on the sample of tissue: A biopsy may result in retinal detachment (the retina separates from other tissues in the eye). This can be repaired by surgery. The prognosis and treatment options depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The tumor is 5 to 16 millimeters in diameter and from 1 to 3 millimeters thick. EnlargeMillimeters (mm). A sharp pencil point is about 1 mm, a new crayon point is about 2 mm, and a new pencil eraser is about 5 mm. The tumor is 16 millimeters or smaller in diameter and from 3.1 to 8 millimeters thick. The tumor is: Though most intraocular melanoma tumors are raised, some are flat. These diffuse tumors grow widely across the uvea. Cancer can spread through tissue, the lymph system, and the blood: If intraocular melanoma spreads to the optic nerve or nearby tissue of the eye socket, it is called extraocular extension. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if intraocular melanoma spreads to the liver, the cancer cells in the liver are actually intraocular melanoma cells. The disease is metastatic intraocular melanoma, not liver cancer. Intraocular melanoma of the ciliary body and choroid has four size categories. The category depends on how wide and thick the tumor is. Category 1 tumors are the smallest and category 4 tumors are the largest. Category 1: Category 2: Category 3: Category 4: In stage I, the tumor is size category 1 and is in the choroid only. Stage II is divided into stages IIA and IIB. Stage III is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor may be any size and has spread: The melanoma may come back in the eye or in other parts of the body. Different types of treatments are available for patients with intraocular melanoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for intraocular melanoma. The following types of surgery may be used: Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Pictures are taken over time to keep track of changes in the size of the tumor and how fast it is growing. Watchful waiting is used for patients who do not have signs or symptoms and the tumor is not growing. It is also used when the tumor is in the only eye with useful vision. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat intraocular melanoma. Photocoagulation is a procedure that uses laser light to destroy blood vessels that bring nutrients to the tumor, causing the tumor cells to die. Photocoagulation may be used to treat small tumors. This is also called light coagulation. Thermotherapy is the use of heat from a laser to destroy cancer cells and shrink the tumor. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of iris melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of ciliary body melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of small choroid melanoma may include the following: Treatment of medium choroid melanoma may include the following: Treatment of large choroid melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of extraocular extension melanoma that has spread to the bone around the eye may include the following: An effective treatment for metastatic intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. An effective treatment for recurrent intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about intraocular (uveal) melanoma, see the Intraocular (Eye) Melanoma Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of intraocular melanoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Intraocular (Uveal) Melanoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/eye/patient/intraocular-melanoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389277] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. 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What is (are) Intraocular (Uveal) Melanoma ?

Key Points - Intraocular melanoma is a disease in which malignant (cancer) cells form in the tissues of the eye. - Being older and having fair skin may increase the risk of intraocular melanoma. - Signs of intraocular melanoma include blurred vision or a dark spot on the iris. - Tests that examine the eye are used to help detect (find) and diagnose intraocular melanoma. - A biopsy of the tumor is rarely needed to diagnose intraocular melanoma. - Certain factors affect prognosis (chance of recovery) and treatment options. Intraocular melanoma is a disease in which malignant (cancer) cells form in the tissues of the eye. Intraocular melanoma begins in the middle of three layers of the wall of the eye. The outer layer includes the white sclera (the &quot;white of the eye&quot;) and the clear cornea at the front of the eye. The inner layer has a lining of nerve tissue, called the retina, which senses light and sends images along the optic nerve to the brain. The middle layer, where intraocular melanoma forms, is called the uvea or uveal tract, and has three main parts: - Iris The iris is the colored area at the front of the eye (the &quot;eye color&quot;). It can be seen through the clear cornea. The pupil is in the center of the iris and it changes size to let more or less light into the eye. Intraocular melanoma of the iris is usually a small tumor that grows slowly and rarely spreads to other parts of the body. - Ciliary body The ciliary body is a ring of tissue with muscle fibers that change the size of the pupil and the shape of the lens. It is found behind the iris. Changes in the shape of the lens help the eye focus. The ciliary body also makes the clear fluid that fills the space between the cornea and the iris. Intraocular melanoma of the ciliary body is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. - Choroid The choroid is a layer of blood vessels that bring oxygen and nutrients to the eye. Most intraocular melanomas begin in the choroid. Intraocular melanoma of the choroid is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. Intraocular melanoma is a rare cancer that forms from cells that make melanin in the iris, ciliary body, and choroid. It is the most common eye cancer in adults.

Intraocular melanoma begins in the middle of three layers of the wall of the eye. The outer layer includes the white sclera (the "white of the eye") and the clear cornea at the front of the eye. The inner layer has a lining of nerve tissue, called the retina, which senses light and sends images along the optic nerve to the brain. The middle layer, where intraocular melanoma forms, is called the uvea or uveal tract, and has three main parts: The iris is the colored area at the front of the eye (the "eye color"). It can be seen through the clear cornea. The pupil is in the center of the iris and it changes size to let more or less light into the eye. Intraocular melanoma of the iris is usually a small tumor that grows slowly and rarely spreads to other parts of the body. The ciliary body is a ring of tissue with muscle fibers that change the shape of the lens. It is found behind the iris. Changes in the shape of the lens help the eye focus. The ciliary body also makes the clear fluid that fills the space between the cornea and the iris. Intraocular melanoma of the ciliary body is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. The choroid is a layer of blood vessels that bring oxygen and nutrients to the eye. Most intraocular melanomas begin in the choroid. Intraocular melanoma of the choroid is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. Intraocular melanoma is a rare cancer that forms from cells that make melanin in the iris, ciliary body, and choroid. It is the most common eye cancer in adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for intraocular melanoma include the following: Intraocular melanoma may not cause early signs or symptoms. It is sometimes found during a regular eye exam when the doctor dilates the pupil and looks into the eye. Signs and symptoms may be caused by intraocular melanoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: A biopsy is the removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. Rarely, a biopsy of the tumor is needed to diagnose intraocular melanoma. Tissue that is removed during a biopsy or surgery to remove the tumor may be tested to get more information about prognosis and which treatment options are best. The following tests may be done on the sample of tissue: A biopsy may result in retinal detachment (the retina separates from other tissues in the eye). This can be repaired by surgery. The prognosis and treatment options depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The tumor is 5 to 16 millimeters in diameter and from 1 to 3 millimeters thick. EnlargeMillimeters (mm). A sharp pencil point is about 1 mm, a new crayon point is about 2 mm, and a new pencil eraser is about 5 mm. The tumor is 16 millimeters or smaller in diameter and from 3.1 to 8 millimeters thick. The tumor is: Though most intraocular melanoma tumors are raised, some are flat. These diffuse tumors grow widely across the uvea. Cancer can spread through tissue, the lymph system, and the blood: If intraocular melanoma spreads to the optic nerve or nearby tissue of the eye socket, it is called extraocular extension. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if intraocular melanoma spreads to the liver, the cancer cells in the liver are actually intraocular melanoma cells. The disease is metastatic intraocular melanoma, not liver cancer. Intraocular melanoma of the ciliary body and choroid has four size categories. The category depends on how wide and thick the tumor is. Category 1 tumors are the smallest and category 4 tumors are the largest. Category 1: Category 2: Category 3: Category 4: In stage I, the tumor is size category 1 and is in the choroid only. Stage II is divided into stages IIA and IIB. Stage III is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor may be any size and has spread: The melanoma may come back in the eye or in other parts of the body. Different types of treatments are available for patients with intraocular melanoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for intraocular melanoma. The following types of surgery may be used: Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Pictures are taken over time to keep track of changes in the size of the tumor and how fast it is growing. Watchful waiting is used for patients who do not have signs or symptoms and the tumor is not growing. It is also used when the tumor is in the only eye with useful vision. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat intraocular melanoma. Photocoagulation is a procedure that uses laser light to destroy blood vessels that bring nutrients to the tumor, causing the tumor cells to die. Photocoagulation may be used to treat small tumors. This is also called light coagulation. Thermotherapy is the use of heat from a laser to destroy cancer cells and shrink the tumor. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of iris melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of ciliary body melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of small choroid melanoma may include the following: Treatment of medium choroid melanoma may include the following: Treatment of large choroid melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of extraocular extension melanoma that has spread to the bone around the eye may include the following: An effective treatment for metastatic intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. An effective treatment for recurrent intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about intraocular (uveal) melanoma, see the Intraocular (Eye) Melanoma Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of intraocular melanoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Intraocular (Uveal) Melanoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/eye/patient/intraocular-melanoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389277] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Intraocular (Uveal) Melanoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Intraocular (Uveal) Melanoma? ?

Being older and having fair skin may increase the risk of intraocular melanoma. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesnt mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for intraocular melanoma include the following: - Having a fair complexion, which includes the following: - Fair skin that freckles and burns easily, does not tan, or tans poorly. - Blue or green or other light-colored eyes. - Older age. - Being white.

Intraocular melanoma begins in the middle of three layers of the wall of the eye. The outer layer includes the white sclera (the "white of the eye") and the clear cornea at the front of the eye. The inner layer has a lining of nerve tissue, called the retina, which senses light and sends images along the optic nerve to the brain. The middle layer, where intraocular melanoma forms, is called the uvea or uveal tract, and has three main parts: The iris is the colored area at the front of the eye (the "eye color"). It can be seen through the clear cornea. The pupil is in the center of the iris and it changes size to let more or less light into the eye. Intraocular melanoma of the iris is usually a small tumor that grows slowly and rarely spreads to other parts of the body. The ciliary body is a ring of tissue with muscle fibers that change the shape of the lens. It is found behind the iris. Changes in the shape of the lens help the eye focus. The ciliary body also makes the clear fluid that fills the space between the cornea and the iris. Intraocular melanoma of the ciliary body is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. The choroid is a layer of blood vessels that bring oxygen and nutrients to the eye. Most intraocular melanomas begin in the choroid. Intraocular melanoma of the choroid is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. Intraocular melanoma is a rare cancer that forms from cells that make melanin in the iris, ciliary body, and choroid. It is the most common eye cancer in adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for intraocular melanoma include the following: Intraocular melanoma may not cause early signs or symptoms. It is sometimes found during a regular eye exam when the doctor dilates the pupil and looks into the eye. Signs and symptoms may be caused by intraocular melanoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: A biopsy is the removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. Rarely, a biopsy of the tumor is needed to diagnose intraocular melanoma. Tissue that is removed during a biopsy or surgery to remove the tumor may be tested to get more information about prognosis and which treatment options are best. The following tests may be done on the sample of tissue: A biopsy may result in retinal detachment (the retina separates from other tissues in the eye). This can be repaired by surgery. The prognosis and treatment options depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The tumor is 5 to 16 millimeters in diameter and from 1 to 3 millimeters thick. EnlargeMillimeters (mm). A sharp pencil point is about 1 mm, a new crayon point is about 2 mm, and a new pencil eraser is about 5 mm. The tumor is 16 millimeters or smaller in diameter and from 3.1 to 8 millimeters thick. The tumor is: Though most intraocular melanoma tumors are raised, some are flat. These diffuse tumors grow widely across the uvea. Cancer can spread through tissue, the lymph system, and the blood: If intraocular melanoma spreads to the optic nerve or nearby tissue of the eye socket, it is called extraocular extension. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if intraocular melanoma spreads to the liver, the cancer cells in the liver are actually intraocular melanoma cells. The disease is metastatic intraocular melanoma, not liver cancer. Intraocular melanoma of the ciliary body and choroid has four size categories. The category depends on how wide and thick the tumor is. Category 1 tumors are the smallest and category 4 tumors are the largest. Category 1: Category 2: Category 3: Category 4: In stage I, the tumor is size category 1 and is in the choroid only. Stage II is divided into stages IIA and IIB. Stage III is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor may be any size and has spread: The melanoma may come back in the eye or in other parts of the body. Different types of treatments are available for patients with intraocular melanoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for intraocular melanoma. The following types of surgery may be used: Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Pictures are taken over time to keep track of changes in the size of the tumor and how fast it is growing. Watchful waiting is used for patients who do not have signs or symptoms and the tumor is not growing. It is also used when the tumor is in the only eye with useful vision. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat intraocular melanoma. Photocoagulation is a procedure that uses laser light to destroy blood vessels that bring nutrients to the tumor, causing the tumor cells to die. Photocoagulation may be used to treat small tumors. This is also called light coagulation. Thermotherapy is the use of heat from a laser to destroy cancer cells and shrink the tumor. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of iris melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of ciliary body melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of small choroid melanoma may include the following: Treatment of medium choroid melanoma may include the following: Treatment of large choroid melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of extraocular extension melanoma that has spread to the bone around the eye may include the following: An effective treatment for metastatic intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. An effective treatment for recurrent intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about intraocular (uveal) melanoma, see the Intraocular (Eye) Melanoma Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of intraocular melanoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Intraocular (Uveal) Melanoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/eye/patient/intraocular-melanoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389277] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Intraocular (Uveal) Melanoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Intraocular (Uveal) Melanoma ?

Signs of intraocular melanoma include blurred vision or a dark spot on the iris. Intraocular melanoma may not cause early signs or symptoms. It is sometimes found during a regular eye exam when the doctor dilates the pupil and looks into the eye. Signs and symptoms may be caused by intraocular melanoma or by other conditions. Check with your doctor if you have any of the following: - Blurred vision or other change in vision. - Floaters (spots that drift in your field of vision) or flashes of light. - A dark spot on the iris. - A change in the size or shape of the pupil. - A change in the position of the eyeball in the eye socket.

Intraocular melanoma begins in the middle of three layers of the wall of the eye. The outer layer includes the white sclera (the "white of the eye") and the clear cornea at the front of the eye. The inner layer has a lining of nerve tissue, called the retina, which senses light and sends images along the optic nerve to the brain. The middle layer, where intraocular melanoma forms, is called the uvea or uveal tract, and has three main parts: The iris is the colored area at the front of the eye (the "eye color"). It can be seen through the clear cornea. The pupil is in the center of the iris and it changes size to let more or less light into the eye. Intraocular melanoma of the iris is usually a small tumor that grows slowly and rarely spreads to other parts of the body. The ciliary body is a ring of tissue with muscle fibers that change the shape of the lens. It is found behind the iris. Changes in the shape of the lens help the eye focus. The ciliary body also makes the clear fluid that fills the space between the cornea and the iris. Intraocular melanoma of the ciliary body is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. The choroid is a layer of blood vessels that bring oxygen and nutrients to the eye. Most intraocular melanomas begin in the choroid. Intraocular melanoma of the choroid is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. Intraocular melanoma is a rare cancer that forms from cells that make melanin in the iris, ciliary body, and choroid. It is the most common eye cancer in adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for intraocular melanoma include the following: Intraocular melanoma may not cause early signs or symptoms. It is sometimes found during a regular eye exam when the doctor dilates the pupil and looks into the eye. Signs and symptoms may be caused by intraocular melanoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: A biopsy is the removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. Rarely, a biopsy of the tumor is needed to diagnose intraocular melanoma. Tissue that is removed during a biopsy or surgery to remove the tumor may be tested to get more information about prognosis and which treatment options are best. The following tests may be done on the sample of tissue: A biopsy may result in retinal detachment (the retina separates from other tissues in the eye). This can be repaired by surgery. The prognosis and treatment options depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The tumor is 5 to 16 millimeters in diameter and from 1 to 3 millimeters thick. EnlargeMillimeters (mm). A sharp pencil point is about 1 mm, a new crayon point is about 2 mm, and a new pencil eraser is about 5 mm. The tumor is 16 millimeters or smaller in diameter and from 3.1 to 8 millimeters thick. The tumor is: Though most intraocular melanoma tumors are raised, some are flat. These diffuse tumors grow widely across the uvea. Cancer can spread through tissue, the lymph system, and the blood: If intraocular melanoma spreads to the optic nerve or nearby tissue of the eye socket, it is called extraocular extension. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if intraocular melanoma spreads to the liver, the cancer cells in the liver are actually intraocular melanoma cells. The disease is metastatic intraocular melanoma, not liver cancer. Intraocular melanoma of the ciliary body and choroid has four size categories. The category depends on how wide and thick the tumor is. Category 1 tumors are the smallest and category 4 tumors are the largest. Category 1: Category 2: Category 3: Category 4: In stage I, the tumor is size category 1 and is in the choroid only. Stage II is divided into stages IIA and IIB. Stage III is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor may be any size and has spread: The melanoma may come back in the eye or in other parts of the body. Different types of treatments are available for patients with intraocular melanoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for intraocular melanoma. The following types of surgery may be used: Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Pictures are taken over time to keep track of changes in the size of the tumor and how fast it is growing. Watchful waiting is used for patients who do not have signs or symptoms and the tumor is not growing. It is also used when the tumor is in the only eye with useful vision. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat intraocular melanoma. Photocoagulation is a procedure that uses laser light to destroy blood vessels that bring nutrients to the tumor, causing the tumor cells to die. Photocoagulation may be used to treat small tumors. This is also called light coagulation. Thermotherapy is the use of heat from a laser to destroy cancer cells and shrink the tumor. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of iris melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of ciliary body melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of small choroid melanoma may include the following: Treatment of medium choroid melanoma may include the following: Treatment of large choroid melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of extraocular extension melanoma that has spread to the bone around the eye may include the following: An effective treatment for metastatic intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. An effective treatment for recurrent intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about intraocular (uveal) melanoma, see the Intraocular (Eye) Melanoma Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of intraocular melanoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Intraocular (Uveal) Melanoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/eye/patient/intraocular-melanoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389277] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Intraocular (Uveal) Melanoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Intraocular (Uveal) Melanoma ?

Tests that examine the eye are used to help detect (find) and diagnose intraocular melanoma. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Eye exam with dilated pupil: An exam of the eye in which the pupil is dilated (enlarged) with medicated eye drops to allow the doctor to look through the lens and pupil to the retina. The inside of the eye, including the retina and the optic nerve, is checked. Pictures may be taken over time to keep track of changes in the size of the tumor. There are several types of eye exams: - Ophthalmoscopy : An exam of the inside of the back of the eye to check the retina and optic nerve using a small magnifying lens and a light. - Slit-lamp biomicroscopy : An exam of the inside of the eye to check the retina, optic nerve, and other parts of the eye using a strong beam of light and a microscope. - Gonioscopy : An exam of the front part of the eye between the cornea and iris. A special instrument is used to see if the area where fluid drains out of the eye is blocked. - Ultrasound exam of the eye: A procedure in which high-energy sound waves (ultrasound) are bounced off the internal tissues of the eye to make echoes. Eye drops are used to numb the eye and a small probe that sends and receives sound waves is placed gently on the surface of the eye. The echoes make a picture of the inside of the eye and the distance from the cornea to the retina is measured. The picture, called a sonogram, shows on the screen of the ultrasound monitor. - High-resolution ultrasound biomicroscopy : A procedure in which high-energy sound waves (ultrasound) are bounced off the internal tissues of the eye to make echoes. Eye drops are used to numb the eye and a small probe that sends and receives sound waves is placed gently on the surface of the eye. The echoes make a more detailed picture of the inside of the eye than a regular ultrasound. The tumor is checked for its size, shape, and thickness, and for signs that the tumor has spread to nearby tissue. - Transillumination of the globe and iris: An exam of the iris, cornea, lens, and ciliary body with a light placed on either the upper or lower lid. - Fluorescein angiography : A procedure to look at blood vessels and the flow of blood inside the eye. An orange fluorescent dye (fluorescein) is injected into a blood vessel in the arm and goes into the bloodstream. As the dye travels through blood vessels of the eye, a special camera takes pictures of the retina and choroid to find any areas that are blocked or leaking. - Indocyanine green angiography: A procedure to look at blood vessels in the choroid layer of the eye. A green dye (indocyanine green) is injected into a blood vessel in the arm and goes into the bloodstream. As the dye travels through blood vessels of the eye, a special camera takes pictures of the retina and choroid to find any areas that are blocked or leaking. - Ocular coherence tomography : An imaging test that uses light waves to take cross-section pictures of the retina, and sometimes the choroid, to see if there is swelling or fluid beneath the retina. A biopsy of the tumor is rarely needed to diagnose intraocular melanoma. A biopsy is the removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. Rarely, a biopsy of the tumor is needed to diagnose intraocular melanoma. Tissue that is removed during a biopsy or surgery to remove the tumor may be tested to get more information about prognosis and which treatment options are best. The following tests may be done on the sample of tissue: - Cytogenetic analysis: A laboratory test in which cells in a sample of tissue are viewed under a microscope to look for certain changes in the chromosomes. - Gene expression profiling : A laboratory test in which cells in a sample of tissue are checked for certain types of RNA. A biopsy may result in retinal detachment (the retina separates from other tissues in the eye). This can be repaired by surgery.

Intraocular melanoma begins in the middle of three layers of the wall of the eye. The outer layer includes the white sclera (the "white of the eye") and the clear cornea at the front of the eye. The inner layer has a lining of nerve tissue, called the retina, which senses light and sends images along the optic nerve to the brain. The middle layer, where intraocular melanoma forms, is called the uvea or uveal tract, and has three main parts: The iris is the colored area at the front of the eye (the "eye color"). It can be seen through the clear cornea. The pupil is in the center of the iris and it changes size to let more or less light into the eye. Intraocular melanoma of the iris is usually a small tumor that grows slowly and rarely spreads to other parts of the body. The ciliary body is a ring of tissue with muscle fibers that change the shape of the lens. It is found behind the iris. Changes in the shape of the lens help the eye focus. The ciliary body also makes the clear fluid that fills the space between the cornea and the iris. Intraocular melanoma of the ciliary body is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. The choroid is a layer of blood vessels that bring oxygen and nutrients to the eye. Most intraocular melanomas begin in the choroid. Intraocular melanoma of the choroid is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. Intraocular melanoma is a rare cancer that forms from cells that make melanin in the iris, ciliary body, and choroid. It is the most common eye cancer in adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for intraocular melanoma include the following: Intraocular melanoma may not cause early signs or symptoms. It is sometimes found during a regular eye exam when the doctor dilates the pupil and looks into the eye. Signs and symptoms may be caused by intraocular melanoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: A biopsy is the removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. Rarely, a biopsy of the tumor is needed to diagnose intraocular melanoma. Tissue that is removed during a biopsy or surgery to remove the tumor may be tested to get more information about prognosis and which treatment options are best. The following tests may be done on the sample of tissue: A biopsy may result in retinal detachment (the retina separates from other tissues in the eye). This can be repaired by surgery. The prognosis and treatment options depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The tumor is 5 to 16 millimeters in diameter and from 1 to 3 millimeters thick. EnlargeMillimeters (mm). A sharp pencil point is about 1 mm, a new crayon point is about 2 mm, and a new pencil eraser is about 5 mm. The tumor is 16 millimeters or smaller in diameter and from 3.1 to 8 millimeters thick. The tumor is: Though most intraocular melanoma tumors are raised, some are flat. These diffuse tumors grow widely across the uvea. Cancer can spread through tissue, the lymph system, and the blood: If intraocular melanoma spreads to the optic nerve or nearby tissue of the eye socket, it is called extraocular extension. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if intraocular melanoma spreads to the liver, the cancer cells in the liver are actually intraocular melanoma cells. The disease is metastatic intraocular melanoma, not liver cancer. Intraocular melanoma of the ciliary body and choroid has four size categories. The category depends on how wide and thick the tumor is. Category 1 tumors are the smallest and category 4 tumors are the largest. Category 1: Category 2: Category 3: Category 4: In stage I, the tumor is size category 1 and is in the choroid only. Stage II is divided into stages IIA and IIB. Stage III is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor may be any size and has spread: The melanoma may come back in the eye or in other parts of the body. Different types of treatments are available for patients with intraocular melanoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for intraocular melanoma. The following types of surgery may be used: Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Pictures are taken over time to keep track of changes in the size of the tumor and how fast it is growing. Watchful waiting is used for patients who do not have signs or symptoms and the tumor is not growing. It is also used when the tumor is in the only eye with useful vision. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat intraocular melanoma. Photocoagulation is a procedure that uses laser light to destroy blood vessels that bring nutrients to the tumor, causing the tumor cells to die. Photocoagulation may be used to treat small tumors. This is also called light coagulation. Thermotherapy is the use of heat from a laser to destroy cancer cells and shrink the tumor. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of iris melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of ciliary body melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of small choroid melanoma may include the following: Treatment of medium choroid melanoma may include the following: Treatment of large choroid melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of extraocular extension melanoma that has spread to the bone around the eye may include the following: An effective treatment for metastatic intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. An effective treatment for recurrent intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about intraocular (uveal) melanoma, see the Intraocular (Eye) Melanoma Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of intraocular melanoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Intraocular (Uveal) Melanoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/eye/patient/intraocular-melanoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389277] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Intraocular (Uveal) Melanoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? 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What is the outlook for Intraocular (Uveal) Melanoma ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options depend on the following: - How the melanoma cells look under a microscope. - The size and thickness of the tumor. - The part of the eye the tumor is in (the iris, ciliary body, or choroid). - Whether the tumor has spread within the eye or to other places in the body. - Whether there are certain changes in the genes linked to intraocular melanoma. - The patient&apos;s age and general health. - Whether the tumor has recurred (come back) after treatment.

Intraocular melanoma begins in the middle of three layers of the wall of the eye. The outer layer includes the white sclera (the "white of the eye") and the clear cornea at the front of the eye. The inner layer has a lining of nerve tissue, called the retina, which senses light and sends images along the optic nerve to the brain. The middle layer, where intraocular melanoma forms, is called the uvea or uveal tract, and has three main parts: The iris is the colored area at the front of the eye (the "eye color"). It can be seen through the clear cornea. The pupil is in the center of the iris and it changes size to let more or less light into the eye. Intraocular melanoma of the iris is usually a small tumor that grows slowly and rarely spreads to other parts of the body. The ciliary body is a ring of tissue with muscle fibers that change the shape of the lens. It is found behind the iris. Changes in the shape of the lens help the eye focus. The ciliary body also makes the clear fluid that fills the space between the cornea and the iris. Intraocular melanoma of the ciliary body is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. The choroid is a layer of blood vessels that bring oxygen and nutrients to the eye. Most intraocular melanomas begin in the choroid. Intraocular melanoma of the choroid is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. Intraocular melanoma is a rare cancer that forms from cells that make melanin in the iris, ciliary body, and choroid. It is the most common eye cancer in adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for intraocular melanoma include the following: Intraocular melanoma may not cause early signs or symptoms. It is sometimes found during a regular eye exam when the doctor dilates the pupil and looks into the eye. Signs and symptoms may be caused by intraocular melanoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: A biopsy is the removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. Rarely, a biopsy of the tumor is needed to diagnose intraocular melanoma. Tissue that is removed during a biopsy or surgery to remove the tumor may be tested to get more information about prognosis and which treatment options are best. The following tests may be done on the sample of tissue: A biopsy may result in retinal detachment (the retina separates from other tissues in the eye). This can be repaired by surgery. The prognosis and treatment options depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The tumor is 5 to 16 millimeters in diameter and from 1 to 3 millimeters thick. EnlargeMillimeters (mm). A sharp pencil point is about 1 mm, a new crayon point is about 2 mm, and a new pencil eraser is about 5 mm. The tumor is 16 millimeters or smaller in diameter and from 3.1 to 8 millimeters thick. The tumor is: Though most intraocular melanoma tumors are raised, some are flat. These diffuse tumors grow widely across the uvea. Cancer can spread through tissue, the lymph system, and the blood: If intraocular melanoma spreads to the optic nerve or nearby tissue of the eye socket, it is called extraocular extension. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if intraocular melanoma spreads to the liver, the cancer cells in the liver are actually intraocular melanoma cells. The disease is metastatic intraocular melanoma, not liver cancer. Intraocular melanoma of the ciliary body and choroid has four size categories. The category depends on how wide and thick the tumor is. Category 1 tumors are the smallest and category 4 tumors are the largest. Category 1: Category 2: Category 3: Category 4: In stage I, the tumor is size category 1 and is in the choroid only. Stage II is divided into stages IIA and IIB. Stage III is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor may be any size and has spread: The melanoma may come back in the eye or in other parts of the body. Different types of treatments are available for patients with intraocular melanoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for intraocular melanoma. The following types of surgery may be used: Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Pictures are taken over time to keep track of changes in the size of the tumor and how fast it is growing. Watchful waiting is used for patients who do not have signs or symptoms and the tumor is not growing. It is also used when the tumor is in the only eye with useful vision. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat intraocular melanoma. Photocoagulation is a procedure that uses laser light to destroy blood vessels that bring nutrients to the tumor, causing the tumor cells to die. Photocoagulation may be used to treat small tumors. This is also called light coagulation. Thermotherapy is the use of heat from a laser to destroy cancer cells and shrink the tumor. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of iris melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of ciliary body melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of small choroid melanoma may include the following: Treatment of medium choroid melanoma may include the following: Treatment of large choroid melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of extraocular extension melanoma that has spread to the bone around the eye may include the following: An effective treatment for metastatic intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. An effective treatment for recurrent intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about intraocular (uveal) melanoma, see the Intraocular (Eye) Melanoma Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of intraocular melanoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Intraocular (Uveal) Melanoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/eye/patient/intraocular-melanoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389277] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Intraocular (Uveal) Melanoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Intraocular (Uveal) Melanoma ?

Key Points - After intraocular melanoma has been diagnosed, tests are done to find out if cancer cells have spread to other parts of the body. - The following sizes are used to describe intraocular melanoma: - Small - Medium - Large - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - There are two staging systems for intraocular melanoma. - The following stages are used for intraocular melanoma of the iris: - Stage I - Stage II - Stage III - Stage IV - The following stages are used for intraocular melanoma of the ciliary body and choroid: - Stage I - Stage II - Stage III - Stage IV After intraocular melanoma has been diagnosed, tests are done to find out if cancer cells have spread to other parts of the body. The process used to find out if cancer has spread to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: - Blood chemistry studies : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease. - Liver function tests : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by the liver. A higher than normal amount of a substance can be a sign the cancer has spread to the liver. - Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs, such as the liver, and make echoes. The echoes form a picture of body tissues called a sonogram. - Chest x-ray : An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the chest, abdomen, or pelvis, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A very small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. Sometimes a PET scan and a CT scan are done at the same time. If there is any cancer, this increases the chance that it will be found. The following sizes are used to describe intraocular melanoma: Small The tumor is 5 to 16 millimeters in diameter and from 1 to 3 millimeters thick. Medium The tumor is 16 millimeters or smaller in diameter and from 3.1 to 8 millimeters thick. Large The tumor is: - more than 8 millimeters thick and any diameter; or - at least 2 millimeters thick and more than 16 millimeters in diameter. Though most intraocular melanoma tumors are raised, some are flat. These diffuse tumors grow widely across the uvea. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if intraocular melanoma spreads to the liver, the cancer cells in the liver are actually intraocular melanoma cells. The disease is metastatic intraocular melanoma, not liver cancer. There are two staging systems for intraocular melanoma. Intraocular melanoma has two staging systems. The staging system used depends on where in the eye the cancer first formed: - Iris. - Ciliary body and choroid. If intraocular melanoma spreads to the optic nerve or nearby tissue of the eye socket, it is called extraocular extension. The following stages are used for intraocular melanoma of the iris: Stage I In stage I, the tumor is in the iris only and is not more than one fourth the size of the iris. Stage II Stage II is divided into stages IIA and IIB. - In stage IIA, the tumor: - is in the iris only and is more than one fourth the size of the iris; or - is in the iris only and has caused glaucoma; or - has spread next to and/or into the ciliary body, choroid, or both. The tumor has caused glaucoma. - In stage IIB, the tumor has spread next to and/or into the ciliary body, choroid, or both, and has also spread into the sclera. The tumor has caused glaucoma. Stage III Stage III is divided into stages IIIA and IIIB. - In stage IIIA, the tumor has spread through the sclera to the outside of the eyeball. The part of the tumor outside the eyeball is not more than 5 millimeters thick. - In stage IIIB, the tumor has spread through the sclera to the outside of the eyeball. The part of the tumor outside the eyeball is more than 5 millimeters thick. Stage IV In stage IV, the tumor may be any size and has spread: - to nearby lymph nodes; or - to other parts of the body, such as the liver, lung, or bone, or to areas under the skin. The following stages are used for intraocular melanoma of the ciliary body and choroid: Intraocular melanoma of the ciliary body and choroid is grouped into four size categories. The category depends on how wide and thick the tumor is. Category 1 tumors are the smallest and category 4 tumors are the biggest. Category 1: - The tumor is not more than 12 millimeters wide and not more than 3 millimeters thick; or - the tumor is not more than 9 millimeters wide and 3.1 to 6 millimeters thick. Category 2: - The tumor is 12.1 to 18 millimeters wide and not more than 3 millimeters thick; or - the tumor is 9.1 to 15 millimeters wide and 3.1 to 6 millimeters thick; or - the tumor is not more than 12 millimeters wide and 6.1 to 9 millimeters thick. Category 3: - The tumor is 15.1 to 18 millimeters wide and 3.1 to 6 millimeters thick; or - the tumor is 12.1 to 18 millimeters wide and 6.1 to 9 millimeters thick; or - the tumor is 3.1 to 18 millimeters wide and 9.1 to 12 millimeters thick; or - the tumor is 9.1 to 15 millimeters wide and 12.1 to 15 millimeters thick. Category 4: - The tumor is more than 18 millimeters wide and may be any thickness; or - the tumor is 15.1 to 18 millimeters wide and more than 12 millimeters thick; or - the tumor is 12.1 to 15 millimeters wide and more than 15 millimeters thick. Stage I In stage I, the tumor is size category 1 and is in the choroid only. Stage II Stage II is divided into stages IIA and IIB. - In stage IIA, the tumor: - is size category 1 and has spread to the ciliary body; or - is size category 1 and has spread through the sclera to the outside of the eyeball. The part of the tumor outside the eyeball is not more than 5 millimeters thick. The tumor may have spread to the ciliary body; or - is size category 2 and is in the choroid only. - In stage IIB, the tumor: - is size category 2 and has spread to the ciliary body; or - is size category 3 and is in the choroid only. Stage III Stage III is divided into stages IIIA, IIIB, and IIIC. - In stage IIIA, the tumor: - is size category 2 and has spread through the sclera to the outside of the eyeball. The part of the tumor outside the eyeball is not more than 5 millimeters thick. The tumor may have spread to the ciliary body; or - is size category 3 and has spread to the ciliary body; or - is size category 3 and has spread through the sclera to the outside of the eyeball. The part of the tumor outside the eyeball is not more than 5 millimeters thick. The tumor has not spread to the ciliary body; or - is size category 4 and is in the choroid only. - In stage IIIB, the tumor: - is size category 3 and has spread through the sclera to the outside of the eyeball. The part of the tumor outside the eyeball is not more than 5 millimeters thick. The tumor has spread to the ciliary body; or - is size category 4 and has spread to the ciliary body; or - is size category 4 and has spread through the sclera to the outside of the eyeball. The part of the tumor outside the eyeball is not more than 5 millimeters thick. The tumor has not spread to the ciliary body. - In stage IIIC, the tumor: - is size category 4 and has spread through the sclera to the outside of the eyeball. The part of the tumor outside the eyeball is not more than 5 millimeters thick. The tumor has spread to the ciliary body; or - may be any size and has spread through the sclera to the outside of the eyeball. The part of the tumor outside the eyeball is more than 5 millimeters thick. The tumor has not spread to the ciliary body. Stage IV In stage IV, the tumor may be any size and has spread: - to nearby lymph nodes; or - to other parts of the body, such as the liver, lung, or bone, or to areas under the skin.

Intraocular melanoma begins in the middle of three layers of the wall of the eye. The outer layer includes the white sclera (the "white of the eye") and the clear cornea at the front of the eye. The inner layer has a lining of nerve tissue, called the retina, which senses light and sends images along the optic nerve to the brain. The middle layer, where intraocular melanoma forms, is called the uvea or uveal tract, and has three main parts: The iris is the colored area at the front of the eye (the "eye color"). It can be seen through the clear cornea. The pupil is in the center of the iris and it changes size to let more or less light into the eye. Intraocular melanoma of the iris is usually a small tumor that grows slowly and rarely spreads to other parts of the body. The ciliary body is a ring of tissue with muscle fibers that change the shape of the lens. It is found behind the iris. Changes in the shape of the lens help the eye focus. The ciliary body also makes the clear fluid that fills the space between the cornea and the iris. Intraocular melanoma of the ciliary body is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. The choroid is a layer of blood vessels that bring oxygen and nutrients to the eye. Most intraocular melanomas begin in the choroid. Intraocular melanoma of the choroid is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. Intraocular melanoma is a rare cancer that forms from cells that make melanin in the iris, ciliary body, and choroid. It is the most common eye cancer in adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for intraocular melanoma include the following: Intraocular melanoma may not cause early signs or symptoms. It is sometimes found during a regular eye exam when the doctor dilates the pupil and looks into the eye. Signs and symptoms may be caused by intraocular melanoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: A biopsy is the removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. Rarely, a biopsy of the tumor is needed to diagnose intraocular melanoma. Tissue that is removed during a biopsy or surgery to remove the tumor may be tested to get more information about prognosis and which treatment options are best. The following tests may be done on the sample of tissue: A biopsy may result in retinal detachment (the retina separates from other tissues in the eye). This can be repaired by surgery. The prognosis and treatment options depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The tumor is 5 to 16 millimeters in diameter and from 1 to 3 millimeters thick. EnlargeMillimeters (mm). A sharp pencil point is about 1 mm, a new crayon point is about 2 mm, and a new pencil eraser is about 5 mm. The tumor is 16 millimeters or smaller in diameter and from 3.1 to 8 millimeters thick. The tumor is: Though most intraocular melanoma tumors are raised, some are flat. These diffuse tumors grow widely across the uvea. Cancer can spread through tissue, the lymph system, and the blood: If intraocular melanoma spreads to the optic nerve or nearby tissue of the eye socket, it is called extraocular extension. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if intraocular melanoma spreads to the liver, the cancer cells in the liver are actually intraocular melanoma cells. The disease is metastatic intraocular melanoma, not liver cancer. Intraocular melanoma of the ciliary body and choroid has four size categories. The category depends on how wide and thick the tumor is. Category 1 tumors are the smallest and category 4 tumors are the largest. Category 1: Category 2: Category 3: Category 4: In stage I, the tumor is size category 1 and is in the choroid only. Stage II is divided into stages IIA and IIB. Stage III is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor may be any size and has spread: The melanoma may come back in the eye or in other parts of the body. Different types of treatments are available for patients with intraocular melanoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for intraocular melanoma. The following types of surgery may be used: Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Pictures are taken over time to keep track of changes in the size of the tumor and how fast it is growing. Watchful waiting is used for patients who do not have signs or symptoms and the tumor is not growing. It is also used when the tumor is in the only eye with useful vision. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat intraocular melanoma. Photocoagulation is a procedure that uses laser light to destroy blood vessels that bring nutrients to the tumor, causing the tumor cells to die. Photocoagulation may be used to treat small tumors. This is also called light coagulation. Thermotherapy is the use of heat from a laser to destroy cancer cells and shrink the tumor. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of iris melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of ciliary body melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of small choroid melanoma may include the following: Treatment of medium choroid melanoma may include the following: Treatment of large choroid melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of extraocular extension melanoma that has spread to the bone around the eye may include the following: An effective treatment for metastatic intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. An effective treatment for recurrent intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about intraocular (uveal) melanoma, see the Intraocular (Eye) Melanoma Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of intraocular melanoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. 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What are the treatments for Intraocular (Uveal) Melanoma ?

Key Points - There are different types of treatments for patients with intraocular melanoma. - Five types of standard treatment are used: - Surgery - Watchful Waiting - Radiation therapy - Photocoagulation - Thermotherapy - New types of treatment are being tested in clinical trials. - Treatment for intraocular (uveal) melanoma may cause side effects. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatments for patients with intraocular melanoma. Different types of treatments are available for patients with intraocular melanoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Five types of standard treatment are used: Surgery Surgery is the most common treatment for intraocular melanoma. The following types of surgery may be used: - Resection: Surgery to remove the tumor and a small amount of healthy tissue around it. - Enucleation: Surgery to remove the eye and part of the optic nerve. This is done if vision cannot be saved and the tumor is large, has spread to the optic nerve, or causes high pressure inside the eye. After surgery, the patient is usually fitted for an artificial eye to match the size and color of the other eye. - Exenteration: Surgery to remove the eye and eyelid, and muscles, nerves, and fat in the eye socket. After surgery, the patient may be fitted for an artificial eye to match the size and color of the other eye or a facial prosthesis. Watchful Waiting Watchful waiting is closely monitoring a patients condition without giving any treatment until signs or symptoms appear or change. Pictures are taken over time to keep track of changes in the size of the tumor and how fast it is growing. Watchful waiting is used for patients who do not have signs or symptoms and the tumor is not growing. It is also used when the tumor is in the only eye with useful vision. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. Certain ways of giving radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of external radiation therapy include the following: - Charged-particle external beam radiation therapy is a type of external-beam radiation therapy. A special radiation therapy machine aims tiny, invisible particles, called protons or helium ions, at the cancer cells to kill them with little damage to nearby normal tissues. Charged-particle radiation therapy uses a different type of radiation than the x-ray type of radiation therapy. - Gamma Knife therapy is a type of stereotactic radiosurgery used for some melanomas. This treatment can be given in one treatment. It aims tightly focused gamma rays directly at the tumor so there is little damage to healthy tissue. Gamma Knife therapy does not use a knife to remove the tumor and is not an operation. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. Certain ways of giving radiation therapy can help keep radiation from damaging healthy tissue. This type of internal radiation therapy may include the following: - Localized plaque radiation therapy is a type of internal radiation therapy that may be used for tumors of the eye. Radioactive seeds are attached to one side of a disk, called a plaque, and placed directly on the outside wall of the eye near the tumor. The side of the plaque with the seeds on it faces the eyeball, aiming radiation at the tumor. The plaque helps protect other nearby tissue from the radiation. The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat intraocular melanoma. Photocoagulation Photocoagulation is a procedure that uses laser light to destroy blood vessels that bring nutrients to the tumor, causing the tumor cells to die. Photocoagulation may be used to treat small tumors. This is also called light coagulation. Thermotherapy Thermotherapy is the use of heat from a laser to destroy cancer cells and shrink the tumor. New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Treatment for intraocular (uveal) melanoma may cause side effects. For information about side effects caused by treatment for cancer, see our Side Effects page. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Intraocular (Uveal) Melanoma Iris Melanoma Treatment of iris melanoma may include the following: - Watchful waiting. - Surgery (resection or enucleation). - Plaque radiation therapy, for tumors that cannot be removed by surgery. Check the list of NCI-supported cancer clinical trials that are now accepting patients with iris melanoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Ciliary Body Melanoma Treatment of tumors in the ciliary body and choroid may include the following: - Plaque radiation therapy. - Charged-particle external-beam radiation therapy. - Surgery (resection or enucleation). Check the list of NCI-supported cancer clinical trials that are now accepting patients with ciliary body and choroid melanoma, small size. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Choroid Melanoma Treatment of small choroid melanoma may include the following: - Watchful waiting. - Plaque radiation therapy. - Charged-particle external-beam radiation therapy. - Gamma Knife therapy. - Thermotherapy. - Surgery (resection or enucleation). Treatment of medium choroid melanoma may include the following: - Plaque radiation therapy with or without photocoagulation or thermotherapy. - Charged-particle external-beam radiation therapy. - Surgery (resection or enucleation). Treatment of large choroid melanoma may include the following: - Enucleation when the tumor is too large for treatments that save the eye. Check the list of NCI-supported cancer clinical trials that are now accepting patients with ciliary body and choroid melanoma, small size. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Extraocular Extension Melanoma and Metastatic Intraocular (Uveal) Melanoma Treatment of extraocular extension melanoma that has spread to the bone around the eye may include the following: - Surgery (exenteration). - A clinical trial. An effective treatment for metastatic intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Check the list of NCI-supported cancer clinical trials that are now accepting patients with extraocular extension melanoma and metastatic intraocular melanoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Recurrent Intraocular (Uveal) Melanoma An effective treatment for recurrent intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Check the list of NCI-supported cancer clinical trials that are now accepting patients with recurrent intraocular melanoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

Intraocular melanoma begins in the middle of three layers of the wall of the eye. The outer layer includes the white sclera (the "white of the eye") and the clear cornea at the front of the eye. The inner layer has a lining of nerve tissue, called the retina, which senses light and sends images along the optic nerve to the brain. The middle layer, where intraocular melanoma forms, is called the uvea or uveal tract, and has three main parts: The iris is the colored area at the front of the eye (the "eye color"). It can be seen through the clear cornea. The pupil is in the center of the iris and it changes size to let more or less light into the eye. Intraocular melanoma of the iris is usually a small tumor that grows slowly and rarely spreads to other parts of the body. The ciliary body is a ring of tissue with muscle fibers that change the shape of the lens. It is found behind the iris. Changes in the shape of the lens help the eye focus. The ciliary body also makes the clear fluid that fills the space between the cornea and the iris. Intraocular melanoma of the ciliary body is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. The choroid is a layer of blood vessels that bring oxygen and nutrients to the eye. Most intraocular melanomas begin in the choroid. Intraocular melanoma of the choroid is often larger and more likely to spread to other parts of the body than intraocular melanoma of the iris. Intraocular melanoma is a rare cancer that forms from cells that make melanin in the iris, ciliary body, and choroid. It is the most common eye cancer in adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for intraocular melanoma include the following: Intraocular melanoma may not cause early signs or symptoms. It is sometimes found during a regular eye exam when the doctor dilates the pupil and looks into the eye. Signs and symptoms may be caused by intraocular melanoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: A biopsy is the removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. Rarely, a biopsy of the tumor is needed to diagnose intraocular melanoma. Tissue that is removed during a biopsy or surgery to remove the tumor may be tested to get more information about prognosis and which treatment options are best. The following tests may be done on the sample of tissue: A biopsy may result in retinal detachment (the retina separates from other tissues in the eye). This can be repaired by surgery. The prognosis and treatment options depend on the following: The process used to find out if cancer has spread to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The tumor is 5 to 16 millimeters in diameter and from 1 to 3 millimeters thick. EnlargeMillimeters (mm). A sharp pencil point is about 1 mm, a new crayon point is about 2 mm, and a new pencil eraser is about 5 mm. The tumor is 16 millimeters or smaller in diameter and from 3.1 to 8 millimeters thick. The tumor is: Though most intraocular melanoma tumors are raised, some are flat. These diffuse tumors grow widely across the uvea. Cancer can spread through tissue, the lymph system, and the blood: If intraocular melanoma spreads to the optic nerve or nearby tissue of the eye socket, it is called extraocular extension. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if intraocular melanoma spreads to the liver, the cancer cells in the liver are actually intraocular melanoma cells. The disease is metastatic intraocular melanoma, not liver cancer. Intraocular melanoma of the ciliary body and choroid has four size categories. The category depends on how wide and thick the tumor is. Category 1 tumors are the smallest and category 4 tumors are the largest. Category 1: Category 2: Category 3: Category 4: In stage I, the tumor is size category 1 and is in the choroid only. Stage II is divided into stages IIA and IIB. Stage III is divided into stages IIIA, IIIB, and IIIC. In stage IV, the tumor may be any size and has spread: The melanoma may come back in the eye or in other parts of the body. Different types of treatments are available for patients with intraocular melanoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for intraocular melanoma. The following types of surgery may be used: Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Pictures are taken over time to keep track of changes in the size of the tumor and how fast it is growing. Watchful waiting is used for patients who do not have signs or symptoms and the tumor is not growing. It is also used when the tumor is in the only eye with useful vision. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat intraocular melanoma. Photocoagulation is a procedure that uses laser light to destroy blood vessels that bring nutrients to the tumor, causing the tumor cells to die. Photocoagulation may be used to treat small tumors. This is also called light coagulation. Thermotherapy is the use of heat from a laser to destroy cancer cells and shrink the tumor. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of iris melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of ciliary body melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of small choroid melanoma may include the following: Treatment of medium choroid melanoma may include the following: Treatment of large choroid melanoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. Treatment of extraocular extension melanoma that has spread to the bone around the eye may include the following: An effective treatment for metastatic intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. An effective treatment for recurrent intraocular melanoma has not been found. A clinical trial may be a treatment option. Talk with your doctor about your treatment options. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about intraocular (uveal) melanoma, see the Intraocular (Eye) Melanoma Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of intraocular melanoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Intraocular (Uveal) Melanoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/eye/patient/intraocular-melanoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389277] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Intraocular (Uveal) Melanoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Intraocular (Uveal) Melanoma ?

New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Soft tissues of the body connect, support, and surround other body parts and organs. The soft tissues of the body include the following: Soft tissue sarcomas can form almost anywhere in the body, including the head, neck, and trunk, but are most common in the arms, legs, abdomen, and retroperitoneum.EnlargeSoft tissue sarcoma forms in soft tissues of the body, including muscle, tendons, fat, blood vessels, lymph vessels, nerves, and tissue around joints. There are many types of soft tissue sarcoma. The cells of each type of sarcoma look different under a microscope, based on the type of soft tissue in which the cancer began. For more information about soft tissue sarcomas, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for soft tissue sarcoma include the following inherited disorders: Other risk factors for soft tissue sarcoma include the following: A sarcoma may appear as a painless lump under the skin, often on an arm or a leg. Sarcomas that begin in the abdomen may not cause signs or symptoms until they get very big. As the sarcoma grows and presses on nearby organs, nerves, muscles, or blood vessels, signs and symptoms may include: Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of these problems. If your doctor thinks you may have a soft tissue sarcoma, a biopsy will be done. The type of biopsy will be based on the size of the tumor and where it is in the body. These types of biopsies may be used: Careful planning of the biopsy should involve the surgeon, a radiation oncologist, and an interventional radiologist who uses medical imaging to guide diagnosis. Samples will be taken from the primary tumor, lymph nodes, and other suspicious areas. A pathologist views the tissue under a microscope to look for cancer cells and to find out the grade of the tumor. The grade of a tumor depends on how abnormal the cancer cells look under a microscope and how quickly the cells are dividing. High-grade tumors usually grow and spread more quickly than low-grade tumors. Because soft tissue sarcoma can be hard to diagnose, the tissue samples should be checked by a pathologist who has experience in diagnosing soft tissue sarcoma. The following tests may be done on the tissue that was removed: The treatment options and prognosis depend on the following: Small, low-grade tumors, especially in the trunk, arms, or legs, are frequently treated with surgery alone. High-grade sarcomas are more difficult to treat and more likely to spread. The process used to find out if cancer has spread within the soft tissue or to other parts of the body is called staging. Staging of soft tissue sarcoma is also based on the grade and size of the tumor, and whether it has spread to the lymph nodes or other parts of the body. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The results of these tests are viewed together with the results of the tumor biopsy to find out the stage of the soft tissue sarcoma before treatment is given. Sometimes chemotherapy or radiation therapy is given as the initial treatment and afterwards the soft tissue sarcoma is staged again. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if soft tissue sarcoma spreads to the lung, the cancer cells in the lung are actually soft tissue sarcoma cells. The disease is metastatic soft tissue sarcoma, not lung cancer. The grade of the tumor describes how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. Low grade, mid grade, and high grade are used to describe soft tissue sarcoma: Stage I soft tissue sarcoma of the trunk, arms, and legs is divided into stages IA and IB: In stage II soft tissue sarcoma of the trunk, arms, and legs, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the trunk, arms, and legs is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the trunk, arms, and legs, one of the following is found: Stage I soft tissue sarcoma of the retroperitoneum is divided into stages IA and IB: In stage II soft tissue sarcoma of the retroperitoneum, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the retroperitoneum is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the retroperitoneum, the tumor is any size, any grade, and may have spread to nearby lymph nodes. Cancer has spread to other parts of the body, such as the lung. The cancer may come back in the same soft tissue or in other parts of the body. Different types of treatments are available for patients with soft tissue sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for soft tissue sarcoma. It may be the only treatment needed for small, low-grade tumors, especially in the trunk, arms, or legs. The following surgical procedures may be used: Radiation therapy or chemotherapy may be given before or after surgery to remove the tumor. When given before surgery, radiation therapy or chemotherapy will make the tumor smaller and reduce the amount of tissue that needs to be removed during surgery. Treatment given before surgery is called neoadjuvant therapy. When given after surgery to remove all of the tumor that can be seen, radiation therapy or chemotherapy will kill any remaining cancer cells. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy and internal radiation therapy may be used to treat soft tissue sarcoma. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Soft Tissue Sarcoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy may cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy. These include: For more information, see Drugs Approved for Soft Tissue Sarcoma. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. Some types of immune system cells, such as T cells, and some cancer cells have certain proteins, called checkpoint proteins, on their surface that keep immune responses in check. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. Types of immune checkpoint inhibitor therapy include the following: Ipilimumab is a type of CTLA-4 inhibitor that is being studied to treat soft tissue sarcoma. Pembrolizumab and nivolumab are PD-1 inhibitors that are used to treat progressive and recurrent soft tissue sarcoma. For more information, see Drugs Approved for Soft Tissue Sarcoma. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage I soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage II soft tissue sarcoma and stage III soft tissue sarcoma that has not spread to lymph nodes may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage III soft tissue sarcoma that has spread to lymph nodes (advanced) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage IV soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about soft tissue sarcomas, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult soft tissue sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Soft Tissue Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/soft-tissue-sarcoma/patient/adult-soft-tissue-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389216] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Soft Tissue Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Adult Soft Tissue Sarcoma ?

Key Points - Adult soft tissue sarcoma is a disease in which malignant (cancer) cells form in the soft tissues of the body. - Having certain inherited disorders can increase the risk of adult soft tissue sarcoma. - A sign of adult soft tissue sarcoma is a lump or swelling in soft tissue of the body. - Adult soft tissue sarcoma is diagnosed with a biopsy. - Certain factors affect treatment options and prognosis (chance of recovery). Adult soft tissue sarcoma is a disease in which malignant (cancer) cells form in the soft tissues of the body. The soft tissues of the body include the muscles, tendons (bands of fiber that connect muscles to bones), fat, blood vessels, lymph vessels, nerves, and tissues around joints. Adult soft tissue sarcomas can form almost anywhere in the body, but are most common in the head, neck, arms, legs, trunk, and abdomen. There are many types of soft tissue sarcoma. The cells of each type of sarcoma look different under a microscope, based on the type of soft tissue in which the cancer began. See the following PDQ summaries for more information on soft tissue sarcomas: - Childhood Soft Tissue Sarcoma Treatment - Ewing Sarcoma Family of Tumors Treatment - Gastrointestinal Stromal Tumors Treatment - Kaposi Sarcoma Treatment - Uterine Sarcoma Treatment

Soft tissues of the body connect, support, and surround other body parts and organs. The soft tissues of the body include the following: Soft tissue sarcomas can form almost anywhere in the body, including the head, neck, and trunk, but are most common in the arms, legs, abdomen, and retroperitoneum.EnlargeSoft tissue sarcoma forms in soft tissues of the body, including muscle, tendons, fat, blood vessels, lymph vessels, nerves, and tissue around joints. There are many types of soft tissue sarcoma. The cells of each type of sarcoma look different under a microscope, based on the type of soft tissue in which the cancer began. For more information about soft tissue sarcomas, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for soft tissue sarcoma include the following inherited disorders: Other risk factors for soft tissue sarcoma include the following: A sarcoma may appear as a painless lump under the skin, often on an arm or a leg. Sarcomas that begin in the abdomen may not cause signs or symptoms until they get very big. As the sarcoma grows and presses on nearby organs, nerves, muscles, or blood vessels, signs and symptoms may include: Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of these problems. If your doctor thinks you may have a soft tissue sarcoma, a biopsy will be done. The type of biopsy will be based on the size of the tumor and where it is in the body. These types of biopsies may be used: Careful planning of the biopsy should involve the surgeon, a radiation oncologist, and an interventional radiologist who uses medical imaging to guide diagnosis. Samples will be taken from the primary tumor, lymph nodes, and other suspicious areas. A pathologist views the tissue under a microscope to look for cancer cells and to find out the grade of the tumor. The grade of a tumor depends on how abnormal the cancer cells look under a microscope and how quickly the cells are dividing. High-grade tumors usually grow and spread more quickly than low-grade tumors. Because soft tissue sarcoma can be hard to diagnose, the tissue samples should be checked by a pathologist who has experience in diagnosing soft tissue sarcoma. The following tests may be done on the tissue that was removed: The treatment options and prognosis depend on the following: Small, low-grade tumors, especially in the trunk, arms, or legs, are frequently treated with surgery alone. High-grade sarcomas are more difficult to treat and more likely to spread. The process used to find out if cancer has spread within the soft tissue or to other parts of the body is called staging. Staging of soft tissue sarcoma is also based on the grade and size of the tumor, and whether it has spread to the lymph nodes or other parts of the body. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The results of these tests are viewed together with the results of the tumor biopsy to find out the stage of the soft tissue sarcoma before treatment is given. Sometimes chemotherapy or radiation therapy is given as the initial treatment and afterwards the soft tissue sarcoma is staged again. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if soft tissue sarcoma spreads to the lung, the cancer cells in the lung are actually soft tissue sarcoma cells. The disease is metastatic soft tissue sarcoma, not lung cancer. The grade of the tumor describes how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. Low grade, mid grade, and high grade are used to describe soft tissue sarcoma: Stage I soft tissue sarcoma of the trunk, arms, and legs is divided into stages IA and IB: In stage II soft tissue sarcoma of the trunk, arms, and legs, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the trunk, arms, and legs is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the trunk, arms, and legs, one of the following is found: Stage I soft tissue sarcoma of the retroperitoneum is divided into stages IA and IB: In stage II soft tissue sarcoma of the retroperitoneum, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the retroperitoneum is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the retroperitoneum, the tumor is any size, any grade, and may have spread to nearby lymph nodes. Cancer has spread to other parts of the body, such as the lung. The cancer may come back in the same soft tissue or in other parts of the body. Different types of treatments are available for patients with soft tissue sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for soft tissue sarcoma. It may be the only treatment needed for small, low-grade tumors, especially in the trunk, arms, or legs. The following surgical procedures may be used: Radiation therapy or chemotherapy may be given before or after surgery to remove the tumor. When given before surgery, radiation therapy or chemotherapy will make the tumor smaller and reduce the amount of tissue that needs to be removed during surgery. Treatment given before surgery is called neoadjuvant therapy. When given after surgery to remove all of the tumor that can be seen, radiation therapy or chemotherapy will kill any remaining cancer cells. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy and internal radiation therapy may be used to treat soft tissue sarcoma. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Soft Tissue Sarcoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy may cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy. These include: For more information, see Drugs Approved for Soft Tissue Sarcoma. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. Some types of immune system cells, such as T cells, and some cancer cells have certain proteins, called checkpoint proteins, on their surface that keep immune responses in check. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. Types of immune checkpoint inhibitor therapy include the following: Ipilimumab is a type of CTLA-4 inhibitor that is being studied to treat soft tissue sarcoma. Pembrolizumab and nivolumab are PD-1 inhibitors that are used to treat progressive and recurrent soft tissue sarcoma. For more information, see Drugs Approved for Soft Tissue Sarcoma. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage I soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage II soft tissue sarcoma and stage III soft tissue sarcoma that has not spread to lymph nodes may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage III soft tissue sarcoma that has spread to lymph nodes (advanced) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage IV soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about soft tissue sarcomas, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult soft tissue sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Soft Tissue Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/soft-tissue-sarcoma/patient/adult-soft-tissue-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389216] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Soft Tissue Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Adult Soft Tissue Sarcoma? ?

Having certain inherited disorders can increase the risk of adult soft tissue sarcoma. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn&apos;t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for soft tissue sarcoma include the following inherited disorders: - Retinoblastoma. - Neurofibromatosis type 1 (NF1; von Recklinghausen disease). - Tuberous sclerosis (Bourneville disease). - Familial adenomatous polyposis (FAP; Gardner syndrome). - Li-Fraumeni syndrome. - Werner syndrome (adult progeria). - Nevoid basal cell carcinoma syndrome (Gorlin syndrome). Other risk factors for soft tissue sarcoma include the following: - Past treatment with radiation therapy for certain cancers. - Being exposed to certain chemicals, such as Thorotrast (thorium dioxide), vinyl chloride, or arsenic. - Having swelling (lymphedema) in the arms or legs for a long time.

Soft tissues of the body connect, support, and surround other body parts and organs. The soft tissues of the body include the following: Soft tissue sarcomas can form almost anywhere in the body, including the head, neck, and trunk, but are most common in the arms, legs, abdomen, and retroperitoneum.EnlargeSoft tissue sarcoma forms in soft tissues of the body, including muscle, tendons, fat, blood vessels, lymph vessels, nerves, and tissue around joints. There are many types of soft tissue sarcoma. The cells of each type of sarcoma look different under a microscope, based on the type of soft tissue in which the cancer began. For more information about soft tissue sarcomas, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for soft tissue sarcoma include the following inherited disorders: Other risk factors for soft tissue sarcoma include the following: A sarcoma may appear as a painless lump under the skin, often on an arm or a leg. Sarcomas that begin in the abdomen may not cause signs or symptoms until they get very big. As the sarcoma grows and presses on nearby organs, nerves, muscles, or blood vessels, signs and symptoms may include: Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of these problems. If your doctor thinks you may have a soft tissue sarcoma, a biopsy will be done. The type of biopsy will be based on the size of the tumor and where it is in the body. These types of biopsies may be used: Careful planning of the biopsy should involve the surgeon, a radiation oncologist, and an interventional radiologist who uses medical imaging to guide diagnosis. Samples will be taken from the primary tumor, lymph nodes, and other suspicious areas. A pathologist views the tissue under a microscope to look for cancer cells and to find out the grade of the tumor. The grade of a tumor depends on how abnormal the cancer cells look under a microscope and how quickly the cells are dividing. High-grade tumors usually grow and spread more quickly than low-grade tumors. Because soft tissue sarcoma can be hard to diagnose, the tissue samples should be checked by a pathologist who has experience in diagnosing soft tissue sarcoma. The following tests may be done on the tissue that was removed: The treatment options and prognosis depend on the following: Small, low-grade tumors, especially in the trunk, arms, or legs, are frequently treated with surgery alone. High-grade sarcomas are more difficult to treat and more likely to spread. The process used to find out if cancer has spread within the soft tissue or to other parts of the body is called staging. Staging of soft tissue sarcoma is also based on the grade and size of the tumor, and whether it has spread to the lymph nodes or other parts of the body. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The results of these tests are viewed together with the results of the tumor biopsy to find out the stage of the soft tissue sarcoma before treatment is given. Sometimes chemotherapy or radiation therapy is given as the initial treatment and afterwards the soft tissue sarcoma is staged again. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if soft tissue sarcoma spreads to the lung, the cancer cells in the lung are actually soft tissue sarcoma cells. The disease is metastatic soft tissue sarcoma, not lung cancer. The grade of the tumor describes how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. Low grade, mid grade, and high grade are used to describe soft tissue sarcoma: Stage I soft tissue sarcoma of the trunk, arms, and legs is divided into stages IA and IB: In stage II soft tissue sarcoma of the trunk, arms, and legs, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the trunk, arms, and legs is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the trunk, arms, and legs, one of the following is found: Stage I soft tissue sarcoma of the retroperitoneum is divided into stages IA and IB: In stage II soft tissue sarcoma of the retroperitoneum, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the retroperitoneum is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the retroperitoneum, the tumor is any size, any grade, and may have spread to nearby lymph nodes. Cancer has spread to other parts of the body, such as the lung. The cancer may come back in the same soft tissue or in other parts of the body. Different types of treatments are available for patients with soft tissue sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for soft tissue sarcoma. It may be the only treatment needed for small, low-grade tumors, especially in the trunk, arms, or legs. The following surgical procedures may be used: Radiation therapy or chemotherapy may be given before or after surgery to remove the tumor. When given before surgery, radiation therapy or chemotherapy will make the tumor smaller and reduce the amount of tissue that needs to be removed during surgery. Treatment given before surgery is called neoadjuvant therapy. When given after surgery to remove all of the tumor that can be seen, radiation therapy or chemotherapy will kill any remaining cancer cells. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy and internal radiation therapy may be used to treat soft tissue sarcoma. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Soft Tissue Sarcoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy may cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy. These include: For more information, see Drugs Approved for Soft Tissue Sarcoma. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. Some types of immune system cells, such as T cells, and some cancer cells have certain proteins, called checkpoint proteins, on their surface that keep immune responses in check. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. Types of immune checkpoint inhibitor therapy include the following: Ipilimumab is a type of CTLA-4 inhibitor that is being studied to treat soft tissue sarcoma. Pembrolizumab and nivolumab are PD-1 inhibitors that are used to treat progressive and recurrent soft tissue sarcoma. For more information, see Drugs Approved for Soft Tissue Sarcoma. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage I soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage II soft tissue sarcoma and stage III soft tissue sarcoma that has not spread to lymph nodes may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage III soft tissue sarcoma that has spread to lymph nodes (advanced) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage IV soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about soft tissue sarcomas, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult soft tissue sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Soft Tissue Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/soft-tissue-sarcoma/patient/adult-soft-tissue-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389216] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Soft Tissue Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Adult Soft Tissue Sarcoma ?

A sign of adult soft tissue sarcoma is a lump or swelling in soft tissue of the body. A sarcoma may appear as a painless lump under the skin, often on an arm or a leg. Sarcomas that begin in the abdomen may not cause signs or symptoms until they get very big. As the sarcoma grows bigger and presses on nearby organs, nerves, muscles, or blood vessels, signs and symptoms may include: - Pain. - Trouble breathing. Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of these problems.

Soft tissues of the body connect, support, and surround other body parts and organs. The soft tissues of the body include the following: Soft tissue sarcomas can form almost anywhere in the body, including the head, neck, and trunk, but are most common in the arms, legs, abdomen, and retroperitoneum.EnlargeSoft tissue sarcoma forms in soft tissues of the body, including muscle, tendons, fat, blood vessels, lymph vessels, nerves, and tissue around joints. There are many types of soft tissue sarcoma. The cells of each type of sarcoma look different under a microscope, based on the type of soft tissue in which the cancer began. For more information about soft tissue sarcomas, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for soft tissue sarcoma include the following inherited disorders: Other risk factors for soft tissue sarcoma include the following: A sarcoma may appear as a painless lump under the skin, often on an arm or a leg. Sarcomas that begin in the abdomen may not cause signs or symptoms until they get very big. As the sarcoma grows and presses on nearby organs, nerves, muscles, or blood vessels, signs and symptoms may include: Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of these problems. If your doctor thinks you may have a soft tissue sarcoma, a biopsy will be done. The type of biopsy will be based on the size of the tumor and where it is in the body. These types of biopsies may be used: Careful planning of the biopsy should involve the surgeon, a radiation oncologist, and an interventional radiologist who uses medical imaging to guide diagnosis. Samples will be taken from the primary tumor, lymph nodes, and other suspicious areas. A pathologist views the tissue under a microscope to look for cancer cells and to find out the grade of the tumor. The grade of a tumor depends on how abnormal the cancer cells look under a microscope and how quickly the cells are dividing. High-grade tumors usually grow and spread more quickly than low-grade tumors. Because soft tissue sarcoma can be hard to diagnose, the tissue samples should be checked by a pathologist who has experience in diagnosing soft tissue sarcoma. The following tests may be done on the tissue that was removed: The treatment options and prognosis depend on the following: Small, low-grade tumors, especially in the trunk, arms, or legs, are frequently treated with surgery alone. High-grade sarcomas are more difficult to treat and more likely to spread. The process used to find out if cancer has spread within the soft tissue or to other parts of the body is called staging. Staging of soft tissue sarcoma is also based on the grade and size of the tumor, and whether it has spread to the lymph nodes or other parts of the body. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The results of these tests are viewed together with the results of the tumor biopsy to find out the stage of the soft tissue sarcoma before treatment is given. Sometimes chemotherapy or radiation therapy is given as the initial treatment and afterwards the soft tissue sarcoma is staged again. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if soft tissue sarcoma spreads to the lung, the cancer cells in the lung are actually soft tissue sarcoma cells. The disease is metastatic soft tissue sarcoma, not lung cancer. The grade of the tumor describes how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. Low grade, mid grade, and high grade are used to describe soft tissue sarcoma: Stage I soft tissue sarcoma of the trunk, arms, and legs is divided into stages IA and IB: In stage II soft tissue sarcoma of the trunk, arms, and legs, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the trunk, arms, and legs is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the trunk, arms, and legs, one of the following is found: Stage I soft tissue sarcoma of the retroperitoneum is divided into stages IA and IB: In stage II soft tissue sarcoma of the retroperitoneum, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the retroperitoneum is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the retroperitoneum, the tumor is any size, any grade, and may have spread to nearby lymph nodes. Cancer has spread to other parts of the body, such as the lung. The cancer may come back in the same soft tissue or in other parts of the body. Different types of treatments are available for patients with soft tissue sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for soft tissue sarcoma. It may be the only treatment needed for small, low-grade tumors, especially in the trunk, arms, or legs. The following surgical procedures may be used: Radiation therapy or chemotherapy may be given before or after surgery to remove the tumor. When given before surgery, radiation therapy or chemotherapy will make the tumor smaller and reduce the amount of tissue that needs to be removed during surgery. Treatment given before surgery is called neoadjuvant therapy. When given after surgery to remove all of the tumor that can be seen, radiation therapy or chemotherapy will kill any remaining cancer cells. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy and internal radiation therapy may be used to treat soft tissue sarcoma. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Soft Tissue Sarcoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy may cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy. These include: For more information, see Drugs Approved for Soft Tissue Sarcoma. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. Some types of immune system cells, such as T cells, and some cancer cells have certain proteins, called checkpoint proteins, on their surface that keep immune responses in check. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. Types of immune checkpoint inhibitor therapy include the following: Ipilimumab is a type of CTLA-4 inhibitor that is being studied to treat soft tissue sarcoma. Pembrolizumab and nivolumab are PD-1 inhibitors that are used to treat progressive and recurrent soft tissue sarcoma. For more information, see Drugs Approved for Soft Tissue Sarcoma. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage I soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage II soft tissue sarcoma and stage III soft tissue sarcoma that has not spread to lymph nodes may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage III soft tissue sarcoma that has spread to lymph nodes (advanced) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage IV soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about soft tissue sarcomas, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult soft tissue sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Soft Tissue Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/soft-tissue-sarcoma/patient/adult-soft-tissue-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389216] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Soft Tissue Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Adult Soft Tissue Sarcoma ?

Adult soft tissue sarcoma is diagnosed with a biopsy. If your doctor thinks you may have a soft tissue sarcoma, a biopsy will be done. The type of biopsy will be based on the size of the tumor and where it is in the body. There are three types of biopsy that may be used: - Incisional biopsy : The removal of part of a lump or a sample of tissue. - Core biopsy : The removal of tissue using a wide needle. - Excisional biopsy : The removal of an entire lump or area of tissue that doesnt look normal. Samples will be taken from the primary tumor, lymph nodes, and other suspicious areas. A pathologist views the tissue under a microscope to look for cancer cells and to find out the grade of the tumor. The grade of a tumor depends on how abnormal the cancer cells look under a microscope and how quickly the cells are dividing. High-grade tumors usually grow and spread more quickly than low-grade tumors. Because soft tissue sarcoma can be hard to diagnose, patients should ask to have tissue samples checked by a pathologist who has experience in diagnosing soft tissue sarcoma. The following tests may be done on the tissue that was removed: - Immunohistochemistry : A test that uses antibodies to check for certain antigens in a sample of tissue. The antibody is usually linked to a radioactive substance or a dye that causes the tissue to light up under a microscope. This type of test may be used to tell the difference between different types of cancer. - Light and electron microscopy : A laboratory test in which cells in a sample of tissue are viewed under regular and high-powered microscopes to look for certain changes in the cells. - Cytogenetic analysis : A laboratory test in which cells in a sample of tissue are viewed under a microscope to look for certain changes in the chromosomes. - FISH (fluorescence in situ hybridization): A laboratory test used to look at genes or chromosomes in cells and tissues. Pieces of DNA that contain a fluorescent dye are made in the laboratory and added to cells or tissues on a glass slide. When these pieces of DNA attach to certain genes or areas of chromosomes on the slide, they light up when viewed under a microscope with a special light. - Flow cytometry : A laboratory test that measures the number of cells in a sample, the percentage of live cells in a sample, and certain characteristics of cells, such as size, shape, and the presence of tumor markers on the cell surface. The cells are stained with a light-sensitive dye, placed in a fluid, and passed in a stream before a laser or other type of light. The measurements are based on how the light-sensitive dye reacts to the light.

Soft tissues of the body connect, support, and surround other body parts and organs. The soft tissues of the body include the following: Soft tissue sarcomas can form almost anywhere in the body, including the head, neck, and trunk, but are most common in the arms, legs, abdomen, and retroperitoneum.EnlargeSoft tissue sarcoma forms in soft tissues of the body, including muscle, tendons, fat, blood vessels, lymph vessels, nerves, and tissue around joints. There are many types of soft tissue sarcoma. The cells of each type of sarcoma look different under a microscope, based on the type of soft tissue in which the cancer began. For more information about soft tissue sarcomas, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for soft tissue sarcoma include the following inherited disorders: Other risk factors for soft tissue sarcoma include the following: A sarcoma may appear as a painless lump under the skin, often on an arm or a leg. Sarcomas that begin in the abdomen may not cause signs or symptoms until they get very big. As the sarcoma grows and presses on nearby organs, nerves, muscles, or blood vessels, signs and symptoms may include: Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of these problems. If your doctor thinks you may have a soft tissue sarcoma, a biopsy will be done. The type of biopsy will be based on the size of the tumor and where it is in the body. These types of biopsies may be used: Careful planning of the biopsy should involve the surgeon, a radiation oncologist, and an interventional radiologist who uses medical imaging to guide diagnosis. Samples will be taken from the primary tumor, lymph nodes, and other suspicious areas. A pathologist views the tissue under a microscope to look for cancer cells and to find out the grade of the tumor. The grade of a tumor depends on how abnormal the cancer cells look under a microscope and how quickly the cells are dividing. High-grade tumors usually grow and spread more quickly than low-grade tumors. Because soft tissue sarcoma can be hard to diagnose, the tissue samples should be checked by a pathologist who has experience in diagnosing soft tissue sarcoma. The following tests may be done on the tissue that was removed: The treatment options and prognosis depend on the following: Small, low-grade tumors, especially in the trunk, arms, or legs, are frequently treated with surgery alone. High-grade sarcomas are more difficult to treat and more likely to spread. The process used to find out if cancer has spread within the soft tissue or to other parts of the body is called staging. Staging of soft tissue sarcoma is also based on the grade and size of the tumor, and whether it has spread to the lymph nodes or other parts of the body. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The results of these tests are viewed together with the results of the tumor biopsy to find out the stage of the soft tissue sarcoma before treatment is given. Sometimes chemotherapy or radiation therapy is given as the initial treatment and afterwards the soft tissue sarcoma is staged again. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if soft tissue sarcoma spreads to the lung, the cancer cells in the lung are actually soft tissue sarcoma cells. The disease is metastatic soft tissue sarcoma, not lung cancer. The grade of the tumor describes how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. Low grade, mid grade, and high grade are used to describe soft tissue sarcoma: Stage I soft tissue sarcoma of the trunk, arms, and legs is divided into stages IA and IB: In stage II soft tissue sarcoma of the trunk, arms, and legs, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the trunk, arms, and legs is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the trunk, arms, and legs, one of the following is found: Stage I soft tissue sarcoma of the retroperitoneum is divided into stages IA and IB: In stage II soft tissue sarcoma of the retroperitoneum, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the retroperitoneum is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the retroperitoneum, the tumor is any size, any grade, and may have spread to nearby lymph nodes. Cancer has spread to other parts of the body, such as the lung. The cancer may come back in the same soft tissue or in other parts of the body. Different types of treatments are available for patients with soft tissue sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for soft tissue sarcoma. It may be the only treatment needed for small, low-grade tumors, especially in the trunk, arms, or legs. The following surgical procedures may be used: Radiation therapy or chemotherapy may be given before or after surgery to remove the tumor. When given before surgery, radiation therapy or chemotherapy will make the tumor smaller and reduce the amount of tissue that needs to be removed during surgery. Treatment given before surgery is called neoadjuvant therapy. When given after surgery to remove all of the tumor that can be seen, radiation therapy or chemotherapy will kill any remaining cancer cells. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy and internal radiation therapy may be used to treat soft tissue sarcoma. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Soft Tissue Sarcoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy may cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy. These include: For more information, see Drugs Approved for Soft Tissue Sarcoma. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. Some types of immune system cells, such as T cells, and some cancer cells have certain proteins, called checkpoint proteins, on their surface that keep immune responses in check. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. Types of immune checkpoint inhibitor therapy include the following: Ipilimumab is a type of CTLA-4 inhibitor that is being studied to treat soft tissue sarcoma. Pembrolizumab and nivolumab are PD-1 inhibitors that are used to treat progressive and recurrent soft tissue sarcoma. For more information, see Drugs Approved for Soft Tissue Sarcoma. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage I soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage II soft tissue sarcoma and stage III soft tissue sarcoma that has not spread to lymph nodes may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage III soft tissue sarcoma that has spread to lymph nodes (advanced) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage IV soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about soft tissue sarcomas, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult soft tissue sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Soft Tissue Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/soft-tissue-sarcoma/patient/adult-soft-tissue-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389216] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Soft Tissue Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Adult Soft Tissue Sarcoma ?

Certain factors affect treatment options and prognosis (chance of recovery). The treatment options and prognosis (chance of recovery) depend on the following: - The type of soft tissue sarcoma. - The size, grade, and stage of the tumor. - How fast the cancer cells are growing and dividing. - Where the tumor is in the body. - Whether all of the tumor is removed by surgery. - The patient&apos;s age and general health. - Whether the cancer has recurred (come back).

Soft tissues of the body connect, support, and surround other body parts and organs. The soft tissues of the body include the following: Soft tissue sarcomas can form almost anywhere in the body, including the head, neck, and trunk, but are most common in the arms, legs, abdomen, and retroperitoneum.EnlargeSoft tissue sarcoma forms in soft tissues of the body, including muscle, tendons, fat, blood vessels, lymph vessels, nerves, and tissue around joints. There are many types of soft tissue sarcoma. The cells of each type of sarcoma look different under a microscope, based on the type of soft tissue in which the cancer began. For more information about soft tissue sarcomas, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for soft tissue sarcoma include the following inherited disorders: Other risk factors for soft tissue sarcoma include the following: A sarcoma may appear as a painless lump under the skin, often on an arm or a leg. Sarcomas that begin in the abdomen may not cause signs or symptoms until they get very big. As the sarcoma grows and presses on nearby organs, nerves, muscles, or blood vessels, signs and symptoms may include: Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of these problems. If your doctor thinks you may have a soft tissue sarcoma, a biopsy will be done. The type of biopsy will be based on the size of the tumor and where it is in the body. These types of biopsies may be used: Careful planning of the biopsy should involve the surgeon, a radiation oncologist, and an interventional radiologist who uses medical imaging to guide diagnosis. Samples will be taken from the primary tumor, lymph nodes, and other suspicious areas. A pathologist views the tissue under a microscope to look for cancer cells and to find out the grade of the tumor. The grade of a tumor depends on how abnormal the cancer cells look under a microscope and how quickly the cells are dividing. High-grade tumors usually grow and spread more quickly than low-grade tumors. Because soft tissue sarcoma can be hard to diagnose, the tissue samples should be checked by a pathologist who has experience in diagnosing soft tissue sarcoma. The following tests may be done on the tissue that was removed: The treatment options and prognosis depend on the following: Small, low-grade tumors, especially in the trunk, arms, or legs, are frequently treated with surgery alone. High-grade sarcomas are more difficult to treat and more likely to spread. The process used to find out if cancer has spread within the soft tissue or to other parts of the body is called staging. Staging of soft tissue sarcoma is also based on the grade and size of the tumor, and whether it has spread to the lymph nodes or other parts of the body. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The results of these tests are viewed together with the results of the tumor biopsy to find out the stage of the soft tissue sarcoma before treatment is given. Sometimes chemotherapy or radiation therapy is given as the initial treatment and afterwards the soft tissue sarcoma is staged again. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if soft tissue sarcoma spreads to the lung, the cancer cells in the lung are actually soft tissue sarcoma cells. The disease is metastatic soft tissue sarcoma, not lung cancer. The grade of the tumor describes how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. Low grade, mid grade, and high grade are used to describe soft tissue sarcoma: Stage I soft tissue sarcoma of the trunk, arms, and legs is divided into stages IA and IB: In stage II soft tissue sarcoma of the trunk, arms, and legs, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the trunk, arms, and legs is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the trunk, arms, and legs, one of the following is found: Stage I soft tissue sarcoma of the retroperitoneum is divided into stages IA and IB: In stage II soft tissue sarcoma of the retroperitoneum, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the retroperitoneum is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the retroperitoneum, the tumor is any size, any grade, and may have spread to nearby lymph nodes. Cancer has spread to other parts of the body, such as the lung. The cancer may come back in the same soft tissue or in other parts of the body. Different types of treatments are available for patients with soft tissue sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for soft tissue sarcoma. It may be the only treatment needed for small, low-grade tumors, especially in the trunk, arms, or legs. The following surgical procedures may be used: Radiation therapy or chemotherapy may be given before or after surgery to remove the tumor. When given before surgery, radiation therapy or chemotherapy will make the tumor smaller and reduce the amount of tissue that needs to be removed during surgery. Treatment given before surgery is called neoadjuvant therapy. When given after surgery to remove all of the tumor that can be seen, radiation therapy or chemotherapy will kill any remaining cancer cells. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy and internal radiation therapy may be used to treat soft tissue sarcoma. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Soft Tissue Sarcoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy may cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy. These include: For more information, see Drugs Approved for Soft Tissue Sarcoma. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. Some types of immune system cells, such as T cells, and some cancer cells have certain proteins, called checkpoint proteins, on their surface that keep immune responses in check. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. Types of immune checkpoint inhibitor therapy include the following: Ipilimumab is a type of CTLA-4 inhibitor that is being studied to treat soft tissue sarcoma. Pembrolizumab and nivolumab are PD-1 inhibitors that are used to treat progressive and recurrent soft tissue sarcoma. For more information, see Drugs Approved for Soft Tissue Sarcoma. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage I soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage II soft tissue sarcoma and stage III soft tissue sarcoma that has not spread to lymph nodes may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage III soft tissue sarcoma that has spread to lymph nodes (advanced) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage IV soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about soft tissue sarcomas, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult soft tissue sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. 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What are the stages of Adult Soft Tissue Sarcoma ?

Key Points - After adult soft tissue sarcoma has been diagnosed, tests are done to find out if cancer cells have spread within the soft tissue or to other parts of the body. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - The following stages are used for adult soft tissue sarcoma: - Stage I - Stage II - Stage III - Stage IV After adult soft tissue sarcoma has been diagnosed, tests are done to find out if cancer cells have spread within the soft tissue or to other parts of the body. The process used to find out if cancer has spread within the soft tissue or to other parts of the body is called staging. Staging of soft tissue sarcoma is also based on the grade and size of the tumor, whether it is superficial (close to the skin&apos;s surface) or deep, and whether it has spread to the lymph nodes or other parts of the body. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Chest x-ray : An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. - Blood chemistry studies : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease. - Complete blood count (CBC): A procedure in which a sample of blood is drawn and checked for the following: - The number of red blood cells, white blood cells, and platelets. - The amount of hemoglobin (the protein that carries oxygen) in the red blood cells. - The portion of the blood sample made up of red blood cells. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside of the body, such as the lung and abdomen, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). - PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. The results of these tests are viewed together with the results of the tumor biopsy to find out the stage of the soft tissue sarcoma before treatment is given. Sometimes chemotherapy or radiation therapy is given as the initial treatment and afterwards the soft tissue sarcoma is staged again. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if soft tissue sarcoma spreads to the lung, the cancer cells in the lung are actually soft tissue sarcoma cells. The disease is metastatic soft tissue sarcoma, not lung cancer. The following stages are used for adult soft tissue sarcoma: Stage I Stage I is divided into stages IA and IB: - In stage IA, the tumor is low-grade (likely to grow and spread slowly) and 5 centimeters or smaller. It may be either superficial (in subcutaneous tissue with no spread into connective tissue or muscle below) or deep (in the muscle and may be in connective or subcutaneous tissue). - In stage IB, the tumor is low-grade (likely to grow and spread slowly) and larger than 5 centimeters. It may be either superficial (in subcutaneous tissue with no spread into connective tissue or muscle below) or deep (in the muscle and may be in connective or subcutaneous tissue). Stage II Stage II is divided into stages IIA and IIB: - In stage IIA, the tumor is mid-grade (somewhat likely to grow and spread quickly) or high-grade (likely to grow and spread quickly) and 5 centimeters or smaller. It may be either superficial (in subcutaneous tissue with no spread into connective tissue or muscle below) or deep (in the muscle and may be in connective or subcutaneous tissue). - In stage IIB, the tumor is mid-grade (somewhat likely to grow and spread quickly) and larger than 5 centimeters. It may be either superficial (in subcutaneous tissue with no spread into connective tissue or muscle below) or deep (in the muscle and may be in connective or subcutaneous tissue). Stage III In stage III, the tumor is either: - high-grade (likely to grow and spread quickly), larger than 5 centimeters, and either superficial (in subcutaneous tissue with no spread into connective tissue or muscle below) or deep (in the muscle and may be in connective or subcutaneous tissue); or - any grade, any size, and has spread to nearby lymph nodes. Stage III cancer that has spread to the lymph nodes is advanced stage III. Stage IV In stage IV, the tumor is any grade, any size, and may have spread to nearby lymph nodes. Cancer has spread to distant parts of the body, such as the lungs.

Soft tissues of the body connect, support, and surround other body parts and organs. The soft tissues of the body include the following: Soft tissue sarcomas can form almost anywhere in the body, including the head, neck, and trunk, but are most common in the arms, legs, abdomen, and retroperitoneum.EnlargeSoft tissue sarcoma forms in soft tissues of the body, including muscle, tendons, fat, blood vessels, lymph vessels, nerves, and tissue around joints. There are many types of soft tissue sarcoma. The cells of each type of sarcoma look different under a microscope, based on the type of soft tissue in which the cancer began. For more information about soft tissue sarcomas, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for soft tissue sarcoma include the following inherited disorders: Other risk factors for soft tissue sarcoma include the following: A sarcoma may appear as a painless lump under the skin, often on an arm or a leg. Sarcomas that begin in the abdomen may not cause signs or symptoms until they get very big. As the sarcoma grows and presses on nearby organs, nerves, muscles, or blood vessels, signs and symptoms may include: Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of these problems. If your doctor thinks you may have a soft tissue sarcoma, a biopsy will be done. The type of biopsy will be based on the size of the tumor and where it is in the body. These types of biopsies may be used: Careful planning of the biopsy should involve the surgeon, a radiation oncologist, and an interventional radiologist who uses medical imaging to guide diagnosis. Samples will be taken from the primary tumor, lymph nodes, and other suspicious areas. A pathologist views the tissue under a microscope to look for cancer cells and to find out the grade of the tumor. The grade of a tumor depends on how abnormal the cancer cells look under a microscope and how quickly the cells are dividing. High-grade tumors usually grow and spread more quickly than low-grade tumors. Because soft tissue sarcoma can be hard to diagnose, the tissue samples should be checked by a pathologist who has experience in diagnosing soft tissue sarcoma. The following tests may be done on the tissue that was removed: The treatment options and prognosis depend on the following: Small, low-grade tumors, especially in the trunk, arms, or legs, are frequently treated with surgery alone. High-grade sarcomas are more difficult to treat and more likely to spread. The process used to find out if cancer has spread within the soft tissue or to other parts of the body is called staging. Staging of soft tissue sarcoma is also based on the grade and size of the tumor, and whether it has spread to the lymph nodes or other parts of the body. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The results of these tests are viewed together with the results of the tumor biopsy to find out the stage of the soft tissue sarcoma before treatment is given. Sometimes chemotherapy or radiation therapy is given as the initial treatment and afterwards the soft tissue sarcoma is staged again. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if soft tissue sarcoma spreads to the lung, the cancer cells in the lung are actually soft tissue sarcoma cells. The disease is metastatic soft tissue sarcoma, not lung cancer. The grade of the tumor describes how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. Low grade, mid grade, and high grade are used to describe soft tissue sarcoma: Stage I soft tissue sarcoma of the trunk, arms, and legs is divided into stages IA and IB: In stage II soft tissue sarcoma of the trunk, arms, and legs, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the trunk, arms, and legs is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the trunk, arms, and legs, one of the following is found: Stage I soft tissue sarcoma of the retroperitoneum is divided into stages IA and IB: In stage II soft tissue sarcoma of the retroperitoneum, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the retroperitoneum is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the retroperitoneum, the tumor is any size, any grade, and may have spread to nearby lymph nodes. Cancer has spread to other parts of the body, such as the lung. The cancer may come back in the same soft tissue or in other parts of the body. Different types of treatments are available for patients with soft tissue sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for soft tissue sarcoma. It may be the only treatment needed for small, low-grade tumors, especially in the trunk, arms, or legs. The following surgical procedures may be used: Radiation therapy or chemotherapy may be given before or after surgery to remove the tumor. When given before surgery, radiation therapy or chemotherapy will make the tumor smaller and reduce the amount of tissue that needs to be removed during surgery. Treatment given before surgery is called neoadjuvant therapy. When given after surgery to remove all of the tumor that can be seen, radiation therapy or chemotherapy will kill any remaining cancer cells. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy and internal radiation therapy may be used to treat soft tissue sarcoma. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Soft Tissue Sarcoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy may cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy. These include: For more information, see Drugs Approved for Soft Tissue Sarcoma. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. Some types of immune system cells, such as T cells, and some cancer cells have certain proteins, called checkpoint proteins, on their surface that keep immune responses in check. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. Types of immune checkpoint inhibitor therapy include the following: Ipilimumab is a type of CTLA-4 inhibitor that is being studied to treat soft tissue sarcoma. Pembrolizumab and nivolumab are PD-1 inhibitors that are used to treat progressive and recurrent soft tissue sarcoma. For more information, see Drugs Approved for Soft Tissue Sarcoma. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage I soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage II soft tissue sarcoma and stage III soft tissue sarcoma that has not spread to lymph nodes may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage III soft tissue sarcoma that has spread to lymph nodes (advanced) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage IV soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about soft tissue sarcomas, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult soft tissue sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Soft Tissue Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/soft-tissue-sarcoma/patient/adult-soft-tissue-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389216] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Soft Tissue Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Adult Soft Tissue Sarcoma ?

Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Soft tissues of the body connect, support, and surround other body parts and organs. The soft tissues of the body include the following: Soft tissue sarcomas can form almost anywhere in the body, including the head, neck, and trunk, but are most common in the arms, legs, abdomen, and retroperitoneum.EnlargeSoft tissue sarcoma forms in soft tissues of the body, including muscle, tendons, fat, blood vessels, lymph vessels, nerves, and tissue around joints. There are many types of soft tissue sarcoma. The cells of each type of sarcoma look different under a microscope, based on the type of soft tissue in which the cancer began. For more information about soft tissue sarcomas, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for soft tissue sarcoma include the following inherited disorders: Other risk factors for soft tissue sarcoma include the following: A sarcoma may appear as a painless lump under the skin, often on an arm or a leg. Sarcomas that begin in the abdomen may not cause signs or symptoms until they get very big. As the sarcoma grows and presses on nearby organs, nerves, muscles, or blood vessels, signs and symptoms may include: Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of these problems. If your doctor thinks you may have a soft tissue sarcoma, a biopsy will be done. The type of biopsy will be based on the size of the tumor and where it is in the body. These types of biopsies may be used: Careful planning of the biopsy should involve the surgeon, a radiation oncologist, and an interventional radiologist who uses medical imaging to guide diagnosis. Samples will be taken from the primary tumor, lymph nodes, and other suspicious areas. A pathologist views the tissue under a microscope to look for cancer cells and to find out the grade of the tumor. The grade of a tumor depends on how abnormal the cancer cells look under a microscope and how quickly the cells are dividing. High-grade tumors usually grow and spread more quickly than low-grade tumors. Because soft tissue sarcoma can be hard to diagnose, the tissue samples should be checked by a pathologist who has experience in diagnosing soft tissue sarcoma. The following tests may be done on the tissue that was removed: The treatment options and prognosis depend on the following: Small, low-grade tumors, especially in the trunk, arms, or legs, are frequently treated with surgery alone. High-grade sarcomas are more difficult to treat and more likely to spread. The process used to find out if cancer has spread within the soft tissue or to other parts of the body is called staging. Staging of soft tissue sarcoma is also based on the grade and size of the tumor, and whether it has spread to the lymph nodes or other parts of the body. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: The results of these tests are viewed together with the results of the tumor biopsy to find out the stage of the soft tissue sarcoma before treatment is given. Sometimes chemotherapy or radiation therapy is given as the initial treatment and afterwards the soft tissue sarcoma is staged again. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if soft tissue sarcoma spreads to the lung, the cancer cells in the lung are actually soft tissue sarcoma cells. The disease is metastatic soft tissue sarcoma, not lung cancer. The grade of the tumor describes how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. Low grade, mid grade, and high grade are used to describe soft tissue sarcoma: Stage I soft tissue sarcoma of the trunk, arms, and legs is divided into stages IA and IB: In stage II soft tissue sarcoma of the trunk, arms, and legs, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the trunk, arms, and legs is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the trunk, arms, and legs, one of the following is found: Stage I soft tissue sarcoma of the retroperitoneum is divided into stages IA and IB: In stage II soft tissue sarcoma of the retroperitoneum, the tumor is 5 centimeters or smaller and is mid grade or high grade. Stage III soft tissue sarcoma of the retroperitoneum is divided into stages IIIA and IIIB: In stage IV soft tissue sarcoma of the retroperitoneum, the tumor is any size, any grade, and may have spread to nearby lymph nodes. Cancer has spread to other parts of the body, such as the lung. The cancer may come back in the same soft tissue or in other parts of the body. Different types of treatments are available for patients with soft tissue sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for soft tissue sarcoma. It may be the only treatment needed for small, low-grade tumors, especially in the trunk, arms, or legs. The following surgical procedures may be used: Radiation therapy or chemotherapy may be given before or after surgery to remove the tumor. When given before surgery, radiation therapy or chemotherapy will make the tumor smaller and reduce the amount of tissue that needs to be removed during surgery. Treatment given before surgery is called neoadjuvant therapy. When given after surgery to remove all of the tumor that can be seen, radiation therapy or chemotherapy will kill any remaining cancer cells. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy and internal radiation therapy may be used to treat soft tissue sarcoma. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Soft Tissue Sarcoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy may cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy. These include: For more information, see Drugs Approved for Soft Tissue Sarcoma. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy is a type of immunotherapy. Some types of immune system cells, such as T cells, and some cancer cells have certain proteins, called checkpoint proteins, on their surface that keep immune responses in check. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. Types of immune checkpoint inhibitor therapy include the following: Ipilimumab is a type of CTLA-4 inhibitor that is being studied to treat soft tissue sarcoma. Pembrolizumab and nivolumab are PD-1 inhibitors that are used to treat progressive and recurrent soft tissue sarcoma. For more information, see Drugs Approved for Soft Tissue Sarcoma. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage I soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage II soft tissue sarcoma and stage III soft tissue sarcoma that has not spread to lymph nodes may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage III soft tissue sarcoma that has spread to lymph nodes (advanced) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of stage IV soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent soft tissue sarcoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about soft tissue sarcomas, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult soft tissue sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Soft Tissue Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/soft-tissue-sarcoma/patient/adult-soft-tissue-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389216] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Soft Tissue Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Adult Soft Tissue Sarcoma ?

Key Points - There are different types of treatment for patients with adult soft tissue sarcoma. - Three types of standard treatment are used: - Surgery - Radiation therapy - Chemotherapy - New types of treatment are being tested in clinical trials. - Regional chemotherapy - Treatment for adult soft tissue sarcoma may cause side effects. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with adult soft tissue sarcoma. Different types of treatments are available for patients with adult soft tissue sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Three types of standard treatment are used: Surgery Surgery is the most common treatment for adult soft tissue sarcoma. For some soft-tissue sarcomas, removal of the tumor in surgery may be the only treatment needed. The following surgical procedures may be used: - Mohs microsurgery: A procedure in which the tumor is cut from the skin in thin layers. During surgery, the edges of the tumor and each layer of tumor removed are viewed through a microscope to check for cancer cells. Layers continue to be removed until no more cancer cells are seen. This type of surgery removes as little normal tissue as possible and is often used where appearance is important, such as on the skin. - Wide local excision: Removal of the tumor along with some normal tissue around it. For tumors of the head, neck, abdomen, and trunk, as little normal tissue as possible is removed. - Limb-sparing surgery: Removal of the tumor in an arm or leg without amputation, so the use and appearance of the limb is saved. Radiation therapy or chemotherapy may be given first to shrink the tumor. The tumor is then removed in a wide local excision. Tissue and bone that are removed may be replaced with a graft using tissue and bone taken from another part of the patient&apos;s body, or with an implant such as artificial bone. - Amputation: Surgery to remove part or all of a limb or appendage, such as an arm or leg. Amputation is rarely used to treat soft tissue sarcoma of the arm or leg. - Lymphadenectomy: A surgical procedure in which lymph nodes are removed and a sample of tissue is checked under a microscope for signs of cancer. This procedure is also called a lymph node dissection. Radiation therapy or chemotherapy may be given before or after surgery to remove the tumor. When given before surgery, radiation therapy or chemotherapy will make the tumor smaller and reduce the amount of tissue that needs to be removed during surgery. Treatment given before surgery is called neoadjuvant therapy. When given after surgery, radiation therapy or chemotherapy will kill any remaining cancer cells. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. Intensity-modulated radiation therapy (IMRT) is a type of 3-dimensional (3-D) radiation therapy that uses a computer to make pictures of the size and shape of the tumor. Thin beams of radiation of different intensities (strengths) are aimed at the tumor from many angles. This type of external radiation therapy causes less damage to nearby healthy tissue and is less likely to cause dry mouth, trouble swallowing, and damage to the skin. The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy and internal radiation therapy may be used to treat adult soft tissue sarcoma. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Soft Tissue Sarcoma for more information. New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Regional chemotherapy Clinical trials are studying ways to improve the effect of chemotherapy on tumor cells, including the following: - Regional hyperthermia therapy: A treatment in which tissue around the tumor is exposed to high temperatures to damage and kill cancer cells or to make cancer cells more sensitive to chemotherapy. - Isolated limb perfusion: A procedure that sends chemotherapy directly to an arm or leg in which the cancer has formed. The flow of blood to and from the limb is temporarily stopped with a tourniquet, and anticancer drugs are put directly into the blood of the limb. This sends a high dose of drugs to the tumor. Treatment for adult soft tissue sarcoma may cause side effects. For information about side effects caused by treatment for cancer, see our Side Effects page. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Adult Soft Tissue Sarcoma Stage I Adult Soft Tissue Sarcoma Treatment of stage I soft tissue sarcoma may include the following: - Surgery to remove the tumor, such as Mohs microsurgery for small sarcomas of the skin, wide local excision, or limb-sparing surgery. - Radiation therapy before and/or after surgery. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I adult soft tissue sarcoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage II Adult Soft Tissue Sarcoma and Stage III Adult Soft Tissue Sarcoma That Has Not Spread to Lymph Nodes Treatment of stage II adult soft tissue sarcoma and stage III adult soft tissue sarcoma that has not spread to lymph nodes may include the following: - Surgery to remove the tumor, such as wide local excision or limb-sparing surgery. - Radiation therapy before or after surgery. - Radiation therapy or chemotherapy before limb-sparing surgery. Radiation therapy may also be given after surgery. - High-dose radiation therapy for tumors that cannot be removed by surgery. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage II adult soft tissue sarcoma and stage III adult soft tissue sarcoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage III Adult Soft Tissue Sarcoma That Has Spread to Lymph Nodes (Advanced) Treatment of stage III adult soft tissue sarcoma that has spread to lymph nodes (advanced) may include the following: - Surgery (wide local excision) with lymphadenectomy. Radiation therapy may also be given after surgery. - A clinical trial of surgery followed by chemotherapy. - A clinical trial of regional hyperthermia therapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage III adult soft tissue sarcoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IV Adult Soft Tissue Sarcoma Treatment of stage IV adult soft tissue sarcoma may include the following: - Chemotherapy. - Surgery to remove cancer that has spread to the lungs. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage IV adult soft tissue sarcoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

Childhood acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Cancers that are acute usually get worse quickly if they are not treated. Cancers that are chronic usually get worse slowly. This summary is about the treatment of childhood AML, transient abnormal myelopoiesis, childhood acute promyelocytic leukemia, juvenile myelomonocytic leukemia, childhood chronic myelogenous leukemia, and childhood myelodysplastic syndromes. For information about the treatment of childhood acute lymphoblastic leukemia, see Childhood Acute Lymphoblastic Leukemia Treatment. In healthy children, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a type of white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts, or leukemia cells, in AML are abnormal and do not become healthy white blood cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This may lead to infection, anemia, or easy bleeding. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called granulocytic sarcoma or chloroma. TAM is a disorder of the bone marrow that can develop in newborns who have Down syndrome. TAM usually goes away on its own within the first 3 months of life. Infants who have TAM have an increased chance of developing AML before the age of 3 years. TAM is also called transient myeloproliferative disorder or transient leukemia. APL is a subtype of AML. In APL, some genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. The promyelocytes (leukemia cells) can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. Problems with severe bleeding and blood clots may also occur. This is a serious health problem that needs treatment as soon as possible. JMML is a rare childhood cancer that is most common in children around the age of 2 years and is more common in boys. In JMML, too many myeloid blood stem cells become myelocytes and monocytes (two types of white blood cells). Some of these myeloid blood stem cells never become mature white blood cells. These immature cells, called blasts, are unable to do their usual work. Over time, the myelocytes, monocytes, and blasts crowd out the healthy white blood cells, red blood cells, and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. CML often begins in an early myeloid blood cell when a certain gene change occurs. A section of genes, that includes the ABL gene, on chromosome 9 changes place with a section of genes on chromosome 22, which has the BCR gene. This makes a very short chromosome 22 (called the Philadelphia chromosome) and a very long chromosome 9. An abnormal BCR-ABL gene is formed on chromosome 22. The BCR-ABL gene tells the blood cells to make too much of a protein called tyrosine kinase. Tyrosine kinase causes too many abnormal white blood cells (leukemia cells) to be made in the bone marrow. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This can lead to infection, anemia, or easy bleeding. CML is rare in children.EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. MDS occur less often in children than in adults. In MDS, the bone marrow makes too few red blood cells, white blood cells, and platelets. These blood cells may not mature and enter the blood. The type of MDS depends on the type of blood cell that is affected. The treatment for MDS depends on how low the numbers of red blood cells, white blood cells, or platelets are. Over time, MDS may become AML. Cancer treatment with certain chemotherapy drugs and/or radiation therapy may cause therapy-related AML (t-AML) or therapy-related MDS (t-MDS). The risk of these therapy-related myeloid diseases depends on the total dose of the chemotherapy drugs used and the radiation dose and treatment field. Some patients also have an inherited risk for t-AML and t-MDS. These therapy-related diseases usually occur within 7 years after treatment, but are rare in children. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. These and other factors may increase the risk of childhood AML, APL, JMML, CML, and MDS: These and other signs and symptoms may be caused by childhood AML, APL, JMML, CML, or MDS or by other conditions. Check with a doctor if your child has any of the following: The signs and symptoms of TAM may include the following: Sometimes TAM does not cause any symptoms at all and is diagnosed after a routine blood test. The following tests and procedures may be used: The following test is a type of cytogenetic analysis: The prognosis and treatment options for childhood acute myeloid leukemia (AML) depend on the following: The prognosis and treatment options for childhood acute promyelocytic leukemia (APL) depends on the following: The prognosis and treatment options for juvenile myelomonocytic leukemia (JMML) depend on the following: The prognosis and treatment options for childhood chronic myelogenous leukemia (CML) depend on the following: The prognosis and treatment options for myelodysplastic syndromes (MDS) depend on the following: The extent or spread of cancer is usually described as stages. Instead of stages, treatment is based on one or more of the following: Newly diagnosed childhood AML Newly diagnosed childhood AML is cancer that has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and has one of the following: or Childhood AML in remission In childhood AML in remission, the disease has been treated and the following are found: Refractory leukemia is cancer that does not respond to treatment. Recurrent leukemia is cancer that has recurred (come back) after it has been treated. The cancer may come back in the blood and bone marrow or in other parts of the body, such as the central nervous system (brain and spinal cord). Different types of treatment are available for children with acute myeloid leukemia (AML), transient abnormal myelopoiesis (TAM), acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia (JMML), chronic myelogenous leukemia (CML), and myelodysplastic syndromes (MDS). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because AML and other myeloid disorders are rare in children, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not yet started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists: The treatment of childhood AML is done in phases: Treatment called central nervous system (CNS) prophylaxis therapy may be given during the induction phase of therapy. Because standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the leukemia cells are able to hide in the CNS. Intrathecal chemotherapy is able to reach leukemia cells in the CNS. It is given to kill the leukemia cells and lessen the chance the leukemia will recur (come back). The treatment of childhood APL includes a third phase called maintenance. The goal of maintenance is to kill any remaining leukemia cells that may regrow and cause a relapse. Often the cancer treatments are given in lower doses than those used during the remission induction and consolidation/intensification phases. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one chemotherapy drug. The way the chemotherapy is given depends on the type of cancer being treated. In AML, chemotherapy given by mouth, vein, or into the cerebrospinal fluid is used. In AML, the leukemia cells may spread to the brain and/or spinal cord. Chemotherapy given by mouth or vein to treat AML may not cross the blood-brain barrier to get into the fluid that surrounds the brain and spinal cord. Instead, chemotherapy is injected into the fluid-filled space to kill leukemia cells that may have spread there (intrathecal chemotherapy).EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. See Drugs Approved for Acute Myeloid Leukemia for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. In childhood AML, external radiation therapy may be used to treat a myeloid sarcoma that does not respond to chemotherapy. Chemotherapy is given to kill cancer cells or other abnormal blood cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: See Drugs Approved for Leukemia for more information. Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change. Arsenic trioxide and tretinoin are drugs that kill certain types of leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of acute promyelocytic leukemia. See Drugs Approved for Acute Myeloid Leukemia for more information. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. It is sometimes used to treat transient abnormal myelopoiesis (TAM). Supportive care is given to lessen the problems caused by the disease or its treatment. All patients with leukemia receive supportive care treatments. Supportive care may include the following: This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Regular follow-up exams are very important. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their child's doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute myeloid leukemia (AML) during the induction phase may include the following: Treatment of childhood AML during the remission phase (consolidation/intensification therapy) depends on the subtype of AML and may include the following: Treatment of refractory childhood AML may include the following: Treatment of recurrent childhood AML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Transient abnormal myelopoiesis (TAM) usually goes away on its own. For newly diagnosed TAM that does not go away on its own or causes other health problems, treatment may include the following: Treatment of newly diagnosed acute myeloid leukemia (AML) in children aged 4 years or younger who have Down syndrome may include the following: Treatment of newly diagnosed AML in children older than 4 years who have Down syndrome may be the same as treatment for children without Down syndrome. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute promyelocytic leukemia (APL) may include the following: Treatment of childhood APL during the remission phase (consolidation/intensification therapy) may include the following: Treatment of childhood APL during the remission phase (maintenance therapy) may include the following: Treatment of recurrent childhood APL may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed juvenile myelomonocytic leukemia (JMML) may include the following: Treatment of refractory or recurrent childhood JMML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed chronic myelogenous leukemia (CML) may include the following: Treatment of refractory or recurrent childhood CML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood myelodysplastic syndromes (MDS) may include the following: If the MDS becomes acute myeloid leukemia (AML), treatment will be the same as treatment for newly diagnosed AML. For more information from the National Cancer Institute about childhood acute myeloid leukemia and other myeloid malignancies, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood acute myeloid leukemia and other myeloid malignancies. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. 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What is (are) Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies ?

Key Points - Childhood acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes a large number of abnormal blood cells. - Leukemia and other diseases of the blood and bone marrow may affect red blood cells, white blood cells, and platelets. - Other myeloid diseases can affect the blood and bone marrow. - Chronic myelogenous leukemia - Juvenile myelomonocytic leukemia - Myelodysplastic syndromes - AML or MDS may occur after treatment with certain anticancer drugs and/or radiation therapy. - The risk factors for childhood AML, childhood CML, JMML, and MDS are similar. - Signs and symptoms of childhood AML, childhood CML, JMML, or MDS include fever, feeling tired, and easy bleeding or bruising. - Tests that examine the blood and bone marrow are used to detect (find) and diagnose childhood AML, childhood CML, JMML, and MDS. - Certain factors affect prognosis (chance of recovery) and treatment options. Childhood acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes a large number of abnormal blood cells. Childhood acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is also called acute myelogenous leukemia, acute myeloblastic leukemia, acute granulocytic leukemia, and acute nonlymphocytic leukemia. Cancers that are acute usually get worse quickly if they are not treated. Cancers that are chronic usually get worse slowly. Leukemia and other diseases of the blood and bone marrow may affect red blood cells, white blood cells, and platelets. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: - Red blood cells that carry oxygen and other substances to all tissues of the body. - White blood cells that fight infection and disease. - Platelets that form blood clots to stop bleeding. In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts, or leukemia cells, in AML are abnormal and do not become healthy white blood cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a granulocytic sarcoma or chloroma. There are subtypes of AML based on the type of blood cell that is affected. The treatment of AML is different when it is a subtype called acute promyelocytic leukemia (APL) or when the child has Down syndrome. Other myeloid diseases can affect the blood and bone marrow. Chronic myelogenous leukemia In chronic myelogenous leukemia (CML), too many bone marrow stem cells become a type of white blood cell called granulocytes. Some of these bone marrow stem cells never become mature white blood cells. These are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. CML is rare in children. Juvenile myelomonocytic leukemia Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children around the age of 2 years and is more common in boys. In JMML, too many bone marrow stem cells become 2 types of white blood cells called myelocytes and monocytes. Some of these bone marrow stem cells never become mature white blood cells. These immature cells, called blasts, are unable to do their usual work. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. Myelodysplastic syndromes Myelodysplastic syndromes (MDS) occur less often in children than in adults. In MDS, the bone marrow makes too few red blood cells, white blood cells, and platelets. These blood cells may not mature and enter the blood. The treatment for MDS depends on how low the numbers of red blood cells, white blood cells, or platelets are. Over time, MDS may become AML. Transient myeloproliferative disorder (TMD) is a type of MDS. This disorder of the bone marrow can develop in newborns who have Down syndrome. It usually goes away on its own within the first 3 weeks of life. Infants who have Down syndrome and TMD have an increased chance of developing AML before the age of 3 years. This summary is about childhood AML, childhood CML, JMML, and MDS. See the following PDQ summaries for more information about other types of leukemia and diseases of the blood and bone marrow in children and adults: - Childhood Acute Lymphoblastic Leukemia Treatment - Adult Acute Myeloid Leukemia Treatment - Adult Acute Lymphoblastic Leukemia Treatment - Chronic Myelogenous Leukemia Treatment - Chronic Lymphocytic Leukemia Treatment - Hairy Cell Leukemia Treatment - Myelodysplastic Syndromes Treatment - Myelodysplastic/Myeloproliferative Neoplasms Treatment AML or MDS may occur after treatment with certain anticancer drugs and/or radiation therapy. Cancer treatment with certain anticancer drugs and/or radiation therapy may cause therapy -related AML (t-AML) or therapy-related MDS (t-MDS). The risk of these therapy-related myeloid diseases depends on the total dose of the anticancer drugs used and the radiation dose and treatment field. Some patients also have an inherited risk for t-AML and t-MDS. These therapy-related diseases usually occur within 7 years after treatment, but are rare in children.

Childhood acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Cancers that are acute usually get worse quickly if they are not treated. Cancers that are chronic usually get worse slowly. This summary is about the treatment of childhood AML, transient abnormal myelopoiesis, childhood acute promyelocytic leukemia, juvenile myelomonocytic leukemia, childhood chronic myelogenous leukemia, and childhood myelodysplastic syndromes. For information about the treatment of childhood acute lymphoblastic leukemia, see Childhood Acute Lymphoblastic Leukemia Treatment. In healthy children, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a type of white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts, or leukemia cells, in AML are abnormal and do not become healthy white blood cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This may lead to infection, anemia, or easy bleeding. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called granulocytic sarcoma or chloroma. TAM is a disorder of the bone marrow that can develop in newborns who have Down syndrome. TAM usually goes away on its own within the first 3 months of life. Infants who have TAM have an increased chance of developing AML before the age of 3 years. TAM is also called transient myeloproliferative disorder or transient leukemia. APL is a subtype of AML. In APL, some genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. The promyelocytes (leukemia cells) can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. Problems with severe bleeding and blood clots may also occur. This is a serious health problem that needs treatment as soon as possible. JMML is a rare childhood cancer that is most common in children around the age of 2 years and is more common in boys. In JMML, too many myeloid blood stem cells become myelocytes and monocytes (two types of white blood cells). Some of these myeloid blood stem cells never become mature white blood cells. These immature cells, called blasts, are unable to do their usual work. Over time, the myelocytes, monocytes, and blasts crowd out the healthy white blood cells, red blood cells, and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. CML often begins in an early myeloid blood cell when a certain gene change occurs. A section of genes, that includes the ABL gene, on chromosome 9 changes place with a section of genes on chromosome 22, which has the BCR gene. This makes a very short chromosome 22 (called the Philadelphia chromosome) and a very long chromosome 9. An abnormal BCR-ABL gene is formed on chromosome 22. The BCR-ABL gene tells the blood cells to make too much of a protein called tyrosine kinase. Tyrosine kinase causes too many abnormal white blood cells (leukemia cells) to be made in the bone marrow. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This can lead to infection, anemia, or easy bleeding. CML is rare in children.EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. MDS occur less often in children than in adults. In MDS, the bone marrow makes too few red blood cells, white blood cells, and platelets. These blood cells may not mature and enter the blood. The type of MDS depends on the type of blood cell that is affected. The treatment for MDS depends on how low the numbers of red blood cells, white blood cells, or platelets are. Over time, MDS may become AML. Cancer treatment with certain chemotherapy drugs and/or radiation therapy may cause therapy-related AML (t-AML) or therapy-related MDS (t-MDS). The risk of these therapy-related myeloid diseases depends on the total dose of the chemotherapy drugs used and the radiation dose and treatment field. Some patients also have an inherited risk for t-AML and t-MDS. These therapy-related diseases usually occur within 7 years after treatment, but are rare in children. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. These and other factors may increase the risk of childhood AML, APL, JMML, CML, and MDS: These and other signs and symptoms may be caused by childhood AML, APL, JMML, CML, or MDS or by other conditions. Check with a doctor if your child has any of the following: The signs and symptoms of TAM may include the following: Sometimes TAM does not cause any symptoms at all and is diagnosed after a routine blood test. The following tests and procedures may be used: The following test is a type of cytogenetic analysis: The prognosis and treatment options for childhood acute myeloid leukemia (AML) depend on the following: The prognosis and treatment options for childhood acute promyelocytic leukemia (APL) depends on the following: The prognosis and treatment options for juvenile myelomonocytic leukemia (JMML) depend on the following: The prognosis and treatment options for childhood chronic myelogenous leukemia (CML) depend on the following: The prognosis and treatment options for myelodysplastic syndromes (MDS) depend on the following: The extent or spread of cancer is usually described as stages. Instead of stages, treatment is based on one or more of the following: Newly diagnosed childhood AML Newly diagnosed childhood AML is cancer that has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and has one of the following: or Childhood AML in remission In childhood AML in remission, the disease has been treated and the following are found: Refractory leukemia is cancer that does not respond to treatment. Recurrent leukemia is cancer that has recurred (come back) after it has been treated. The cancer may come back in the blood and bone marrow or in other parts of the body, such as the central nervous system (brain and spinal cord). Different types of treatment are available for children with acute myeloid leukemia (AML), transient abnormal myelopoiesis (TAM), acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia (JMML), chronic myelogenous leukemia (CML), and myelodysplastic syndromes (MDS). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because AML and other myeloid disorders are rare in children, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not yet started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists: The treatment of childhood AML is done in phases: Treatment called central nervous system (CNS) prophylaxis therapy may be given during the induction phase of therapy. Because standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the leukemia cells are able to hide in the CNS. Intrathecal chemotherapy is able to reach leukemia cells in the CNS. It is given to kill the leukemia cells and lessen the chance the leukemia will recur (come back). The treatment of childhood APL includes a third phase called maintenance. The goal of maintenance is to kill any remaining leukemia cells that may regrow and cause a relapse. Often the cancer treatments are given in lower doses than those used during the remission induction and consolidation/intensification phases. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one chemotherapy drug. The way the chemotherapy is given depends on the type of cancer being treated. In AML, chemotherapy given by mouth, vein, or into the cerebrospinal fluid is used. In AML, the leukemia cells may spread to the brain and/or spinal cord. Chemotherapy given by mouth or vein to treat AML may not cross the blood-brain barrier to get into the fluid that surrounds the brain and spinal cord. Instead, chemotherapy is injected into the fluid-filled space to kill leukemia cells that may have spread there (intrathecal chemotherapy).EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. See Drugs Approved for Acute Myeloid Leukemia for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. In childhood AML, external radiation therapy may be used to treat a myeloid sarcoma that does not respond to chemotherapy. Chemotherapy is given to kill cancer cells or other abnormal blood cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: See Drugs Approved for Leukemia for more information. Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change. Arsenic trioxide and tretinoin are drugs that kill certain types of leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of acute promyelocytic leukemia. See Drugs Approved for Acute Myeloid Leukemia for more information. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. It is sometimes used to treat transient abnormal myelopoiesis (TAM). Supportive care is given to lessen the problems caused by the disease or its treatment. All patients with leukemia receive supportive care treatments. Supportive care may include the following: This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Regular follow-up exams are very important. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their child's doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute myeloid leukemia (AML) during the induction phase may include the following: Treatment of childhood AML during the remission phase (consolidation/intensification therapy) depends on the subtype of AML and may include the following: Treatment of refractory childhood AML may include the following: Treatment of recurrent childhood AML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Transient abnormal myelopoiesis (TAM) usually goes away on its own. For newly diagnosed TAM that does not go away on its own or causes other health problems, treatment may include the following: Treatment of newly diagnosed acute myeloid leukemia (AML) in children aged 4 years or younger who have Down syndrome may include the following: Treatment of newly diagnosed AML in children older than 4 years who have Down syndrome may be the same as treatment for children without Down syndrome. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute promyelocytic leukemia (APL) may include the following: Treatment of childhood APL during the remission phase (consolidation/intensification therapy) may include the following: Treatment of childhood APL during the remission phase (maintenance therapy) may include the following: Treatment of recurrent childhood APL may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed juvenile myelomonocytic leukemia (JMML) may include the following: Treatment of refractory or recurrent childhood JMML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed chronic myelogenous leukemia (CML) may include the following: Treatment of refractory or recurrent childhood CML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood myelodysplastic syndromes (MDS) may include the following: If the MDS becomes acute myeloid leukemia (AML), treatment will be the same as treatment for newly diagnosed AML. For more information from the National Cancer Institute about childhood acute myeloid leukemia and other myeloid malignancies, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood acute myeloid leukemia and other myeloid malignancies. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/child-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389303] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies? ?

The risk factors for childhood AML, childhood CML, JMML, and MDS are similar. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesnt mean that you will not get cancer. Talk with your childs doctor if you think your child may be at risk. These and other factors may increase the risk of childhood AML, childhood CML, JMML, and MDS: - Having a brother or sister, especially a twin, with leukemia. - Being Hispanic. - Being exposed to cigarette smoke or alcohol before birth. - Having a personal history of aplastic anemia. - Having a personal or family history of MDS. - Having a family history of AML. - Past treatment with chemotherapy or radiation therapy. - Being exposed to ionizing radiation or chemicals such as benzene. - Having certain genetic disorders, such as: - Down syndrome. - Fanconi anemia. - Neurofibromatosis type 1. - Noonan syndrome. - Shwachman-Diamond syndrome.

Childhood acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Cancers that are acute usually get worse quickly if they are not treated. Cancers that are chronic usually get worse slowly. This summary is about the treatment of childhood AML, transient abnormal myelopoiesis, childhood acute promyelocytic leukemia, juvenile myelomonocytic leukemia, childhood chronic myelogenous leukemia, and childhood myelodysplastic syndromes. For information about the treatment of childhood acute lymphoblastic leukemia, see Childhood Acute Lymphoblastic Leukemia Treatment. In healthy children, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a type of white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts, or leukemia cells, in AML are abnormal and do not become healthy white blood cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This may lead to infection, anemia, or easy bleeding. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called granulocytic sarcoma or chloroma. TAM is a disorder of the bone marrow that can develop in newborns who have Down syndrome. TAM usually goes away on its own within the first 3 months of life. Infants who have TAM have an increased chance of developing AML before the age of 3 years. TAM is also called transient myeloproliferative disorder or transient leukemia. APL is a subtype of AML. In APL, some genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. The promyelocytes (leukemia cells) can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. Problems with severe bleeding and blood clots may also occur. This is a serious health problem that needs treatment as soon as possible. JMML is a rare childhood cancer that is most common in children around the age of 2 years and is more common in boys. In JMML, too many myeloid blood stem cells become myelocytes and monocytes (two types of white blood cells). Some of these myeloid blood stem cells never become mature white blood cells. These immature cells, called blasts, are unable to do their usual work. Over time, the myelocytes, monocytes, and blasts crowd out the healthy white blood cells, red blood cells, and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. CML often begins in an early myeloid blood cell when a certain gene change occurs. A section of genes, that includes the ABL gene, on chromosome 9 changes place with a section of genes on chromosome 22, which has the BCR gene. This makes a very short chromosome 22 (called the Philadelphia chromosome) and a very long chromosome 9. An abnormal BCR-ABL gene is formed on chromosome 22. The BCR-ABL gene tells the blood cells to make too much of a protein called tyrosine kinase. Tyrosine kinase causes too many abnormal white blood cells (leukemia cells) to be made in the bone marrow. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This can lead to infection, anemia, or easy bleeding. CML is rare in children.EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. MDS occur less often in children than in adults. In MDS, the bone marrow makes too few red blood cells, white blood cells, and platelets. These blood cells may not mature and enter the blood. The type of MDS depends on the type of blood cell that is affected. The treatment for MDS depends on how low the numbers of red blood cells, white blood cells, or platelets are. Over time, MDS may become AML. Cancer treatment with certain chemotherapy drugs and/or radiation therapy may cause therapy-related AML (t-AML) or therapy-related MDS (t-MDS). The risk of these therapy-related myeloid diseases depends on the total dose of the chemotherapy drugs used and the radiation dose and treatment field. Some patients also have an inherited risk for t-AML and t-MDS. These therapy-related diseases usually occur within 7 years after treatment, but are rare in children. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. These and other factors may increase the risk of childhood AML, APL, JMML, CML, and MDS: These and other signs and symptoms may be caused by childhood AML, APL, JMML, CML, or MDS or by other conditions. Check with a doctor if your child has any of the following: The signs and symptoms of TAM may include the following: Sometimes TAM does not cause any symptoms at all and is diagnosed after a routine blood test. The following tests and procedures may be used: The following test is a type of cytogenetic analysis: The prognosis and treatment options for childhood acute myeloid leukemia (AML) depend on the following: The prognosis and treatment options for childhood acute promyelocytic leukemia (APL) depends on the following: The prognosis and treatment options for juvenile myelomonocytic leukemia (JMML) depend on the following: The prognosis and treatment options for childhood chronic myelogenous leukemia (CML) depend on the following: The prognosis and treatment options for myelodysplastic syndromes (MDS) depend on the following: The extent or spread of cancer is usually described as stages. Instead of stages, treatment is based on one or more of the following: Newly diagnosed childhood AML Newly diagnosed childhood AML is cancer that has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and has one of the following: or Childhood AML in remission In childhood AML in remission, the disease has been treated and the following are found: Refractory leukemia is cancer that does not respond to treatment. Recurrent leukemia is cancer that has recurred (come back) after it has been treated. The cancer may come back in the blood and bone marrow or in other parts of the body, such as the central nervous system (brain and spinal cord). Different types of treatment are available for children with acute myeloid leukemia (AML), transient abnormal myelopoiesis (TAM), acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia (JMML), chronic myelogenous leukemia (CML), and myelodysplastic syndromes (MDS). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because AML and other myeloid disorders are rare in children, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not yet started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists: The treatment of childhood AML is done in phases: Treatment called central nervous system (CNS) prophylaxis therapy may be given during the induction phase of therapy. Because standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the leukemia cells are able to hide in the CNS. Intrathecal chemotherapy is able to reach leukemia cells in the CNS. It is given to kill the leukemia cells and lessen the chance the leukemia will recur (come back). The treatment of childhood APL includes a third phase called maintenance. The goal of maintenance is to kill any remaining leukemia cells that may regrow and cause a relapse. Often the cancer treatments are given in lower doses than those used during the remission induction and consolidation/intensification phases. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one chemotherapy drug. The way the chemotherapy is given depends on the type of cancer being treated. In AML, chemotherapy given by mouth, vein, or into the cerebrospinal fluid is used. In AML, the leukemia cells may spread to the brain and/or spinal cord. Chemotherapy given by mouth or vein to treat AML may not cross the blood-brain barrier to get into the fluid that surrounds the brain and spinal cord. Instead, chemotherapy is injected into the fluid-filled space to kill leukemia cells that may have spread there (intrathecal chemotherapy).EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. See Drugs Approved for Acute Myeloid Leukemia for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. In childhood AML, external radiation therapy may be used to treat a myeloid sarcoma that does not respond to chemotherapy. Chemotherapy is given to kill cancer cells or other abnormal blood cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: See Drugs Approved for Leukemia for more information. Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change. Arsenic trioxide and tretinoin are drugs that kill certain types of leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of acute promyelocytic leukemia. See Drugs Approved for Acute Myeloid Leukemia for more information. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. It is sometimes used to treat transient abnormal myelopoiesis (TAM). Supportive care is given to lessen the problems caused by the disease or its treatment. All patients with leukemia receive supportive care treatments. Supportive care may include the following: This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Regular follow-up exams are very important. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their child's doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute myeloid leukemia (AML) during the induction phase may include the following: Treatment of childhood AML during the remission phase (consolidation/intensification therapy) depends on the subtype of AML and may include the following: Treatment of refractory childhood AML may include the following: Treatment of recurrent childhood AML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Transient abnormal myelopoiesis (TAM) usually goes away on its own. For newly diagnosed TAM that does not go away on its own or causes other health problems, treatment may include the following: Treatment of newly diagnosed acute myeloid leukemia (AML) in children aged 4 years or younger who have Down syndrome may include the following: Treatment of newly diagnosed AML in children older than 4 years who have Down syndrome may be the same as treatment for children without Down syndrome. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute promyelocytic leukemia (APL) may include the following: Treatment of childhood APL during the remission phase (consolidation/intensification therapy) may include the following: Treatment of childhood APL during the remission phase (maintenance therapy) may include the following: Treatment of recurrent childhood APL may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed juvenile myelomonocytic leukemia (JMML) may include the following: Treatment of refractory or recurrent childhood JMML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed chronic myelogenous leukemia (CML) may include the following: Treatment of refractory or recurrent childhood CML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood myelodysplastic syndromes (MDS) may include the following: If the MDS becomes acute myeloid leukemia (AML), treatment will be the same as treatment for newly diagnosed AML. For more information from the National Cancer Institute about childhood acute myeloid leukemia and other myeloid malignancies, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood acute myeloid leukemia and other myeloid malignancies. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/child-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389303] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies ?

Signs and symptoms of childhood AML, childhood CML, JMML, or MDS include fever, feeling tired, and easy bleeding or bruising. These and other signs and symptoms may be caused by childhood AML, childhood CML, JMML, or MDS or by other conditions. Check with a doctor if your child has any of the following: - Fever with or without an infection. - Night sweats. - Shortness of breath. - Weakness or feeling tired. - Easy bruising or bleeding. - Petechiae (flat, pinpoint spots under the skin caused by bleeding). - Pain in the bones or joints. - Pain or feeling of fullness below the ribs. - Painless lumps in the neck, underarm, stomach, groin, or other parts of the body. In childhood AML, these lumps, called leukemia cutis, may be blue or purple. - Painless lumps that are sometimes around the eyes. These lumps, called chloromas, are sometimes seen in childhood AML and may be blue-green. - An eczema -like skin rash. The signs and symptoms of TMD may include the following: - Swelling all over the body. - Shortness of breath. - Trouble breathing. - Weakness or feeling tired. - Pain below the ribs.

Childhood acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Cancers that are acute usually get worse quickly if they are not treated. Cancers that are chronic usually get worse slowly. This summary is about the treatment of childhood AML, transient abnormal myelopoiesis, childhood acute promyelocytic leukemia, juvenile myelomonocytic leukemia, childhood chronic myelogenous leukemia, and childhood myelodysplastic syndromes. For information about the treatment of childhood acute lymphoblastic leukemia, see Childhood Acute Lymphoblastic Leukemia Treatment. In healthy children, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a type of white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts, or leukemia cells, in AML are abnormal and do not become healthy white blood cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This may lead to infection, anemia, or easy bleeding. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called granulocytic sarcoma or chloroma. TAM is a disorder of the bone marrow that can develop in newborns who have Down syndrome. TAM usually goes away on its own within the first 3 months of life. Infants who have TAM have an increased chance of developing AML before the age of 3 years. TAM is also called transient myeloproliferative disorder or transient leukemia. APL is a subtype of AML. In APL, some genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. The promyelocytes (leukemia cells) can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. Problems with severe bleeding and blood clots may also occur. This is a serious health problem that needs treatment as soon as possible. JMML is a rare childhood cancer that is most common in children around the age of 2 years and is more common in boys. In JMML, too many myeloid blood stem cells become myelocytes and monocytes (two types of white blood cells). Some of these myeloid blood stem cells never become mature white blood cells. These immature cells, called blasts, are unable to do their usual work. Over time, the myelocytes, monocytes, and blasts crowd out the healthy white blood cells, red blood cells, and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. CML often begins in an early myeloid blood cell when a certain gene change occurs. A section of genes, that includes the ABL gene, on chromosome 9 changes place with a section of genes on chromosome 22, which has the BCR gene. This makes a very short chromosome 22 (called the Philadelphia chromosome) and a very long chromosome 9. An abnormal BCR-ABL gene is formed on chromosome 22. The BCR-ABL gene tells the blood cells to make too much of a protein called tyrosine kinase. Tyrosine kinase causes too many abnormal white blood cells (leukemia cells) to be made in the bone marrow. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This can lead to infection, anemia, or easy bleeding. CML is rare in children.EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. MDS occur less often in children than in adults. In MDS, the bone marrow makes too few red blood cells, white blood cells, and platelets. These blood cells may not mature and enter the blood. The type of MDS depends on the type of blood cell that is affected. The treatment for MDS depends on how low the numbers of red blood cells, white blood cells, or platelets are. Over time, MDS may become AML. Cancer treatment with certain chemotherapy drugs and/or radiation therapy may cause therapy-related AML (t-AML) or therapy-related MDS (t-MDS). The risk of these therapy-related myeloid diseases depends on the total dose of the chemotherapy drugs used and the radiation dose and treatment field. Some patients also have an inherited risk for t-AML and t-MDS. These therapy-related diseases usually occur within 7 years after treatment, but are rare in children. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. These and other factors may increase the risk of childhood AML, APL, JMML, CML, and MDS: These and other signs and symptoms may be caused by childhood AML, APL, JMML, CML, or MDS or by other conditions. Check with a doctor if your child has any of the following: The signs and symptoms of TAM may include the following: Sometimes TAM does not cause any symptoms at all and is diagnosed after a routine blood test. The following tests and procedures may be used: The following test is a type of cytogenetic analysis: The prognosis and treatment options for childhood acute myeloid leukemia (AML) depend on the following: The prognosis and treatment options for childhood acute promyelocytic leukemia (APL) depends on the following: The prognosis and treatment options for juvenile myelomonocytic leukemia (JMML) depend on the following: The prognosis and treatment options for childhood chronic myelogenous leukemia (CML) depend on the following: The prognosis and treatment options for myelodysplastic syndromes (MDS) depend on the following: The extent or spread of cancer is usually described as stages. Instead of stages, treatment is based on one or more of the following: Newly diagnosed childhood AML Newly diagnosed childhood AML is cancer that has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and has one of the following: or Childhood AML in remission In childhood AML in remission, the disease has been treated and the following are found: Refractory leukemia is cancer that does not respond to treatment. Recurrent leukemia is cancer that has recurred (come back) after it has been treated. The cancer may come back in the blood and bone marrow or in other parts of the body, such as the central nervous system (brain and spinal cord). Different types of treatment are available for children with acute myeloid leukemia (AML), transient abnormal myelopoiesis (TAM), acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia (JMML), chronic myelogenous leukemia (CML), and myelodysplastic syndromes (MDS). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because AML and other myeloid disorders are rare in children, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not yet started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists: The treatment of childhood AML is done in phases: Treatment called central nervous system (CNS) prophylaxis therapy may be given during the induction phase of therapy. Because standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the leukemia cells are able to hide in the CNS. Intrathecal chemotherapy is able to reach leukemia cells in the CNS. It is given to kill the leukemia cells and lessen the chance the leukemia will recur (come back). The treatment of childhood APL includes a third phase called maintenance. The goal of maintenance is to kill any remaining leukemia cells that may regrow and cause a relapse. Often the cancer treatments are given in lower doses than those used during the remission induction and consolidation/intensification phases. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one chemotherapy drug. The way the chemotherapy is given depends on the type of cancer being treated. In AML, chemotherapy given by mouth, vein, or into the cerebrospinal fluid is used. In AML, the leukemia cells may spread to the brain and/or spinal cord. Chemotherapy given by mouth or vein to treat AML may not cross the blood-brain barrier to get into the fluid that surrounds the brain and spinal cord. Instead, chemotherapy is injected into the fluid-filled space to kill leukemia cells that may have spread there (intrathecal chemotherapy).EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. See Drugs Approved for Acute Myeloid Leukemia for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. In childhood AML, external radiation therapy may be used to treat a myeloid sarcoma that does not respond to chemotherapy. Chemotherapy is given to kill cancer cells or other abnormal blood cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: See Drugs Approved for Leukemia for more information. Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change. Arsenic trioxide and tretinoin are drugs that kill certain types of leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of acute promyelocytic leukemia. See Drugs Approved for Acute Myeloid Leukemia for more information. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. It is sometimes used to treat transient abnormal myelopoiesis (TAM). Supportive care is given to lessen the problems caused by the disease or its treatment. All patients with leukemia receive supportive care treatments. Supportive care may include the following: This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Regular follow-up exams are very important. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their child's doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute myeloid leukemia (AML) during the induction phase may include the following: Treatment of childhood AML during the remission phase (consolidation/intensification therapy) depends on the subtype of AML and may include the following: Treatment of refractory childhood AML may include the following: Treatment of recurrent childhood AML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Transient abnormal myelopoiesis (TAM) usually goes away on its own. For newly diagnosed TAM that does not go away on its own or causes other health problems, treatment may include the following: Treatment of newly diagnosed acute myeloid leukemia (AML) in children aged 4 years or younger who have Down syndrome may include the following: Treatment of newly diagnosed AML in children older than 4 years who have Down syndrome may be the same as treatment for children without Down syndrome. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute promyelocytic leukemia (APL) may include the following: Treatment of childhood APL during the remission phase (consolidation/intensification therapy) may include the following: Treatment of childhood APL during the remission phase (maintenance therapy) may include the following: Treatment of recurrent childhood APL may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed juvenile myelomonocytic leukemia (JMML) may include the following: Treatment of refractory or recurrent childhood JMML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed chronic myelogenous leukemia (CML) may include the following: Treatment of refractory or recurrent childhood CML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood myelodysplastic syndromes (MDS) may include the following: If the MDS becomes acute myeloid leukemia (AML), treatment will be the same as treatment for newly diagnosed AML. For more information from the National Cancer Institute about childhood acute myeloid leukemia and other myeloid malignancies, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood acute myeloid leukemia and other myeloid malignancies. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. 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How to diagnose Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies ?

Tests that examine the blood and bone marrow are used to detect (find) and diagnose childhood AML, childhood CML, JMML, and MDS. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Complete blood count (CBC) with differential: A procedure in which a sample of blood is drawn and checked for the following: - The number of red blood cells and platelets. - The number and type of white blood cells. - The amount of hemoglobin (the protein that carries oxygen) in the red blood cells. - The portion of the blood sample made up of red blood cells. - Peripheral blood smear : A procedure in which a sample of blood is checked for blast cells, the number and kinds of white blood cells, number of platelets, and changes in the shape of the blood cells. - Blood chemistry studies : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease. - Chest x-ray : An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. - Biopsy : The removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for signs of cancer. Biopsies that may be done include the following: - Bone marrow aspiration and biopsy : The removal of bone marrow, blood, and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. - Tumor biopsy: A biopsy of a chloroma may be done. - Lymph node biopsy: The removal of all or part of a lymph node. - Cytogenetic analysis : A laboratory test in which cells in a sample of blood or bone marrow are viewed under a microscope to look for certain changes in the chromosomes. Changes in the chromosomes may include when part of one chromosome is switched with part of another chromosome, part of one chromosome is missing or repeated, or part of one chromosome is turned upside down. The following test is a type of cytogenetic analysis: - FISH (fluorescence in situ hybridization): A laboratory technique used to look at genes or chromosomes in cells and tissues. Pieces of DNA that contain a fluorescent dye are made in the laboratory and added to cells or tissues on a glass slide. When these pieces of DNA bind to specific genes or areas of chromosomes on the slide, they light up when viewed under a microscope with a special light. - Reverse transcriptionpolymerase chain reaction (RTPCR) test: A laboratory test in which cells in a sample of tissue are studied using chemicals to look for certain changes in the structure or function of genes. - Immunophenotyping : A process used to identify cells, based on the types of antigens or markers on the surface of the cell, that may include special staining of the blood and bone marrow cells. This process is used to diagnose the subtype of AML by comparing the cancer cells to normal cells of the immune system. - Molecular testing : A laboratory test to check for certain genes, proteins, or other molecules in a sample of blood or bone marrow. Molecular tests also check for certain changes in a gene or chromosome that may cause or affect the chance of developing AML. A molecular test may be used to help plan treatment, find out how well treatment is working, or make a prognosis. - Lumbar puncture : A procedure used to collect a sample of cerebrospinal fluid (CSF) from the spinal column. This is done by placing a needle between two bones in the spine and into the CSF around the spinal cord and removing a sample of the fluid. The sample of CSF is checked under a microscope for signs that leukemia cells have spread to the brain and spinal cord. This procedure is also called an LP or spinal tap.

Childhood acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Cancers that are acute usually get worse quickly if they are not treated. Cancers that are chronic usually get worse slowly. This summary is about the treatment of childhood AML, transient abnormal myelopoiesis, childhood acute promyelocytic leukemia, juvenile myelomonocytic leukemia, childhood chronic myelogenous leukemia, and childhood myelodysplastic syndromes. For information about the treatment of childhood acute lymphoblastic leukemia, see Childhood Acute Lymphoblastic Leukemia Treatment. In healthy children, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a type of white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts, or leukemia cells, in AML are abnormal and do not become healthy white blood cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This may lead to infection, anemia, or easy bleeding. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called granulocytic sarcoma or chloroma. TAM is a disorder of the bone marrow that can develop in newborns who have Down syndrome. TAM usually goes away on its own within the first 3 months of life. Infants who have TAM have an increased chance of developing AML before the age of 3 years. TAM is also called transient myeloproliferative disorder or transient leukemia. APL is a subtype of AML. In APL, some genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. The promyelocytes (leukemia cells) can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. Problems with severe bleeding and blood clots may also occur. This is a serious health problem that needs treatment as soon as possible. JMML is a rare childhood cancer that is most common in children around the age of 2 years and is more common in boys. In JMML, too many myeloid blood stem cells become myelocytes and monocytes (two types of white blood cells). Some of these myeloid blood stem cells never become mature white blood cells. These immature cells, called blasts, are unable to do their usual work. Over time, the myelocytes, monocytes, and blasts crowd out the healthy white blood cells, red blood cells, and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. CML often begins in an early myeloid blood cell when a certain gene change occurs. A section of genes, that includes the ABL gene, on chromosome 9 changes place with a section of genes on chromosome 22, which has the BCR gene. This makes a very short chromosome 22 (called the Philadelphia chromosome) and a very long chromosome 9. An abnormal BCR-ABL gene is formed on chromosome 22. The BCR-ABL gene tells the blood cells to make too much of a protein called tyrosine kinase. Tyrosine kinase causes too many abnormal white blood cells (leukemia cells) to be made in the bone marrow. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This can lead to infection, anemia, or easy bleeding. CML is rare in children.EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. MDS occur less often in children than in adults. In MDS, the bone marrow makes too few red blood cells, white blood cells, and platelets. These blood cells may not mature and enter the blood. The type of MDS depends on the type of blood cell that is affected. The treatment for MDS depends on how low the numbers of red blood cells, white blood cells, or platelets are. Over time, MDS may become AML. Cancer treatment with certain chemotherapy drugs and/or radiation therapy may cause therapy-related AML (t-AML) or therapy-related MDS (t-MDS). The risk of these therapy-related myeloid diseases depends on the total dose of the chemotherapy drugs used and the radiation dose and treatment field. Some patients also have an inherited risk for t-AML and t-MDS. These therapy-related diseases usually occur within 7 years after treatment, but are rare in children. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. These and other factors may increase the risk of childhood AML, APL, JMML, CML, and MDS: These and other signs and symptoms may be caused by childhood AML, APL, JMML, CML, or MDS or by other conditions. Check with a doctor if your child has any of the following: The signs and symptoms of TAM may include the following: Sometimes TAM does not cause any symptoms at all and is diagnosed after a routine blood test. The following tests and procedures may be used: The following test is a type of cytogenetic analysis: The prognosis and treatment options for childhood acute myeloid leukemia (AML) depend on the following: The prognosis and treatment options for childhood acute promyelocytic leukemia (APL) depends on the following: The prognosis and treatment options for juvenile myelomonocytic leukemia (JMML) depend on the following: The prognosis and treatment options for childhood chronic myelogenous leukemia (CML) depend on the following: The prognosis and treatment options for myelodysplastic syndromes (MDS) depend on the following: The extent or spread of cancer is usually described as stages. Instead of stages, treatment is based on one or more of the following: Newly diagnosed childhood AML Newly diagnosed childhood AML is cancer that has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and has one of the following: or Childhood AML in remission In childhood AML in remission, the disease has been treated and the following are found: Refractory leukemia is cancer that does not respond to treatment. Recurrent leukemia is cancer that has recurred (come back) after it has been treated. The cancer may come back in the blood and bone marrow or in other parts of the body, such as the central nervous system (brain and spinal cord). Different types of treatment are available for children with acute myeloid leukemia (AML), transient abnormal myelopoiesis (TAM), acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia (JMML), chronic myelogenous leukemia (CML), and myelodysplastic syndromes (MDS). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because AML and other myeloid disorders are rare in children, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not yet started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists: The treatment of childhood AML is done in phases: Treatment called central nervous system (CNS) prophylaxis therapy may be given during the induction phase of therapy. Because standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the leukemia cells are able to hide in the CNS. Intrathecal chemotherapy is able to reach leukemia cells in the CNS. It is given to kill the leukemia cells and lessen the chance the leukemia will recur (come back). The treatment of childhood APL includes a third phase called maintenance. The goal of maintenance is to kill any remaining leukemia cells that may regrow and cause a relapse. Often the cancer treatments are given in lower doses than those used during the remission induction and consolidation/intensification phases. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one chemotherapy drug. The way the chemotherapy is given depends on the type of cancer being treated. In AML, chemotherapy given by mouth, vein, or into the cerebrospinal fluid is used. In AML, the leukemia cells may spread to the brain and/or spinal cord. Chemotherapy given by mouth or vein to treat AML may not cross the blood-brain barrier to get into the fluid that surrounds the brain and spinal cord. Instead, chemotherapy is injected into the fluid-filled space to kill leukemia cells that may have spread there (intrathecal chemotherapy).EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. See Drugs Approved for Acute Myeloid Leukemia for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. In childhood AML, external radiation therapy may be used to treat a myeloid sarcoma that does not respond to chemotherapy. Chemotherapy is given to kill cancer cells or other abnormal blood cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: See Drugs Approved for Leukemia for more information. Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change. Arsenic trioxide and tretinoin are drugs that kill certain types of leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of acute promyelocytic leukemia. See Drugs Approved for Acute Myeloid Leukemia for more information. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. It is sometimes used to treat transient abnormal myelopoiesis (TAM). Supportive care is given to lessen the problems caused by the disease or its treatment. All patients with leukemia receive supportive care treatments. Supportive care may include the following: This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Regular follow-up exams are very important. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their child's doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute myeloid leukemia (AML) during the induction phase may include the following: Treatment of childhood AML during the remission phase (consolidation/intensification therapy) depends on the subtype of AML and may include the following: Treatment of refractory childhood AML may include the following: Treatment of recurrent childhood AML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Transient abnormal myelopoiesis (TAM) usually goes away on its own. For newly diagnosed TAM that does not go away on its own or causes other health problems, treatment may include the following: Treatment of newly diagnosed acute myeloid leukemia (AML) in children aged 4 years or younger who have Down syndrome may include the following: Treatment of newly diagnosed AML in children older than 4 years who have Down syndrome may be the same as treatment for children without Down syndrome. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute promyelocytic leukemia (APL) may include the following: Treatment of childhood APL during the remission phase (consolidation/intensification therapy) may include the following: Treatment of childhood APL during the remission phase (maintenance therapy) may include the following: Treatment of recurrent childhood APL may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed juvenile myelomonocytic leukemia (JMML) may include the following: Treatment of refractory or recurrent childhood JMML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed chronic myelogenous leukemia (CML) may include the following: Treatment of refractory or recurrent childhood CML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood myelodysplastic syndromes (MDS) may include the following: If the MDS becomes acute myeloid leukemia (AML), treatment will be the same as treatment for newly diagnosed AML. For more information from the National Cancer Institute about childhood acute myeloid leukemia and other myeloid malignancies, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood acute myeloid leukemia and other myeloid malignancies. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/child-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389303] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options for childhood AML depend on the following: - The age of the child when the cancer is diagnosed. - The race or ethnic group of the child. - Whether the child is greatly overweight. - Number of white blood cells in the blood at diagnosis. - Whether the AML occurred after previous cancer treatment. - The subtype of AML. - Whether there are certain chromosome or gene changes in the leukemia cells. - Whether the child has Down syndrome. Most children with AML and Down syndrome can be cured of their leukemia. - Whether the leukemia is in the central nervous system (brain and spinal cord). - How quickly the leukemia responds to treatment. - Whether the AML is newly diagnosed (untreated) or has recurred (come back) after being treated. - The length of time since treatment ended, for AML that has recurred. The prognosis and treatment options for childhood CML depend on how long it has been since the patient was diagnosed and how many blast cells are in the blood. The prognosis (chance of recovery) and treatment options for JMML depend on the following: - The age of the child when the cancer is diagnosed. - The type of gene affected and the number of genes that have changes. - How many red blood cells, white blood cells, or platelets are in the blood. - Whether the JMML is newly diagnosed (untreated) or has recurred after treatment. The prognosis (chance of recovery) and treatment options for MDS depend on the following: - Whether the MDS was caused by previous cancer treatment. - How low the numbers of red blood cells, white blood cells, or platelets are. - Whether the MDS is newly diagnosed (untreated) or has recurred after treatment.

Childhood acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Cancers that are acute usually get worse quickly if they are not treated. Cancers that are chronic usually get worse slowly. This summary is about the treatment of childhood AML, transient abnormal myelopoiesis, childhood acute promyelocytic leukemia, juvenile myelomonocytic leukemia, childhood chronic myelogenous leukemia, and childhood myelodysplastic syndromes. For information about the treatment of childhood acute lymphoblastic leukemia, see Childhood Acute Lymphoblastic Leukemia Treatment. In healthy children, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a type of white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts, or leukemia cells, in AML are abnormal and do not become healthy white blood cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This may lead to infection, anemia, or easy bleeding. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called granulocytic sarcoma or chloroma. TAM is a disorder of the bone marrow that can develop in newborns who have Down syndrome. TAM usually goes away on its own within the first 3 months of life. Infants who have TAM have an increased chance of developing AML before the age of 3 years. TAM is also called transient myeloproliferative disorder or transient leukemia. APL is a subtype of AML. In APL, some genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. The promyelocytes (leukemia cells) can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. Problems with severe bleeding and blood clots may also occur. This is a serious health problem that needs treatment as soon as possible. JMML is a rare childhood cancer that is most common in children around the age of 2 years and is more common in boys. In JMML, too many myeloid blood stem cells become myelocytes and monocytes (two types of white blood cells). Some of these myeloid blood stem cells never become mature white blood cells. These immature cells, called blasts, are unable to do their usual work. Over time, the myelocytes, monocytes, and blasts crowd out the healthy white blood cells, red blood cells, and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. CML often begins in an early myeloid blood cell when a certain gene change occurs. A section of genes, that includes the ABL gene, on chromosome 9 changes place with a section of genes on chromosome 22, which has the BCR gene. This makes a very short chromosome 22 (called the Philadelphia chromosome) and a very long chromosome 9. An abnormal BCR-ABL gene is formed on chromosome 22. The BCR-ABL gene tells the blood cells to make too much of a protein called tyrosine kinase. Tyrosine kinase causes too many abnormal white blood cells (leukemia cells) to be made in the bone marrow. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This can lead to infection, anemia, or easy bleeding. CML is rare in children.EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. MDS occur less often in children than in adults. In MDS, the bone marrow makes too few red blood cells, white blood cells, and platelets. These blood cells may not mature and enter the blood. The type of MDS depends on the type of blood cell that is affected. The treatment for MDS depends on how low the numbers of red blood cells, white blood cells, or platelets are. Over time, MDS may become AML. Cancer treatment with certain chemotherapy drugs and/or radiation therapy may cause therapy-related AML (t-AML) or therapy-related MDS (t-MDS). The risk of these therapy-related myeloid diseases depends on the total dose of the chemotherapy drugs used and the radiation dose and treatment field. Some patients also have an inherited risk for t-AML and t-MDS. These therapy-related diseases usually occur within 7 years after treatment, but are rare in children. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. These and other factors may increase the risk of childhood AML, APL, JMML, CML, and MDS: These and other signs and symptoms may be caused by childhood AML, APL, JMML, CML, or MDS or by other conditions. Check with a doctor if your child has any of the following: The signs and symptoms of TAM may include the following: Sometimes TAM does not cause any symptoms at all and is diagnosed after a routine blood test. The following tests and procedures may be used: The following test is a type of cytogenetic analysis: The prognosis and treatment options for childhood acute myeloid leukemia (AML) depend on the following: The prognosis and treatment options for childhood acute promyelocytic leukemia (APL) depends on the following: The prognosis and treatment options for juvenile myelomonocytic leukemia (JMML) depend on the following: The prognosis and treatment options for childhood chronic myelogenous leukemia (CML) depend on the following: The prognosis and treatment options for myelodysplastic syndromes (MDS) depend on the following: The extent or spread of cancer is usually described as stages. Instead of stages, treatment is based on one or more of the following: Newly diagnosed childhood AML Newly diagnosed childhood AML is cancer that has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and has one of the following: or Childhood AML in remission In childhood AML in remission, the disease has been treated and the following are found: Refractory leukemia is cancer that does not respond to treatment. Recurrent leukemia is cancer that has recurred (come back) after it has been treated. The cancer may come back in the blood and bone marrow or in other parts of the body, such as the central nervous system (brain and spinal cord). Different types of treatment are available for children with acute myeloid leukemia (AML), transient abnormal myelopoiesis (TAM), acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia (JMML), chronic myelogenous leukemia (CML), and myelodysplastic syndromes (MDS). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because AML and other myeloid disorders are rare in children, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not yet started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists: The treatment of childhood AML is done in phases: Treatment called central nervous system (CNS) prophylaxis therapy may be given during the induction phase of therapy. Because standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the leukemia cells are able to hide in the CNS. Intrathecal chemotherapy is able to reach leukemia cells in the CNS. It is given to kill the leukemia cells and lessen the chance the leukemia will recur (come back). The treatment of childhood APL includes a third phase called maintenance. The goal of maintenance is to kill any remaining leukemia cells that may regrow and cause a relapse. Often the cancer treatments are given in lower doses than those used during the remission induction and consolidation/intensification phases. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one chemotherapy drug. The way the chemotherapy is given depends on the type of cancer being treated. In AML, chemotherapy given by mouth, vein, or into the cerebrospinal fluid is used. In AML, the leukemia cells may spread to the brain and/or spinal cord. Chemotherapy given by mouth or vein to treat AML may not cross the blood-brain barrier to get into the fluid that surrounds the brain and spinal cord. Instead, chemotherapy is injected into the fluid-filled space to kill leukemia cells that may have spread there (intrathecal chemotherapy).EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. See Drugs Approved for Acute Myeloid Leukemia for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. In childhood AML, external radiation therapy may be used to treat a myeloid sarcoma that does not respond to chemotherapy. Chemotherapy is given to kill cancer cells or other abnormal blood cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: See Drugs Approved for Leukemia for more information. Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change. Arsenic trioxide and tretinoin are drugs that kill certain types of leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of acute promyelocytic leukemia. See Drugs Approved for Acute Myeloid Leukemia for more information. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. It is sometimes used to treat transient abnormal myelopoiesis (TAM). Supportive care is given to lessen the problems caused by the disease or its treatment. All patients with leukemia receive supportive care treatments. Supportive care may include the following: This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Regular follow-up exams are very important. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their child's doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute myeloid leukemia (AML) during the induction phase may include the following: Treatment of childhood AML during the remission phase (consolidation/intensification therapy) depends on the subtype of AML and may include the following: Treatment of refractory childhood AML may include the following: Treatment of recurrent childhood AML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Transient abnormal myelopoiesis (TAM) usually goes away on its own. For newly diagnosed TAM that does not go away on its own or causes other health problems, treatment may include the following: Treatment of newly diagnosed acute myeloid leukemia (AML) in children aged 4 years or younger who have Down syndrome may include the following: Treatment of newly diagnosed AML in children older than 4 years who have Down syndrome may be the same as treatment for children without Down syndrome. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute promyelocytic leukemia (APL) may include the following: Treatment of childhood APL during the remission phase (consolidation/intensification therapy) may include the following: Treatment of childhood APL during the remission phase (maintenance therapy) may include the following: Treatment of recurrent childhood APL may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed juvenile myelomonocytic leukemia (JMML) may include the following: Treatment of refractory or recurrent childhood JMML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed chronic myelogenous leukemia (CML) may include the following: Treatment of refractory or recurrent childhood CML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood myelodysplastic syndromes (MDS) may include the following: If the MDS becomes acute myeloid leukemia (AML), treatment will be the same as treatment for newly diagnosed AML. For more information from the National Cancer Institute about childhood acute myeloid leukemia and other myeloid malignancies, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood acute myeloid leukemia and other myeloid malignancies. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/child-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389303] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies ?

Key Points - Once childhood acute myeloid leukemia (AML) has been diagnosed, tests are done to find out if the cancer has spread to other parts of the body. - There is no standard staging system for childhood AML, childhood chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), or myelodysplastic syndromes (MDS). Once childhood acute myeloid leukemia (AML) has been diagnosed, tests are done to find out if the cancer has spread to other parts of the body. The following tests and procedures may be used to determine if the leukemia has spread: - Lumbar puncture : A procedure used to collect a sample of cerebrospinal fluid (CSF) from the spinal column. This is done by placing a needle between two bones in the spine and into the CSF around the spinal cord and removing a sample of the fluid. The sample of CSF is checked under a microscope for signs that leukemia cells have spread to the brain and spinal cord. This procedure is also called an LP or spinal tap. - Biopsy of the testicles, ovaries, or skin: The removal of cells or tissues from the testicles, ovaries, or skin so they can be viewed under a microscope to check for signs of cancer. This is done only if something unusual about the testicles, ovaries, or skin is found during the physical exam. There is no standard staging system for childhood AML, childhood chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), or myelodysplastic syndromes (MDS). The extent or spread of cancer is usually described as stages. Instead of stages, treatment of childhood AML, childhood CML, JMML, and MDS is based on one or more of the following: - The type of disease or the subtype of AML. - Whether leukemia has spread outside the blood and bone marrow. - Whether the disease is newly diagnosed, in remission, or recurrent. Newly diagnosed childhood AML Newly diagnosed childhood AML has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and one of the following is true: - More than 20% of the cells in the bone marrow are blasts (leukemia cells). or - Less than 20% of the cells in the bone marrow are blasts and there is a specific change in the chromosome. Childhood AML in remission In childhood AML in remission, the disease has been treated and the following are true: - The complete blood count is almost normal. - Less than 5% of the cells in the bone marrow are blasts (leukemia cells). - There are no signs or symptoms of leukemia in the brain, spinal cord, or other parts of the body.

Childhood acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Cancers that are acute usually get worse quickly if they are not treated. Cancers that are chronic usually get worse slowly. This summary is about the treatment of childhood AML, transient abnormal myelopoiesis, childhood acute promyelocytic leukemia, juvenile myelomonocytic leukemia, childhood chronic myelogenous leukemia, and childhood myelodysplastic syndromes. For information about the treatment of childhood acute lymphoblastic leukemia, see Childhood Acute Lymphoblastic Leukemia Treatment. In healthy children, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a type of white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts, or leukemia cells, in AML are abnormal and do not become healthy white blood cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This may lead to infection, anemia, or easy bleeding. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called granulocytic sarcoma or chloroma. TAM is a disorder of the bone marrow that can develop in newborns who have Down syndrome. TAM usually goes away on its own within the first 3 months of life. Infants who have TAM have an increased chance of developing AML before the age of 3 years. TAM is also called transient myeloproliferative disorder or transient leukemia. APL is a subtype of AML. In APL, some genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. The promyelocytes (leukemia cells) can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. Problems with severe bleeding and blood clots may also occur. This is a serious health problem that needs treatment as soon as possible. JMML is a rare childhood cancer that is most common in children around the age of 2 years and is more common in boys. In JMML, too many myeloid blood stem cells become myelocytes and monocytes (two types of white blood cells). Some of these myeloid blood stem cells never become mature white blood cells. These immature cells, called blasts, are unable to do their usual work. Over time, the myelocytes, monocytes, and blasts crowd out the healthy white blood cells, red blood cells, and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. CML often begins in an early myeloid blood cell when a certain gene change occurs. A section of genes, that includes the ABL gene, on chromosome 9 changes place with a section of genes on chromosome 22, which has the BCR gene. This makes a very short chromosome 22 (called the Philadelphia chromosome) and a very long chromosome 9. An abnormal BCR-ABL gene is formed on chromosome 22. The BCR-ABL gene tells the blood cells to make too much of a protein called tyrosine kinase. Tyrosine kinase causes too many abnormal white blood cells (leukemia cells) to be made in the bone marrow. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This can lead to infection, anemia, or easy bleeding. CML is rare in children.EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. MDS occur less often in children than in adults. In MDS, the bone marrow makes too few red blood cells, white blood cells, and platelets. These blood cells may not mature and enter the blood. The type of MDS depends on the type of blood cell that is affected. The treatment for MDS depends on how low the numbers of red blood cells, white blood cells, or platelets are. Over time, MDS may become AML. Cancer treatment with certain chemotherapy drugs and/or radiation therapy may cause therapy-related AML (t-AML) or therapy-related MDS (t-MDS). The risk of these therapy-related myeloid diseases depends on the total dose of the chemotherapy drugs used and the radiation dose and treatment field. Some patients also have an inherited risk for t-AML and t-MDS. These therapy-related diseases usually occur within 7 years after treatment, but are rare in children. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. These and other factors may increase the risk of childhood AML, APL, JMML, CML, and MDS: These and other signs and symptoms may be caused by childhood AML, APL, JMML, CML, or MDS or by other conditions. Check with a doctor if your child has any of the following: The signs and symptoms of TAM may include the following: Sometimes TAM does not cause any symptoms at all and is diagnosed after a routine blood test. The following tests and procedures may be used: The following test is a type of cytogenetic analysis: The prognosis and treatment options for childhood acute myeloid leukemia (AML) depend on the following: The prognosis and treatment options for childhood acute promyelocytic leukemia (APL) depends on the following: The prognosis and treatment options for juvenile myelomonocytic leukemia (JMML) depend on the following: The prognosis and treatment options for childhood chronic myelogenous leukemia (CML) depend on the following: The prognosis and treatment options for myelodysplastic syndromes (MDS) depend on the following: The extent or spread of cancer is usually described as stages. Instead of stages, treatment is based on one or more of the following: Newly diagnosed childhood AML Newly diagnosed childhood AML is cancer that has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and has one of the following: or Childhood AML in remission In childhood AML in remission, the disease has been treated and the following are found: Refractory leukemia is cancer that does not respond to treatment. Recurrent leukemia is cancer that has recurred (come back) after it has been treated. The cancer may come back in the blood and bone marrow or in other parts of the body, such as the central nervous system (brain and spinal cord). Different types of treatment are available for children with acute myeloid leukemia (AML), transient abnormal myelopoiesis (TAM), acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia (JMML), chronic myelogenous leukemia (CML), and myelodysplastic syndromes (MDS). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because AML and other myeloid disorders are rare in children, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not yet started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists: The treatment of childhood AML is done in phases: Treatment called central nervous system (CNS) prophylaxis therapy may be given during the induction phase of therapy. Because standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the leukemia cells are able to hide in the CNS. Intrathecal chemotherapy is able to reach leukemia cells in the CNS. It is given to kill the leukemia cells and lessen the chance the leukemia will recur (come back). The treatment of childhood APL includes a third phase called maintenance. The goal of maintenance is to kill any remaining leukemia cells that may regrow and cause a relapse. Often the cancer treatments are given in lower doses than those used during the remission induction and consolidation/intensification phases. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one chemotherapy drug. The way the chemotherapy is given depends on the type of cancer being treated. In AML, chemotherapy given by mouth, vein, or into the cerebrospinal fluid is used. In AML, the leukemia cells may spread to the brain and/or spinal cord. Chemotherapy given by mouth or vein to treat AML may not cross the blood-brain barrier to get into the fluid that surrounds the brain and spinal cord. Instead, chemotherapy is injected into the fluid-filled space to kill leukemia cells that may have spread there (intrathecal chemotherapy).EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. See Drugs Approved for Acute Myeloid Leukemia for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. In childhood AML, external radiation therapy may be used to treat a myeloid sarcoma that does not respond to chemotherapy. Chemotherapy is given to kill cancer cells or other abnormal blood cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: See Drugs Approved for Leukemia for more information. Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change. Arsenic trioxide and tretinoin are drugs that kill certain types of leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of acute promyelocytic leukemia. See Drugs Approved for Acute Myeloid Leukemia for more information. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. It is sometimes used to treat transient abnormal myelopoiesis (TAM). Supportive care is given to lessen the problems caused by the disease or its treatment. All patients with leukemia receive supportive care treatments. Supportive care may include the following: This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Regular follow-up exams are very important. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their child's doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute myeloid leukemia (AML) during the induction phase may include the following: Treatment of childhood AML during the remission phase (consolidation/intensification therapy) depends on the subtype of AML and may include the following: Treatment of refractory childhood AML may include the following: Treatment of recurrent childhood AML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Transient abnormal myelopoiesis (TAM) usually goes away on its own. For newly diagnosed TAM that does not go away on its own or causes other health problems, treatment may include the following: Treatment of newly diagnosed acute myeloid leukemia (AML) in children aged 4 years or younger who have Down syndrome may include the following: Treatment of newly diagnosed AML in children older than 4 years who have Down syndrome may be the same as treatment for children without Down syndrome. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute promyelocytic leukemia (APL) may include the following: Treatment of childhood APL during the remission phase (consolidation/intensification therapy) may include the following: Treatment of childhood APL during the remission phase (maintenance therapy) may include the following: Treatment of recurrent childhood APL may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed juvenile myelomonocytic leukemia (JMML) may include the following: Treatment of refractory or recurrent childhood JMML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed chronic myelogenous leukemia (CML) may include the following: Treatment of refractory or recurrent childhood CML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood myelodysplastic syndromes (MDS) may include the following: If the MDS becomes acute myeloid leukemia (AML), treatment will be the same as treatment for newly diagnosed AML. For more information from the National Cancer Institute about childhood acute myeloid leukemia and other myeloid malignancies, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood acute myeloid leukemia and other myeloid malignancies. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/child-aml-treatment-pdq. Accessed <MM/DD/YYYY>. 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If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies ?

New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Biologic therapy Biologic therapy is a treatment that uses the patients immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the bodys natural defenses against cancer. This type of cancer treatment is also called biotherapy or immunotherapy. Natural killer (NK) cells are a type of biologic therapy. NK cells are white blood cells that can kill tumor cells. These may be taken from a donor and given to the patient by infusion to help kill leukemia cells. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Childhood acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Cancers that are acute usually get worse quickly if they are not treated. Cancers that are chronic usually get worse slowly. This summary is about the treatment of childhood AML, transient abnormal myelopoiesis, childhood acute promyelocytic leukemia, juvenile myelomonocytic leukemia, childhood chronic myelogenous leukemia, and childhood myelodysplastic syndromes. For information about the treatment of childhood acute lymphoblastic leukemia, see Childhood Acute Lymphoblastic Leukemia Treatment. In healthy children, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a type of white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts, or leukemia cells, in AML are abnormal and do not become healthy white blood cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This may lead to infection, anemia, or easy bleeding. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called granulocytic sarcoma or chloroma. TAM is a disorder of the bone marrow that can develop in newborns who have Down syndrome. TAM usually goes away on its own within the first 3 months of life. Infants who have TAM have an increased chance of developing AML before the age of 3 years. TAM is also called transient myeloproliferative disorder or transient leukemia. APL is a subtype of AML. In APL, some genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. The promyelocytes (leukemia cells) can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. Problems with severe bleeding and blood clots may also occur. This is a serious health problem that needs treatment as soon as possible. JMML is a rare childhood cancer that is most common in children around the age of 2 years and is more common in boys. In JMML, too many myeloid blood stem cells become myelocytes and monocytes (two types of white blood cells). Some of these myeloid blood stem cells never become mature white blood cells. These immature cells, called blasts, are unable to do their usual work. Over time, the myelocytes, monocytes, and blasts crowd out the healthy white blood cells, red blood cells, and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. CML often begins in an early myeloid blood cell when a certain gene change occurs. A section of genes, that includes the ABL gene, on chromosome 9 changes place with a section of genes on chromosome 22, which has the BCR gene. This makes a very short chromosome 22 (called the Philadelphia chromosome) and a very long chromosome 9. An abnormal BCR-ABL gene is formed on chromosome 22. The BCR-ABL gene tells the blood cells to make too much of a protein called tyrosine kinase. Tyrosine kinase causes too many abnormal white blood cells (leukemia cells) to be made in the bone marrow. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. This can lead to infection, anemia, or easy bleeding. CML is rare in children.EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. MDS occur less often in children than in adults. In MDS, the bone marrow makes too few red blood cells, white blood cells, and platelets. These blood cells may not mature and enter the blood. The type of MDS depends on the type of blood cell that is affected. The treatment for MDS depends on how low the numbers of red blood cells, white blood cells, or platelets are. Over time, MDS may become AML. Cancer treatment with certain chemotherapy drugs and/or radiation therapy may cause therapy-related AML (t-AML) or therapy-related MDS (t-MDS). The risk of these therapy-related myeloid diseases depends on the total dose of the chemotherapy drugs used and the radiation dose and treatment field. Some patients also have an inherited risk for t-AML and t-MDS. These therapy-related diseases usually occur within 7 years after treatment, but are rare in children. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. These and other factors may increase the risk of childhood AML, APL, JMML, CML, and MDS: These and other signs and symptoms may be caused by childhood AML, APL, JMML, CML, or MDS or by other conditions. Check with a doctor if your child has any of the following: The signs and symptoms of TAM may include the following: Sometimes TAM does not cause any symptoms at all and is diagnosed after a routine blood test. The following tests and procedures may be used: The following test is a type of cytogenetic analysis: The prognosis and treatment options for childhood acute myeloid leukemia (AML) depend on the following: The prognosis and treatment options for childhood acute promyelocytic leukemia (APL) depends on the following: The prognosis and treatment options for juvenile myelomonocytic leukemia (JMML) depend on the following: The prognosis and treatment options for childhood chronic myelogenous leukemia (CML) depend on the following: The prognosis and treatment options for myelodysplastic syndromes (MDS) depend on the following: The extent or spread of cancer is usually described as stages. Instead of stages, treatment is based on one or more of the following: Newly diagnosed childhood AML Newly diagnosed childhood AML is cancer that has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and has one of the following: or Childhood AML in remission In childhood AML in remission, the disease has been treated and the following are found: Refractory leukemia is cancer that does not respond to treatment. Recurrent leukemia is cancer that has recurred (come back) after it has been treated. The cancer may come back in the blood and bone marrow or in other parts of the body, such as the central nervous system (brain and spinal cord). Different types of treatment are available for children with acute myeloid leukemia (AML), transient abnormal myelopoiesis (TAM), acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia (JMML), chronic myelogenous leukemia (CML), and myelodysplastic syndromes (MDS). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because AML and other myeloid disorders are rare in children, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not yet started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists: The treatment of childhood AML is done in phases: Treatment called central nervous system (CNS) prophylaxis therapy may be given during the induction phase of therapy. Because standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the leukemia cells are able to hide in the CNS. Intrathecal chemotherapy is able to reach leukemia cells in the CNS. It is given to kill the leukemia cells and lessen the chance the leukemia will recur (come back). The treatment of childhood APL includes a third phase called maintenance. The goal of maintenance is to kill any remaining leukemia cells that may regrow and cause a relapse. Often the cancer treatments are given in lower doses than those used during the remission induction and consolidation/intensification phases. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one chemotherapy drug. The way the chemotherapy is given depends on the type of cancer being treated. In AML, chemotherapy given by mouth, vein, or into the cerebrospinal fluid is used. In AML, the leukemia cells may spread to the brain and/or spinal cord. Chemotherapy given by mouth or vein to treat AML may not cross the blood-brain barrier to get into the fluid that surrounds the brain and spinal cord. Instead, chemotherapy is injected into the fluid-filled space to kill leukemia cells that may have spread there (intrathecal chemotherapy).EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. See Drugs Approved for Acute Myeloid Leukemia for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. In childhood AML, external radiation therapy may be used to treat a myeloid sarcoma that does not respond to chemotherapy. Chemotherapy is given to kill cancer cells or other abnormal blood cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: See Drugs Approved for Leukemia for more information. Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change. Arsenic trioxide and tretinoin are drugs that kill certain types of leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of acute promyelocytic leukemia. See Drugs Approved for Acute Myeloid Leukemia for more information. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. It is sometimes used to treat transient abnormal myelopoiesis (TAM). Supportive care is given to lessen the problems caused by the disease or its treatment. All patients with leukemia receive supportive care treatments. Supportive care may include the following: This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Regular follow-up exams are very important. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their child's doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute myeloid leukemia (AML) during the induction phase may include the following: Treatment of childhood AML during the remission phase (consolidation/intensification therapy) depends on the subtype of AML and may include the following: Treatment of refractory childhood AML may include the following: Treatment of recurrent childhood AML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Transient abnormal myelopoiesis (TAM) usually goes away on its own. For newly diagnosed TAM that does not go away on its own or causes other health problems, treatment may include the following: Treatment of newly diagnosed acute myeloid leukemia (AML) in children aged 4 years or younger who have Down syndrome may include the following: Treatment of newly diagnosed AML in children older than 4 years who have Down syndrome may be the same as treatment for children without Down syndrome. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood acute promyelocytic leukemia (APL) may include the following: Treatment of childhood APL during the remission phase (consolidation/intensification therapy) may include the following: Treatment of childhood APL during the remission phase (maintenance therapy) may include the following: Treatment of recurrent childhood APL may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed juvenile myelomonocytic leukemia (JMML) may include the following: Treatment of refractory or recurrent childhood JMML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed chronic myelogenous leukemia (CML) may include the following: Treatment of refractory or recurrent childhood CML may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood myelodysplastic syndromes (MDS) may include the following: If the MDS becomes acute myeloid leukemia (AML), treatment will be the same as treatment for newly diagnosed AML. For more information from the National Cancer Institute about childhood acute myeloid leukemia and other myeloid malignancies, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood acute myeloid leukemia and other myeloid malignancies. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. 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What are the treatments for Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies ?

Key Points - There are different types of treatment for children with acute myeloid leukemia (AML), chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), or myelodysplastic syndromes (MDS). - Treatment is planned by a team of health care providers who are experts in treating childhood leukemia and other diseases of the blood. - Some cancer treatments cause side effects months or years after treatment has ended. - The treatment of childhood AML usually has two phases. - Seven types of standard treatment are used for childhood AML, childhood CML, JMML, or MDS. - Chemotherapy - Radiation therapy - Stem cell transplant - Targeted therapy - Other drug therapy - Watchful waiting - Supportive care - New types of treatment are being tested in clinical trials. - Biologic therapy - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for children with acute myeloid leukemia (AML), chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), or myelodysplastic syndromes (MDS). Different types of treatment are available for children with AML, CML, JMML, or MDS. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment is planned by a team of health care providers who are experts in treating childhood leukemia and other diseases of the blood. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists: - Pediatrician. - Hematologist. - Medical oncologist. - Pediatric surgeon. - Radiation oncologist. - Neurologist. - Neuropathologist. - Neuroradiologist. - Pediatric nurse specialist. - Social worker. - Rehabilitation specialist. - Psychologist. Some cancer treatments cause side effects months or years after treatment has ended. Regular follow-up exams are very important. Some cancer treatments cause side effects that continue or appear months or years after cancer treatment has ended. These are called late effects. Late effects of cancer treatment may include: - Physical problems. - Changes in mood, feelings, thinking, learning, or memory. - Second cancers (new types of cancer). Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). The treatment of childhood AML usually has two phases. The treatment of childhood AML is done in phases: - Induction therapy: This is the first phase of treatment. The goal is to kill the leukemia cells in the blood and bone marrow. This puts the leukemia into remission. - Consolidation /intensification therapy: This is the second phase of treatment. It begins once the leukemia is in remission. The goal of therapy is to kill any remaining leukemia cells that may not be active but could begin to regrow and cause a relapse. Treatment called central nervous system (CNS) sanctuary therapy may be given during the induction phase of therapy. Because standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the cells are able to find sanctuary (hide) in the CNS. Intrathecal chemotherapy is able to reach leukemia cells in the CNS. It is given to kill the leukemia cells and lessen the chance the leukemia will recur (come back). CNS sanctuary therapy is also called CNS prophylaxis. Seven types of standard treatment are used for childhood AML, childhood CML, JMML, or MDS. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type of cancer being treated. In AML, the leukemia cells may spread to the brain and/or spinal cord. Chemotherapy given by mouth or vein to treat AML may not cross the blood-brain barrier to get into the fluid that surrounds the brain and spinal cord. Instead, chemotherapy is injected into the fluid-filled space to kill leukemia cells that may have spread there (intrathecal chemotherapy). See Drugs Approved for Acute Myeloid Leukemia for more information. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type of the cancer being treated. In childhood AML, external radiation therapy may be used to treat a chloroma that does not respond to chemotherapy. Stem cell transplant Stem cell transplant is a way of giving chemotherapy and replacing blood-forming cells that are abnormal or destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body&apos;s blood cells. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Types of targeted therapy include the following: - Tyrosine kinase inhibitor therapy: Tyrosine kinase inhibitor (TKI) therapy blocks signals needed for tumors to grow. TKIs block the enzyme (tyrosine kinase) that causes stem cells to become more white blood cells (granulocytes or blasts) than the body needs. TKIs may be used with other anticancer drugs as adjuvant therapy (treatment given after the initial treatment, to lower the risk that the cancer will come back). - Imatinib is a type of TKI that is approved to treat childhood CML. - Sorafenib, dasatinib, and nilotinib are being studied in the treatment of childhood leukemia. - Monoclonal antibody therapy: Monoclonal antibody therapy uses antibodies made in the laboratory, from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. - Gemtuzumab is a type of monoclonal antibody used in the treatment of a subtype of AML called acute promyelocytic leukemia (APL). Gemtuzumab is not available in the United States unless special approval is given. Monoclonal antibodies may be used with chemotherapy as adjuvant therapy. - Proteasome inhibitor therapy: Proteasome inhibitors break down proteins in cancer cells and kill them. - Bortezomib is a proteasome inhibitor used to treat childhood APL. See Drugs Approved for Leukemia for more information. Other drug therapy Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change. Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of acute promyelocytic leukemia. See Drugs Approved for Acute Myeloid Leukemia for more information. Watchful waiting Watchful waiting is closely monitoring a patients condition without giving any treatment until signs or symptoms appear or change. It is sometimes used to treat MDS or TMD. Supportive care Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include the following: - Transfusion therapy: A way of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. The blood may be donated from another person or it may have been taken from the patient earlier and stored until needed. - Drug therapy, such as antibiotics or antifungal agents. - Leukapheresis: A procedure in which a special machine is used to remove white blood cells from the blood. Blood is taken from the patient and put through a blood cell separator where the white blood cells are removed. The rest of the blood is then returned to the patient&apos;s bloodstream. New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Biologic therapy Biologic therapy is a treatment that uses the patients immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the bodys natural defenses against cancer. This type of cancer treatment is also called biotherapy or immunotherapy. Natural killer (NK) cells are a type of biologic therapy. NK cells are white blood cells that can kill tumor cells. These may be taken from a donor and given to the patient by infusion to help kill leukemia cells. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child&apos;s condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Childhood Acute Myeloid Leukemia, Childhood Chronic Myelogenous Leukemia, Juvenile Myelomonocytic Leukemia, and Myelodysplastic Syndromes Newly Diagnosed Childhood Acute Myeloid Leukemia Treatment of newly diagnosed childhood acute myeloid leukemia may include the following: - Combination chemotherapy plus central nervous system sanctuary therapy with intrathecal chemotherapy. - A clinical trial comparing different chemotherapy regimens (doses and schedules of treatment). - A clinical trial of combination chemotherapy and targeted therapy with a proteasome inhibitor or a tyrosine kinase inhibitor with or without stem cell transplant. Treatment of newly diagnosed childhood acute leukemia with a granulocytic sarcoma (chloroma) may include chemotherapy with or without radiation therapy. Treatment of therapy -related AML is usually the same as for newly diagnosed AML, followed by stem cell transplant. Check the list of NCI-supported cancer clinical trials that are now accepting patients with untreated childhood acute myeloid leukemia and other myeloid malignancies. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Children with Newly Diagnosed Childhood AML and Down Syndrome Treatment of acute myeloid leukemia (AML) in children aged 4 years or younger who have Down syndrome may include the following: - Combination chemotherapy plus central nervous system sanctuary therapy with intrathecal chemotherapy. Treatment of AML in children older than 4 years who have Down syndrome may be the same as treatment for children without Down syndrome. Childhood Acute Myeloid Leukemia in Remission Treatment of childhood acute myeloid leukemia (AML) during the remission phase (consolidation /intensification therapy) depends on the subtype of AML and may include the following: - Combination chemotherapy. - High-dose chemotherapy followed by stem cell transplant using blood stem cells from a donor. - A clinical trial of chemotherapy followed by an infusion of natural killer cells. - A clinical trial of combination chemotherapy and targeted therapy with a proteasome inhibitor or a tyrosine kinase inhibitor with or without stem cell transplant. Check the list of NCI-supported cancer clinical trials that are now accepting patients with childhood acute myeloid leukemia in remission. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Recurrent Childhood Acute Myeloid Leukemia Treatment of recurrent childhood acute myeloid leukemia (AML) may include the following: - Combination chemotherapy. - Combination chemotherapy and stem cell transplant. - A second stem cell transplant. - A clinical trial of combinations of new anticancer drugs, new biologic agents, and stem cell transplant using different sources of stem cells. Treatment of recurrent AML in children with Down syndrome is chemotherapy. It is not clear if stem cell transplant after chemotherapy is helpful in treating these children. Check the list of NCI-supported cancer clinical trials that are now accepting patients with recurrent childhood acute myeloid leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Acute Promyelocytic Leukemia Treatment of acute promyelocytic leukemia may include the following: - All-trans retinoic acid (ATRA) plus chemotherapy. - Arsenic trioxide therapy. - Central nervous system sanctuary therapy with intrathecal chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with childhood acute promyelocytic leukemia (M3). For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Recurrent Acute Promyelocytic Leukemia Treatment of recurrent acute promyelocytic leukemia may include the following: - All-trans retinoic acid therapy (ATRA) plus chemotherapy. - Arsenic trioxide therapy. - Targeted therapy with a monoclonal antibody (gemtuzumab), if special approval is given. - Stem cell transplant using blood stem cells from the patient or a donor. Childhood Chronic Myelogenous Leukemia Treatment for childhood chronic myelogenous leukemia may include the following: - Targeted therapy with a tyrosine kinase inhibitor (imatinib). - A clinical trial of targeted therapy with other tyrosine kinase inhibitors. For patients whose disease does not respond to therapy with imatinib or whose disease comes back after treatment, treatment may include the following: - Stem cell transplant using blood stem cells from a donor. - A clinical trial of targeted therapy with other tyrosine kinase inhibitors. Check the list of NCI-supported cancer clinical trials that are now accepting patients with childhood chronic myelogenous leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Juvenile Myelomonocytic Leukemia Treatment of juvenile myelomonocytic leukemia (JMML) may include the following: - Combination chemotherapy followed by stem cell transplant. If JMML recurs after stem cell transplant, a second stem cell transplant may be done. Check the list of NCI-supported cancer clinical trials that are now accepting patients with juvenile myelomonocytic leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Myelodysplastic Syndromes Treatment of myelodysplastic syndromes (MDS) may include the following: - Watchful waiting. - Stem cell transplant using blood stem cells from a donor. - Combination chemotherapy. - Lenalidomide therapy. - A clinical trial of stem cell transplant using lower doses of chemotherapy. - A clinical trial of a new anticancer drug or targeted therapy. If the MDS becomes acute myeloid leukemia (AML), treatment will be the same as treatment for newly diagnosed AML. Treatment of therapy-related MDS is usually the same as for newly diagnosed AML, followed by stem cell transplant. Transient myeloproliferative disorder (TMD), a type of MDS, usually goes away on its own. For TMD that does not go away on its own, treatment may include the following: - Transfusion therapy. - Leukapheresis. - Chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with childhood myelodysplastic syndromes. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website.

Germ cell tumors begin in the reproductive cells (egg or sperm) of the body. Ovarian germ cell tumors usually occur in teenage girls or young women and most often affect just one ovary. The ovaries are a pair of organs in the female reproductive system. They are in the pelvis, one on each side of the uterus (the hollow, pear-shaped organ where a fetus grows). Each ovary is about the size and shape of an almond. The ovaries make eggs and female hormones. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Ovarian germ cell tumor is a general name that is used to describe several different types of cancer. The most common ovarian germ cell tumor is called dysgerminoma. See the following PDQ summaries for information about other types of ovarian tumors: Ovarian germ cell tumors can be hard to diagnose (find) early. Often there are no symptoms in the early stages, but tumors may be found during regular gynecologic exams (checkups). Check with your doctor if you have either of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: Ovarian germ cell tumors are usually cured if found and treated early. The process used to find out whether cancer has spread within the ovary or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. Unless a doctor is sure the cancer has spread from the ovaries to other parts of the body, an operation called a laparotomy is done to see if the cancer has spread. The doctor must cut into the abdomen and carefully look at all the organs to see if they have cancer in them. The doctor will cut out small pieces of tissue so they can be checked under a microscope for signs of cancer. The doctor may also wash the abdominal cavity with fluid, which is also checked under a microscope to see if it has cancer cells in it. Usually the doctor will remove the cancer and other organs that have cancer in them during the laparotomy. It is important to know the stage in order to plan treatment. Many of the tests used to diagnose ovarian germ cell tumor are also used for staging. The following tests and procedures may also be used for staging: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of tumor as the primary tumor. For example, if an ovarian germ cell tumor spreads to the liver, the tumor cells in the liver are actually cancerous ovarian germ cells. The disease is metastatic ovarian germ cell tumor, not liver cancer. In stage I, cancer is found in one or both ovaries or fallopian tubes. Stage I is divided into stage IA, stage IB, and stage IC. In stage II, cancer is found in one or both ovaries or fallopian tubes and has spread into other areas of the pelvis, or primary peritoneal cancer is found within the pelvis. Stage II is divided into stage IIA and stage IIB. In stage III, cancer is found in one or both ovaries or fallopian tubes, or is primary peritoneal cancer, and has spread outside the pelvis to other parts of the abdomen and/or to nearby lymph nodes. Stage III is divided into stage IIIA, stage IIIB, and stage IIIC. In stage IV, cancer has spread beyond the abdomen to other parts of the body. Stage IV is divided into stage IVA and stage IVB. The cancer may come back in the other ovary or in other parts of the body. Different types of treatment are available for patients with ovarian germ cell tumor. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of ovarian germ cell tumor. A doctor may take out the cancer using one of the following types of surgery. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. After chemotherapy for an ovarian germ cell tumor, a second-look laparotomy may be done. This is similar to the laparotomy that is done to find out the stage of the cancer. Second-look laparotomy is a surgical procedure to find out if tumor cells are left after primary treatment. During this procedure, the doctor will take samples of lymph nodes and other tissues in the abdomen to see if any cancer is left. This procedure is not done for dysgerminomas. Observation is closely watching a patient’s condition without giving any treatment unless signs or symptoms appear or change. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Ovarian, Fallopian Tube, or Primary Peritoneal Cancer for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy with bone marrow transplant is a method of giving very high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells. Combination chemotherapy (the use of more than one anticancer drug) is being tested in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may be either: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be either: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about ovarian germ cell tumors, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of ovarian germ cell tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Ovarian Germ Cell Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/ovarian/patient/ovarian-germ-cell-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389363] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Ovarian Germ Cell Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Ovarian Germ Cell Tumors ?

Key Points - Ovarian germ cell tumor is a disease in which malignant (cancer) cells form in the germ (egg) cells of the ovary. - Signs of ovarian germ cell tumor are swelling of the abdomen or vaginal bleeding after menopause. - Tests that examine the ovaries, pelvic area, blood, and ovarian tissue are used to detect (find) and diagnose ovarian germ cell tumor. - Certain factors affect prognosis (chance of recovery and treatment options). Ovarian germ cell tumor is a disease in which malignant (cancer) cells form in the germ (egg) cells of the ovary. Germ cell tumors begin in the reproductive cells (egg or sperm) of the body. Ovarian germ cell tumors usually occur in teenage girls or young women and most often affect just one ovary. The ovaries are a pair of organs in the female reproductive system. They are in the pelvis, one on each side of the uterus (the hollow, pear-shaped organ where a fetus grows). Each ovary is about the size and shape of an almond. The ovaries make eggs and female hormones. Ovarian germ cell tumor is a general name that is used to describe several different types of cancer. The most common ovarian germ cell tumor is called dysgerminoma. See the following PDQ summaries for information about other types of ovarian tumors: - Ovarian Epithelial, Fallopian Tube, and Primary Peritoneal Cancer Treatment - Ovarian Low Malignant Potential Tumors Treatment

Germ cell tumors begin in the reproductive cells (egg or sperm) of the body. Ovarian germ cell tumors usually occur in teenage girls or young women and most often affect just one ovary. The ovaries are a pair of organs in the female reproductive system. They are in the pelvis, one on each side of the uterus (the hollow, pear-shaped organ where a fetus grows). Each ovary is about the size and shape of an almond. The ovaries make eggs and female hormones. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Ovarian germ cell tumor is a general name that is used to describe several different types of cancer. The most common ovarian germ cell tumor is called dysgerminoma. See the following PDQ summaries for information about other types of ovarian tumors: Ovarian germ cell tumors can be hard to diagnose (find) early. Often there are no symptoms in the early stages, but tumors may be found during regular gynecologic exams (checkups). Check with your doctor if you have either of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: Ovarian germ cell tumors are usually cured if found and treated early. The process used to find out whether cancer has spread within the ovary or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. Unless a doctor is sure the cancer has spread from the ovaries to other parts of the body, an operation called a laparotomy is done to see if the cancer has spread. The doctor must cut into the abdomen and carefully look at all the organs to see if they have cancer in them. The doctor will cut out small pieces of tissue so they can be checked under a microscope for signs of cancer. The doctor may also wash the abdominal cavity with fluid, which is also checked under a microscope to see if it has cancer cells in it. Usually the doctor will remove the cancer and other organs that have cancer in them during the laparotomy. It is important to know the stage in order to plan treatment. Many of the tests used to diagnose ovarian germ cell tumor are also used for staging. The following tests and procedures may also be used for staging: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of tumor as the primary tumor. For example, if an ovarian germ cell tumor spreads to the liver, the tumor cells in the liver are actually cancerous ovarian germ cells. The disease is metastatic ovarian germ cell tumor, not liver cancer. In stage I, cancer is found in one or both ovaries or fallopian tubes. Stage I is divided into stage IA, stage IB, and stage IC. In stage II, cancer is found in one or both ovaries or fallopian tubes and has spread into other areas of the pelvis, or primary peritoneal cancer is found within the pelvis. Stage II is divided into stage IIA and stage IIB. In stage III, cancer is found in one or both ovaries or fallopian tubes, or is primary peritoneal cancer, and has spread outside the pelvis to other parts of the abdomen and/or to nearby lymph nodes. Stage III is divided into stage IIIA, stage IIIB, and stage IIIC. In stage IV, cancer has spread beyond the abdomen to other parts of the body. Stage IV is divided into stage IVA and stage IVB. The cancer may come back in the other ovary or in other parts of the body. Different types of treatment are available for patients with ovarian germ cell tumor. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of ovarian germ cell tumor. A doctor may take out the cancer using one of the following types of surgery. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. After chemotherapy for an ovarian germ cell tumor, a second-look laparotomy may be done. This is similar to the laparotomy that is done to find out the stage of the cancer. Second-look laparotomy is a surgical procedure to find out if tumor cells are left after primary treatment. During this procedure, the doctor will take samples of lymph nodes and other tissues in the abdomen to see if any cancer is left. This procedure is not done for dysgerminomas. Observation is closely watching a patient’s condition without giving any treatment unless signs or symptoms appear or change. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Ovarian, Fallopian Tube, or Primary Peritoneal Cancer for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy with bone marrow transplant is a method of giving very high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells. Combination chemotherapy (the use of more than one anticancer drug) is being tested in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may be either: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be either: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about ovarian germ cell tumors, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of ovarian germ cell tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Ovarian Germ Cell Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/ovarian/patient/ovarian-germ-cell-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389363] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Ovarian Germ Cell Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Ovarian Germ Cell Tumors ?

Signs of ovarian germ cell tumor are swelling of the abdomen or vaginal bleeding after menopause. Ovarian germ cell tumors can be hard to diagnose (find) early. Often there are no symptoms in the early stages, but tumors may be found during regular gynecologic exams (checkups). Check with your doctor if you have either of the following: - Swollen abdomen without weight gain in other parts of the body. - Bleeding from the vagina after menopause (when you are no longer having menstrual periods).

Germ cell tumors begin in the reproductive cells (egg or sperm) of the body. Ovarian germ cell tumors usually occur in teenage girls or young women and most often affect just one ovary. The ovaries are a pair of organs in the female reproductive system. They are in the pelvis, one on each side of the uterus (the hollow, pear-shaped organ where a fetus grows). Each ovary is about the size and shape of an almond. The ovaries make eggs and female hormones. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Ovarian germ cell tumor is a general name that is used to describe several different types of cancer. The most common ovarian germ cell tumor is called dysgerminoma. See the following PDQ summaries for information about other types of ovarian tumors: Ovarian germ cell tumors can be hard to diagnose (find) early. Often there are no symptoms in the early stages, but tumors may be found during regular gynecologic exams (checkups). Check with your doctor if you have either of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: Ovarian germ cell tumors are usually cured if found and treated early. The process used to find out whether cancer has spread within the ovary or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. Unless a doctor is sure the cancer has spread from the ovaries to other parts of the body, an operation called a laparotomy is done to see if the cancer has spread. The doctor must cut into the abdomen and carefully look at all the organs to see if they have cancer in them. The doctor will cut out small pieces of tissue so they can be checked under a microscope for signs of cancer. The doctor may also wash the abdominal cavity with fluid, which is also checked under a microscope to see if it has cancer cells in it. Usually the doctor will remove the cancer and other organs that have cancer in them during the laparotomy. It is important to know the stage in order to plan treatment. Many of the tests used to diagnose ovarian germ cell tumor are also used for staging. The following tests and procedures may also be used for staging: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of tumor as the primary tumor. For example, if an ovarian germ cell tumor spreads to the liver, the tumor cells in the liver are actually cancerous ovarian germ cells. The disease is metastatic ovarian germ cell tumor, not liver cancer. In stage I, cancer is found in one or both ovaries or fallopian tubes. Stage I is divided into stage IA, stage IB, and stage IC. In stage II, cancer is found in one or both ovaries or fallopian tubes and has spread into other areas of the pelvis, or primary peritoneal cancer is found within the pelvis. Stage II is divided into stage IIA and stage IIB. In stage III, cancer is found in one or both ovaries or fallopian tubes, or is primary peritoneal cancer, and has spread outside the pelvis to other parts of the abdomen and/or to nearby lymph nodes. Stage III is divided into stage IIIA, stage IIIB, and stage IIIC. In stage IV, cancer has spread beyond the abdomen to other parts of the body. Stage IV is divided into stage IVA and stage IVB. The cancer may come back in the other ovary or in other parts of the body. Different types of treatment are available for patients with ovarian germ cell tumor. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of ovarian germ cell tumor. A doctor may take out the cancer using one of the following types of surgery. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. After chemotherapy for an ovarian germ cell tumor, a second-look laparotomy may be done. This is similar to the laparotomy that is done to find out the stage of the cancer. Second-look laparotomy is a surgical procedure to find out if tumor cells are left after primary treatment. During this procedure, the doctor will take samples of lymph nodes and other tissues in the abdomen to see if any cancer is left. This procedure is not done for dysgerminomas. Observation is closely watching a patient’s condition without giving any treatment unless signs or symptoms appear or change. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Ovarian, Fallopian Tube, or Primary Peritoneal Cancer for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy with bone marrow transplant is a method of giving very high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells. Combination chemotherapy (the use of more than one anticancer drug) is being tested in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may be either: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be either: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about ovarian germ cell tumors, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of ovarian germ cell tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Ovarian Germ Cell Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/ovarian/patient/ovarian-germ-cell-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389363] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Ovarian Germ Cell Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Ovarian Germ Cell Tumors ?

Tests that examine the ovaries, pelvic area, blood, and ovarian tissue are used to detect (find) and diagnose ovarian germ cell tumor. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Pelvic exam : An exam of the vagina, cervix, uterus, fallopian tubes, ovaries, and rectum. A speculum is inserted into the vagina and the doctor or nurse looks at the vagina and cervix for signs of disease. A Pap test of the cervix is usually done. The doctor or nurse also inserts one or two lubricated, gloved fingers of one hand into the vagina and places the other hand over the lower abdomen to feel the size, shape, and position of the uterus and ovaries. The doctor or nurse also inserts a lubricated, gloved finger into the rectum to feel for lumps or abnormal areas. - Laparotomy : A surgical procedure in which an incision (cut) is made in the wall of the abdomen to check the inside of the abdomen for signs of disease. The size of the incision depends on the reason the laparotomy is being done. Sometimes organs are removed or tissue samples are taken and checked under a microscope for signs of disease. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - Serum tumor marker test : A procedure in which a sample of blood is checked to measure the amounts of certain substances released into the blood by organs, tissues, or tumor cells in the body. Certain substances are linked to specific types of cancer when found in increased levels in the blood. These are called tumor markers. An increased level of alpha fetoprotein (AFP) or human chorionic gonadotropin (HCG) in the blood may be a sign of ovarian germ cell tumor.

Germ cell tumors begin in the reproductive cells (egg or sperm) of the body. Ovarian germ cell tumors usually occur in teenage girls or young women and most often affect just one ovary. The ovaries are a pair of organs in the female reproductive system. They are in the pelvis, one on each side of the uterus (the hollow, pear-shaped organ where a fetus grows). Each ovary is about the size and shape of an almond. The ovaries make eggs and female hormones. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Ovarian germ cell tumor is a general name that is used to describe several different types of cancer. The most common ovarian germ cell tumor is called dysgerminoma. See the following PDQ summaries for information about other types of ovarian tumors: Ovarian germ cell tumors can be hard to diagnose (find) early. Often there are no symptoms in the early stages, but tumors may be found during regular gynecologic exams (checkups). Check with your doctor if you have either of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: Ovarian germ cell tumors are usually cured if found and treated early. The process used to find out whether cancer has spread within the ovary or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. Unless a doctor is sure the cancer has spread from the ovaries to other parts of the body, an operation called a laparotomy is done to see if the cancer has spread. The doctor must cut into the abdomen and carefully look at all the organs to see if they have cancer in them. The doctor will cut out small pieces of tissue so they can be checked under a microscope for signs of cancer. The doctor may also wash the abdominal cavity with fluid, which is also checked under a microscope to see if it has cancer cells in it. Usually the doctor will remove the cancer and other organs that have cancer in them during the laparotomy. It is important to know the stage in order to plan treatment. Many of the tests used to diagnose ovarian germ cell tumor are also used for staging. The following tests and procedures may also be used for staging: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of tumor as the primary tumor. For example, if an ovarian germ cell tumor spreads to the liver, the tumor cells in the liver are actually cancerous ovarian germ cells. The disease is metastatic ovarian germ cell tumor, not liver cancer. In stage I, cancer is found in one or both ovaries or fallopian tubes. Stage I is divided into stage IA, stage IB, and stage IC. In stage II, cancer is found in one or both ovaries or fallopian tubes and has spread into other areas of the pelvis, or primary peritoneal cancer is found within the pelvis. Stage II is divided into stage IIA and stage IIB. In stage III, cancer is found in one or both ovaries or fallopian tubes, or is primary peritoneal cancer, and has spread outside the pelvis to other parts of the abdomen and/or to nearby lymph nodes. Stage III is divided into stage IIIA, stage IIIB, and stage IIIC. In stage IV, cancer has spread beyond the abdomen to other parts of the body. Stage IV is divided into stage IVA and stage IVB. The cancer may come back in the other ovary or in other parts of the body. Different types of treatment are available for patients with ovarian germ cell tumor. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of ovarian germ cell tumor. A doctor may take out the cancer using one of the following types of surgery. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. After chemotherapy for an ovarian germ cell tumor, a second-look laparotomy may be done. This is similar to the laparotomy that is done to find out the stage of the cancer. Second-look laparotomy is a surgical procedure to find out if tumor cells are left after primary treatment. During this procedure, the doctor will take samples of lymph nodes and other tissues in the abdomen to see if any cancer is left. This procedure is not done for dysgerminomas. Observation is closely watching a patient’s condition without giving any treatment unless signs or symptoms appear or change. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Ovarian, Fallopian Tube, or Primary Peritoneal Cancer for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy with bone marrow transplant is a method of giving very high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells. Combination chemotherapy (the use of more than one anticancer drug) is being tested in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may be either: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be either: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about ovarian germ cell tumors, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of ovarian germ cell tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Ovarian Germ Cell Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/ovarian/patient/ovarian-germ-cell-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389363] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Ovarian Germ Cell Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Ovarian Germ Cell Tumors ?

Certain factors affect prognosis (chance of recovery and treatment options). The prognosis (chance of recovery) and treatment options depend on the following: - The type of cancer. - The size of the tumor. - The stage of cancer (whether it affects part of the ovary, involves the whole ovary, or has spread to other places in the body). - The way the cancer cells look under a microscope. - The patients general health. Ovarian germ cell tumors are usually cured if found and treated early.

Germ cell tumors begin in the reproductive cells (egg or sperm) of the body. Ovarian germ cell tumors usually occur in teenage girls or young women and most often affect just one ovary. The ovaries are a pair of organs in the female reproductive system. They are in the pelvis, one on each side of the uterus (the hollow, pear-shaped organ where a fetus grows). Each ovary is about the size and shape of an almond. The ovaries make eggs and female hormones. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Ovarian germ cell tumor is a general name that is used to describe several different types of cancer. The most common ovarian germ cell tumor is called dysgerminoma. See the following PDQ summaries for information about other types of ovarian tumors: Ovarian germ cell tumors can be hard to diagnose (find) early. Often there are no symptoms in the early stages, but tumors may be found during regular gynecologic exams (checkups). Check with your doctor if you have either of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: Ovarian germ cell tumors are usually cured if found and treated early. The process used to find out whether cancer has spread within the ovary or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. Unless a doctor is sure the cancer has spread from the ovaries to other parts of the body, an operation called a laparotomy is done to see if the cancer has spread. The doctor must cut into the abdomen and carefully look at all the organs to see if they have cancer in them. The doctor will cut out small pieces of tissue so they can be checked under a microscope for signs of cancer. The doctor may also wash the abdominal cavity with fluid, which is also checked under a microscope to see if it has cancer cells in it. Usually the doctor will remove the cancer and other organs that have cancer in them during the laparotomy. It is important to know the stage in order to plan treatment. Many of the tests used to diagnose ovarian germ cell tumor are also used for staging. The following tests and procedures may also be used for staging: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of tumor as the primary tumor. For example, if an ovarian germ cell tumor spreads to the liver, the tumor cells in the liver are actually cancerous ovarian germ cells. The disease is metastatic ovarian germ cell tumor, not liver cancer. In stage I, cancer is found in one or both ovaries or fallopian tubes. Stage I is divided into stage IA, stage IB, and stage IC. In stage II, cancer is found in one or both ovaries or fallopian tubes and has spread into other areas of the pelvis, or primary peritoneal cancer is found within the pelvis. Stage II is divided into stage IIA and stage IIB. In stage III, cancer is found in one or both ovaries or fallopian tubes, or is primary peritoneal cancer, and has spread outside the pelvis to other parts of the abdomen and/or to nearby lymph nodes. Stage III is divided into stage IIIA, stage IIIB, and stage IIIC. In stage IV, cancer has spread beyond the abdomen to other parts of the body. Stage IV is divided into stage IVA and stage IVB. The cancer may come back in the other ovary or in other parts of the body. Different types of treatment are available for patients with ovarian germ cell tumor. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of ovarian germ cell tumor. A doctor may take out the cancer using one of the following types of surgery. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. After chemotherapy for an ovarian germ cell tumor, a second-look laparotomy may be done. This is similar to the laparotomy that is done to find out the stage of the cancer. Second-look laparotomy is a surgical procedure to find out if tumor cells are left after primary treatment. During this procedure, the doctor will take samples of lymph nodes and other tissues in the abdomen to see if any cancer is left. This procedure is not done for dysgerminomas. Observation is closely watching a patient’s condition without giving any treatment unless signs or symptoms appear or change. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Ovarian, Fallopian Tube, or Primary Peritoneal Cancer for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy with bone marrow transplant is a method of giving very high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells. Combination chemotherapy (the use of more than one anticancer drug) is being tested in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may be either: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be either: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about ovarian germ cell tumors, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of ovarian germ cell tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Ovarian Germ Cell Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/ovarian/patient/ovarian-germ-cell-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389363] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Ovarian Germ Cell Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Ovarian Germ Cell Tumors ?

Key Points - After ovarian germ cell tumor has been diagnosed, tests are done to find out if cancer cells have spread within the ovary or to other parts of the body. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - The following stages are used for ovarian germ cell tumors: - Stage I - Stage II - Stage III - Stage IV After ovarian germ cell tumor has been diagnosed, tests are done to find out if cancer cells have spread within the ovary or to other parts of the body. The process used to find out whether cancer has spread within the ovary or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. Unless a doctor is sure the cancer has spread from the ovaries to other parts of the body, an operation called a laparotomy is done to see if the cancer has spread. The doctor must cut into the abdomen and carefully look at all the organs to see if they have cancer in them. The doctor will cut out small pieces of tissue so they can be checked under a microscope for signs of cancer. The doctor may also wash the abdominal cavity with fluid, which is also checked under a microscope to see if it has cancer cells in it. Usually the doctor will remove the cancer and other organs that have cancer in them during the laparotomy. It is important to know the stage in order to plan treatment. Many of the tests used to diagnose ovarian germ cell tumor are also used for staging. The following tests and procedures may also be used for staging: - PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). - Transvaginal ultrasound exam: A procedure used to examine the vagina, uterus, fallopian tubes, and bladder. An ultrasound transducer (probe) is inserted into the vagina and used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The doctor can identify tumors by looking at the sonogram. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of tumor as the primary tumor. For example, if an ovarian germ cell tumor spreads to the liver, the tumor cells in the liver are actually cancerous ovarian germ cells. The disease is metastatic ovarian germ cell tumor, not liver cancer. The following stages are used for ovarian germ cell tumors: Stage I In stage I, cancer is found in one or both ovaries. Stage I is divided into stage IA, stage IB, and stage IC. - Stage IA: Cancer is found inside a single ovary. - Stage IB: Cancer is found inside both ovaries. - Stage IC: Cancer is found inside one or both ovaries and one of the following is true: - cancer is also found on the outside surface of one or both ovaries; or - the capsule (outer covering) of the ovary has ruptured (broken open); or - cancer cells are found in the fluid of the peritoneal cavity (the body cavity that contains most of the organs in the abdomen) or in washings of the peritoneum (tissue lining the peritoneal cavity). Stage II In stage II, cancer is found in one or both ovaries and has spread into other areas of the pelvis. Stage II is divided into stage IIA, stage IIB, and stage IIC. - Stage IIA: Cancer has spread to the uterus and/or fallopian tubes (the long slender tubes through which eggs pass from the ovaries to the uterus). - Stage IIB: Cancer has spread to other tissue within the pelvis. - Stage IIC: Cancer is found inside one or both ovaries and has spread to the uterus and/or fallopian tubes, or to other tissue within the pelvis. Also, one of the following is true: - cancer is found on the outside surface of one or both ovaries; or - the capsule (outer covering) of the ovary has ruptured (broken open); or - cancer cells are found in the fluid of the peritoneal cavity (the body cavity that contains most of the organs in the abdomen) or in washings of the peritoneum (tissue lining the peritoneal cavity). Stage III In stage III, cancer is found in one or both ovaries and has spread outside the pelvis to other parts of the abdomen and/or nearby lymph nodes. Stage III is divided into stage IIIA, stage IIIB, and stage IIIC. - Stage IIIA: The tumor is found in the pelvis only, but cancer cells that can be seen only with a microscope have spread to the surface of the peritoneum (tissue that lines the abdominal wall and covers most of the organs in the abdomen), the small intestines, or the tissue that connects the small intestines to the wall of the abdomen. - Stage IIIB: Cancer has spread to the peritoneum and the cancer in the peritoneum is 2 centimeters or smaller. - Stage IIIC: Cancer has spread to the peritoneum and the cancer in the peritoneum is larger than 2 centimeters and/or cancer has spread to lymph nodes in the abdomen. Cancer that has spread to the surface of the liver is also considered stage III ovarian cancer. Stage IV In stage IV, cancer has spread beyond the abdomen to other parts of the body, such as the lungs or tissue inside the liver. Cancer cells in the fluid around the lungs is also considered stage IV ovarian cancer.

Germ cell tumors begin in the reproductive cells (egg or sperm) of the body. Ovarian germ cell tumors usually occur in teenage girls or young women and most often affect just one ovary. The ovaries are a pair of organs in the female reproductive system. They are in the pelvis, one on each side of the uterus (the hollow, pear-shaped organ where a fetus grows). Each ovary is about the size and shape of an almond. The ovaries make eggs and female hormones. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Ovarian germ cell tumor is a general name that is used to describe several different types of cancer. The most common ovarian germ cell tumor is called dysgerminoma. See the following PDQ summaries for information about other types of ovarian tumors: Ovarian germ cell tumors can be hard to diagnose (find) early. Often there are no symptoms in the early stages, but tumors may be found during regular gynecologic exams (checkups). Check with your doctor if you have either of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: Ovarian germ cell tumors are usually cured if found and treated early. The process used to find out whether cancer has spread within the ovary or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. Unless a doctor is sure the cancer has spread from the ovaries to other parts of the body, an operation called a laparotomy is done to see if the cancer has spread. The doctor must cut into the abdomen and carefully look at all the organs to see if they have cancer in them. The doctor will cut out small pieces of tissue so they can be checked under a microscope for signs of cancer. The doctor may also wash the abdominal cavity with fluid, which is also checked under a microscope to see if it has cancer cells in it. Usually the doctor will remove the cancer and other organs that have cancer in them during the laparotomy. It is important to know the stage in order to plan treatment. Many of the tests used to diagnose ovarian germ cell tumor are also used for staging. The following tests and procedures may also be used for staging: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of tumor as the primary tumor. For example, if an ovarian germ cell tumor spreads to the liver, the tumor cells in the liver are actually cancerous ovarian germ cells. The disease is metastatic ovarian germ cell tumor, not liver cancer. In stage I, cancer is found in one or both ovaries or fallopian tubes. Stage I is divided into stage IA, stage IB, and stage IC. In stage II, cancer is found in one or both ovaries or fallopian tubes and has spread into other areas of the pelvis, or primary peritoneal cancer is found within the pelvis. Stage II is divided into stage IIA and stage IIB. In stage III, cancer is found in one or both ovaries or fallopian tubes, or is primary peritoneal cancer, and has spread outside the pelvis to other parts of the abdomen and/or to nearby lymph nodes. Stage III is divided into stage IIIA, stage IIIB, and stage IIIC. In stage IV, cancer has spread beyond the abdomen to other parts of the body. Stage IV is divided into stage IVA and stage IVB. The cancer may come back in the other ovary or in other parts of the body. Different types of treatment are available for patients with ovarian germ cell tumor. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of ovarian germ cell tumor. A doctor may take out the cancer using one of the following types of surgery. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. After chemotherapy for an ovarian germ cell tumor, a second-look laparotomy may be done. This is similar to the laparotomy that is done to find out the stage of the cancer. Second-look laparotomy is a surgical procedure to find out if tumor cells are left after primary treatment. During this procedure, the doctor will take samples of lymph nodes and other tissues in the abdomen to see if any cancer is left. This procedure is not done for dysgerminomas. Observation is closely watching a patient’s condition without giving any treatment unless signs or symptoms appear or change. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Ovarian, Fallopian Tube, or Primary Peritoneal Cancer for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy with bone marrow transplant is a method of giving very high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells. Combination chemotherapy (the use of more than one anticancer drug) is being tested in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may be either: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be either: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about ovarian germ cell tumors, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of ovarian germ cell tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Ovarian Germ Cell Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/ovarian/patient/ovarian-germ-cell-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389363] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Ovarian Germ Cell Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Ovarian Germ Cell Tumors ?

Key Points - There are different types of treatment for patients with ovarian germ cell tumors. - Four types of standard treatment are used: - Surgery - Observation - Chemotherapy - Radiation therapy - New types of treatment are being tested in clinical trials. - High-dose chemotherapy with bone marrow transplant - New treatment options - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with ovarian germ cell tumors. Different types of treatment are available for patients with ovarian germ cell tumor. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Four types of standard treatment are used: Surgery Surgery is the most common treatment of ovarian germ cell tumor. A doctor may take out the cancer using one of the following types of surgery. - Unilateral salpingo-oophorectomy: A surgical procedure to remove one ovary and one fallopian tube. - Total hysterectomy: A surgical procedure to remove the uterus, including the cervix. If the uterus and cervix are taken out through the vagina, the operation is called a vaginal hysterectomy. If the uterus and cervix are taken out through a large incision (cut) in the abdomen, the operation is called a total abdominal hysterectomy. If the uterus and cervix are taken out through a small incision (cut) in the abdomen using a laparoscope, the operation is called a total laparoscopic hysterectomy. - Bilateral salpingo-oophorectomy: A surgical procedure to remove both ovaries and both fallopian tubes. - Tumor debulking: A surgical procedure in which as much of the tumor as possible is removed. Some tumors cannot be completely removed. Even if the doctor removes all the cancer that can be seen at the time of the operation, some patients may be offered chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. After chemotherapy for an ovarian germ cell tumor, a second-look laparotomy may be done. This is similar to the laparotomy that is done to find out the stage of the cancer. Second-look laparotomy is a surgical procedure to find out if tumor cells are left after primary treatment. During this procedure, the doctor will take samples of lymph nodes and other tissues in the abdomen to see if any cancer is left. This procedure is not done for dysgerminomas. Observation Observation is closely watching a patients condition without giving any treatment unless signs or symptoms appear or change. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Ovarian, Fallopian Tube, or Primary Peritoneal Cancer for more information. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy is used to treat ovarian germ cell tumors. New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy with bone marrow transplant High-dose chemotherapy with bone marrow transplant is a method of giving very high doses of chemotherapy and replacing blood -forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the bodys blood cells. New treatment options Combination chemotherapy (the use of more than one anticancer drug) is being tested in clinical trials. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options By Stage Stage I Ovarian Germ Cell Tumors Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: - Unilateral salpingo-oophorectomy with or without lymphangiography or CT scan. - Unilateral salpingo-oophorectomy followed by observation. - Unilateral salpingo-oophorectomy followed by radiation therapy. - Unilateral salpingo-oophorectomy followed by chemotherapy. Treatment of other ovarian germ cell tumors may be either: - unilateral salpingo-oophorectomy followed by careful observation; or - unilateral salpingo-oophorectomy, sometimes followed by combination chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I ovarian germ cell tumor. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage II Ovarian Germ Cell Tumors Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be either: - total abdominal hysterectomy and bilateral salpingo-oophorectomy followed by radiation therapy or combination chemotherapy; or - unilateral salpingo-oophorectomy followed by chemotherapy. Treatment of other ovarian germ cell tumors may include the following: - Unilateral salpingo-oophorectomy followed by combination chemotherapy. - Second-look laparotomy (surgery done after primary treatment to see if tumor cells remain). - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage II ovarian germ cell tumor. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage III Ovarian Germ Cell Tumors Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: - Total abdominal hysterectomy and bilateral salpingo-oophorectomy, with removal of as much of the cancer in the pelvis and abdomen as possible. - Unilateral salpingo-oophorectomy followed by chemotherapy. Treatment of other ovarian germ cell tumors may include the following: - Total abdominal hysterectomy and bilateral salpingo-oophorectomy, with removal of as much of the cancer in the pelvis and abdomen as possible. Chemotherapy will be given before and/or after surgery. - Unilateral salpingo-oophorectomy followed by chemotherapy. - Second-look laparotomy (surgery done after primary treatment to see if tumor cells remain). - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage III ovarian germ cell tumor. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IV Ovarian Germ Cell Tumors Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: - Total abdominal hysterectomy and bilateral salpingo-oophorectomy followed by chemotherapy, with removal of as much of the cancer in the pelvis and abdomen as possible. - Unilateral salpingo-oophorectomy followed by chemotherapy. Treatment of other ovarian germ cell tumors may include the following: - Total abdominal hysterectomy and bilateral salpingo-oophorectomy, with removal of as much of the cancer in the pelvis and abdomen as possible. Chemotherapy will be given before and/or after surgery. - Unilateral salpingo-oophorectomy followed by chemotherapy. - Second-look laparotomy (surgery done after primary treatment to see if tumor cells remain). - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage IV ovarian germ cell tumor. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

Germ cell tumors begin in the reproductive cells (egg or sperm) of the body. Ovarian germ cell tumors usually occur in teenage girls or young women and most often affect just one ovary. The ovaries are a pair of organs in the female reproductive system. They are in the pelvis, one on each side of the uterus (the hollow, pear-shaped organ where a fetus grows). Each ovary is about the size and shape of an almond. The ovaries make eggs and female hormones. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Ovarian germ cell tumor is a general name that is used to describe several different types of cancer. The most common ovarian germ cell tumor is called dysgerminoma. See the following PDQ summaries for information about other types of ovarian tumors: Ovarian germ cell tumors can be hard to diagnose (find) early. Often there are no symptoms in the early stages, but tumors may be found during regular gynecologic exams (checkups). Check with your doctor if you have either of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: Ovarian germ cell tumors are usually cured if found and treated early. The process used to find out whether cancer has spread within the ovary or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. Unless a doctor is sure the cancer has spread from the ovaries to other parts of the body, an operation called a laparotomy is done to see if the cancer has spread. The doctor must cut into the abdomen and carefully look at all the organs to see if they have cancer in them. The doctor will cut out small pieces of tissue so they can be checked under a microscope for signs of cancer. The doctor may also wash the abdominal cavity with fluid, which is also checked under a microscope to see if it has cancer cells in it. Usually the doctor will remove the cancer and other organs that have cancer in them during the laparotomy. It is important to know the stage in order to plan treatment. Many of the tests used to diagnose ovarian germ cell tumor are also used for staging. The following tests and procedures may also be used for staging: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of tumor as the primary tumor. For example, if an ovarian germ cell tumor spreads to the liver, the tumor cells in the liver are actually cancerous ovarian germ cells. The disease is metastatic ovarian germ cell tumor, not liver cancer. In stage I, cancer is found in one or both ovaries or fallopian tubes. Stage I is divided into stage IA, stage IB, and stage IC. In stage II, cancer is found in one or both ovaries or fallopian tubes and has spread into other areas of the pelvis, or primary peritoneal cancer is found within the pelvis. Stage II is divided into stage IIA and stage IIB. In stage III, cancer is found in one or both ovaries or fallopian tubes, or is primary peritoneal cancer, and has spread outside the pelvis to other parts of the abdomen and/or to nearby lymph nodes. Stage III is divided into stage IIIA, stage IIIB, and stage IIIC. In stage IV, cancer has spread beyond the abdomen to other parts of the body. Stage IV is divided into stage IVA and stage IVB. The cancer may come back in the other ovary or in other parts of the body. Different types of treatment are available for patients with ovarian germ cell tumor. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of ovarian germ cell tumor. A doctor may take out the cancer using one of the following types of surgery. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. After chemotherapy for an ovarian germ cell tumor, a second-look laparotomy may be done. This is similar to the laparotomy that is done to find out the stage of the cancer. Second-look laparotomy is a surgical procedure to find out if tumor cells are left after primary treatment. During this procedure, the doctor will take samples of lymph nodes and other tissues in the abdomen to see if any cancer is left. This procedure is not done for dysgerminomas. Observation is closely watching a patient’s condition without giving any treatment unless signs or symptoms appear or change. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Ovarian, Fallopian Tube, or Primary Peritoneal Cancer for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy with bone marrow transplant is a method of giving very high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells. Combination chemotherapy (the use of more than one anticancer drug) is being tested in clinical trials. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may be either: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be either: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may include the following: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment depends on whether the tumor is a dysgerminoma or another type of ovarian germ cell tumor. Treatment of dysgerminoma may be: Treatment of other ovarian germ cell tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about ovarian germ cell tumors, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of ovarian germ cell tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Ovarian Germ Cell Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/ovarian/patient/ovarian-germ-cell-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389363] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. 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In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Ovarian Germ Cell Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Ovarian Germ Cell Tumors ?

New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy with bone marrow transplant High-dose chemotherapy with bone marrow transplant is a method of giving very high doses of chemotherapy and replacing blood -forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the bodys blood cells. New treatment options Combination chemotherapy (the use of more than one anticancer drug) is being tested in clinical trials. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Chronic myelogenous leukemia (also called CML or chronic granulocytic leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age, and rarely occurs in children. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In CML, too many blood stem cells become a type of white blood cell called granulocytes. These granulocytes are abnormal and do not become healthy white blood cells. They are also called leukemia cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. This summary is about chronic myelogenous leukemia. See the following PDQ summaries for more information about leukemia: These and other signs and symptoms may be caused by CML or by other conditions. Check with your doctor if you have any of the following: Sometimes CML does not cause any symptoms at all. Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the “Philadelphia chromosome.” It results in the bone marrow making a protein, called tyrosine kinase, that causes too many stem cells to become white blood cells (granulocytes or blasts). The Philadelphia chromosome is not passed from parent to child. EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. The following tests and procedures may be used: One of the following tests may be done on the samples of blood or bone marrow tissue that are removed: The prognosis and treatment options depend on the following: The extent or spread of cancer is usually described as stages. In chronic myelogenous leukemia (CML), the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The information from tests and procedures done to diagnose chronic myelogenous leukemia is also used to plan treatment. As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of signs or symptoms determine the phase of the disease. In chronic phase CML, fewer than 10% of the cells in the blood and bone marrow are blast cells. In accelerated phase CML, 10% to 19% of the cells in the blood and bone marrow are blast cells. In blastic phase CML, 20% or more of the cells in the blood or bone marrow are blast cells. When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis. In relapsed CML, the number of blast cells increases after a remission. Different types of treatment are available for patients with chronic myelogenous leukemia (CML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information about new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Chronic Myelogenous Leukemia for more information. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Chronic Myelogenous Leukemia for more information. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Donor lymphocyte infusion (DLI) is a cancer treatment that may be used after stem cell transplant. Lymphocytes (a type of white blood cell) from the stem cell transplant donor are removed from the donor’s blood and may be frozen for storage. The donor’s lymphocytes are thawed if they were frozen and then given to the patient through one or more infusions. The lymphocytes see the patient’s cancer cells as not belonging to the body and attack them. Splenectomy is surgery to remove the spleen. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of accelerated phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of blastic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. In relapsed CML, the number of blast cells increases after a remission. Treatment of relapsed chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myelogenous leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myelogenous leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myelogenous Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/cml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389183] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myelogenous Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Chronic Myelogenous Leukemia ?

Key Points - Chronic myelogenous leukemia is a disease in which the bone marrow makes too many white blood cells. - Leukemia may affect red blood cells, white blood cells, and platelets. - Signs and symptoms of chronic myelogenous leukemia include fever, night sweats, and tiredness. - Most people with CML have a gene mutation (change) called the Philadelphia chromosome. - Tests that examine the blood and bone marrow are used to detect (find) and diagnose chronic myelogenous leukemia. - Certain factors affect prognosis (chance of recovery) and treatment options. Chronic myelogenous leukemia is a disease in which the bone marrow makes too many white blood cells. Chronic myelogenous leukemia (also called CML or chronic granulocytic leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age, and rarely occurs in children. Leukemia may affect red blood cells, white blood cells, and platelets. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: - Red blood cells that carry oxygen and other substances to all tissues of the body. - Platelets that form blood clots to stop bleeding. - Granulocytes (white blood cells) that fight infection and disease. In CML, too many blood stem cells become a type of white blood cell called granulocytes. These granulocytes are abnormal and do not become healthy white blood cells. They are also called leukemia cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. This summary is about chronic myelogenous leukemia. See the following PDQ summaries for more information about leukemia: - Adult Acute Lymphoblastic Leukemia Treatment - Childhood Acute Lymphoblastic Leukemia Treatment - Adult Acute Myeloid Leukemia Treatment - Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment - Chronic Lymphocytic Leukemia Treatment - Hairy Cell Leukemia Treatment

Chronic myelogenous leukemia (also called CML or chronic granulocytic leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age, and rarely occurs in children. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In CML, too many blood stem cells become a type of white blood cell called granulocytes. These granulocytes are abnormal and do not become healthy white blood cells. They are also called leukemia cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. This summary is about chronic myelogenous leukemia. See the following PDQ summaries for more information about leukemia: These and other signs and symptoms may be caused by CML or by other conditions. Check with your doctor if you have any of the following: Sometimes CML does not cause any symptoms at all. Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the “Philadelphia chromosome.” It results in the bone marrow making a protein, called tyrosine kinase, that causes too many stem cells to become white blood cells (granulocytes or blasts). The Philadelphia chromosome is not passed from parent to child. EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. The following tests and procedures may be used: One of the following tests may be done on the samples of blood or bone marrow tissue that are removed: The prognosis and treatment options depend on the following: The extent or spread of cancer is usually described as stages. In chronic myelogenous leukemia (CML), the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The information from tests and procedures done to diagnose chronic myelogenous leukemia is also used to plan treatment. As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of signs or symptoms determine the phase of the disease. In chronic phase CML, fewer than 10% of the cells in the blood and bone marrow are blast cells. In accelerated phase CML, 10% to 19% of the cells in the blood and bone marrow are blast cells. In blastic phase CML, 20% or more of the cells in the blood or bone marrow are blast cells. When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis. In relapsed CML, the number of blast cells increases after a remission. Different types of treatment are available for patients with chronic myelogenous leukemia (CML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information about new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Chronic Myelogenous Leukemia for more information. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Chronic Myelogenous Leukemia for more information. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Donor lymphocyte infusion (DLI) is a cancer treatment that may be used after stem cell transplant. Lymphocytes (a type of white blood cell) from the stem cell transplant donor are removed from the donor’s blood and may be frozen for storage. The donor’s lymphocytes are thawed if they were frozen and then given to the patient through one or more infusions. The lymphocytes see the patient’s cancer cells as not belonging to the body and attack them. Splenectomy is surgery to remove the spleen. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of accelerated phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of blastic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. In relapsed CML, the number of blast cells increases after a remission. Treatment of relapsed chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myelogenous leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myelogenous leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myelogenous Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/cml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389183] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myelogenous Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Chronic Myelogenous Leukemia ?

Signs and symptoms of chronic myelogenous leukemia include fever, night sweats, and tiredness. These and other signs and symptoms may be caused by CML or by other conditions. Check with your doctor if you have any of the following: - Feeling very tired. - Weight loss for no known reason. - Night sweats. - Fever. - Pain or a feeling of fullness below the ribs on the left side. Sometimes CML does not cause any symptoms at all.

Chronic myelogenous leukemia (also called CML or chronic granulocytic leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age, and rarely occurs in children. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In CML, too many blood stem cells become a type of white blood cell called granulocytes. These granulocytes are abnormal and do not become healthy white blood cells. They are also called leukemia cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. This summary is about chronic myelogenous leukemia. See the following PDQ summaries for more information about leukemia: These and other signs and symptoms may be caused by CML or by other conditions. Check with your doctor if you have any of the following: Sometimes CML does not cause any symptoms at all. Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the “Philadelphia chromosome.” It results in the bone marrow making a protein, called tyrosine kinase, that causes too many stem cells to become white blood cells (granulocytes or blasts). The Philadelphia chromosome is not passed from parent to child. EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. The following tests and procedures may be used: One of the following tests may be done on the samples of blood or bone marrow tissue that are removed: The prognosis and treatment options depend on the following: The extent or spread of cancer is usually described as stages. In chronic myelogenous leukemia (CML), the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The information from tests and procedures done to diagnose chronic myelogenous leukemia is also used to plan treatment. As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of signs or symptoms determine the phase of the disease. In chronic phase CML, fewer than 10% of the cells in the blood and bone marrow are blast cells. In accelerated phase CML, 10% to 19% of the cells in the blood and bone marrow are blast cells. In blastic phase CML, 20% or more of the cells in the blood or bone marrow are blast cells. When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis. In relapsed CML, the number of blast cells increases after a remission. Different types of treatment are available for patients with chronic myelogenous leukemia (CML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information about new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Chronic Myelogenous Leukemia for more information. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Chronic Myelogenous Leukemia for more information. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Donor lymphocyte infusion (DLI) is a cancer treatment that may be used after stem cell transplant. Lymphocytes (a type of white blood cell) from the stem cell transplant donor are removed from the donor’s blood and may be frozen for storage. The donor’s lymphocytes are thawed if they were frozen and then given to the patient through one or more infusions. The lymphocytes see the patient’s cancer cells as not belonging to the body and attack them. Splenectomy is surgery to remove the spleen. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of accelerated phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of blastic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. In relapsed CML, the number of blast cells increases after a remission. Treatment of relapsed chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myelogenous leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myelogenous leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myelogenous Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/cml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389183] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myelogenous Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the genetic changes related to Chronic Myelogenous Leukemia ?

Most people with CML have a gene mutation (change) called the Philadelphia chromosome. Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the Philadelphia chromosome. It results in the bone marrow making an enzyme, called tyrosine kinase, that causes too many stem cells to become white blood cells (granulocytes or blasts). The Philadelphia chromosome is not passed from parent to child.

Chronic myelogenous leukemia (also called CML or chronic granulocytic leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age, and rarely occurs in children. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In CML, too many blood stem cells become a type of white blood cell called granulocytes. These granulocytes are abnormal and do not become healthy white blood cells. They are also called leukemia cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. This summary is about chronic myelogenous leukemia. See the following PDQ summaries for more information about leukemia: These and other signs and symptoms may be caused by CML or by other conditions. Check with your doctor if you have any of the following: Sometimes CML does not cause any symptoms at all. Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the “Philadelphia chromosome.” It results in the bone marrow making a protein, called tyrosine kinase, that causes too many stem cells to become white blood cells (granulocytes or blasts). The Philadelphia chromosome is not passed from parent to child. EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. The following tests and procedures may be used: One of the following tests may be done on the samples of blood or bone marrow tissue that are removed: The prognosis and treatment options depend on the following: The extent or spread of cancer is usually described as stages. In chronic myelogenous leukemia (CML), the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The information from tests and procedures done to diagnose chronic myelogenous leukemia is also used to plan treatment. As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of signs or symptoms determine the phase of the disease. In chronic phase CML, fewer than 10% of the cells in the blood and bone marrow are blast cells. In accelerated phase CML, 10% to 19% of the cells in the blood and bone marrow are blast cells. In blastic phase CML, 20% or more of the cells in the blood or bone marrow are blast cells. When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis. In relapsed CML, the number of blast cells increases after a remission. Different types of treatment are available for patients with chronic myelogenous leukemia (CML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information about new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Chronic Myelogenous Leukemia for more information. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Chronic Myelogenous Leukemia for more information. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Donor lymphocyte infusion (DLI) is a cancer treatment that may be used after stem cell transplant. Lymphocytes (a type of white blood cell) from the stem cell transplant donor are removed from the donor’s blood and may be frozen for storage. The donor’s lymphocytes are thawed if they were frozen and then given to the patient through one or more infusions. The lymphocytes see the patient’s cancer cells as not belonging to the body and attack them. Splenectomy is surgery to remove the spleen. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of accelerated phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of blastic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. In relapsed CML, the number of blast cells increases after a remission. Treatment of relapsed chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myelogenous leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myelogenous leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myelogenous Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/cml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389183] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myelogenous Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Chronic Myelogenous Leukemia ?

Tests that examine the blood and bone marrow are used to detect (find) and diagnose chronic myelogenous leukemia.. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease such as an enlarged spleen. A history of the patients health habits and past illnesses and treatments will also be taken. - Complete blood count (CBC) with differential : A procedure in which a sample of blood is drawn and checked for the following: - The number of red blood cells and platelets. - The number and type of white blood cells. - The amount of hemoglobin (the protein that carries oxygen) in the red blood cells. - The portion of the blood sample made up of red blood cells. - Blood chemistry studies : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease. - Bone marrow aspiration and biopsy : The removal of bone marrow, blood, and a small piece of bone by inserting a needle into the hipbone or breastbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for abnormal cells. One of the following tests may be done on the samples of blood or bone marrow tissue that are removed: - Cytogenetic analysis: A test in which cells in a sample of blood or bone marrow are viewed under a microscope to look for certain changes in the chromosomes, such as the Philadelphia chromosome. - FISH (fluorescence in situ hybridization): A laboratory technique used to look at genes or chromosomes in cells and tissues. Pieces of DNA that contain a fluorescent dye are made in the laboratory and added to cells or tissues on a glass slide. When these pieces of DNA bind to specific genes or areas of chromosomes on the slide, they light up when viewed under a microscope with a special light. - Reverse transcriptionpolymerase chain reaction (RTPCR): A laboratory test in which cells in a sample of tissue are studied using chemicals to look for certain changes in the structure or function of genes.

Chronic myelogenous leukemia (also called CML or chronic granulocytic leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age, and rarely occurs in children. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In CML, too many blood stem cells become a type of white blood cell called granulocytes. These granulocytes are abnormal and do not become healthy white blood cells. They are also called leukemia cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. This summary is about chronic myelogenous leukemia. See the following PDQ summaries for more information about leukemia: These and other signs and symptoms may be caused by CML or by other conditions. Check with your doctor if you have any of the following: Sometimes CML does not cause any symptoms at all. Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the “Philadelphia chromosome.” It results in the bone marrow making a protein, called tyrosine kinase, that causes too many stem cells to become white blood cells (granulocytes or blasts). The Philadelphia chromosome is not passed from parent to child. EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. The following tests and procedures may be used: One of the following tests may be done on the samples of blood or bone marrow tissue that are removed: The prognosis and treatment options depend on the following: The extent or spread of cancer is usually described as stages. In chronic myelogenous leukemia (CML), the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The information from tests and procedures done to diagnose chronic myelogenous leukemia is also used to plan treatment. As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of signs or symptoms determine the phase of the disease. In chronic phase CML, fewer than 10% of the cells in the blood and bone marrow are blast cells. In accelerated phase CML, 10% to 19% of the cells in the blood and bone marrow are blast cells. In blastic phase CML, 20% or more of the cells in the blood or bone marrow are blast cells. When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis. In relapsed CML, the number of blast cells increases after a remission. Different types of treatment are available for patients with chronic myelogenous leukemia (CML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information about new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Chronic Myelogenous Leukemia for more information. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Chronic Myelogenous Leukemia for more information. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Donor lymphocyte infusion (DLI) is a cancer treatment that may be used after stem cell transplant. Lymphocytes (a type of white blood cell) from the stem cell transplant donor are removed from the donor’s blood and may be frozen for storage. The donor’s lymphocytes are thawed if they were frozen and then given to the patient through one or more infusions. The lymphocytes see the patient’s cancer cells as not belonging to the body and attack them. Splenectomy is surgery to remove the spleen. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of accelerated phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of blastic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. In relapsed CML, the number of blast cells increases after a remission. Treatment of relapsed chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myelogenous leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myelogenous leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myelogenous Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/cml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389183] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myelogenous Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Chronic Myelogenous Leukemia ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options depend on the following: - The patients age. - The phase of CML. - The amount of blasts in the blood or bone marrow. - The size of the spleen at diagnosis. - The patients general health.

Chronic myelogenous leukemia (also called CML or chronic granulocytic leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age, and rarely occurs in children. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In CML, too many blood stem cells become a type of white blood cell called granulocytes. These granulocytes are abnormal and do not become healthy white blood cells. They are also called leukemia cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. This summary is about chronic myelogenous leukemia. See the following PDQ summaries for more information about leukemia: These and other signs and symptoms may be caused by CML or by other conditions. Check with your doctor if you have any of the following: Sometimes CML does not cause any symptoms at all. Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the “Philadelphia chromosome.” It results in the bone marrow making a protein, called tyrosine kinase, that causes too many stem cells to become white blood cells (granulocytes or blasts). The Philadelphia chromosome is not passed from parent to child. EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. The following tests and procedures may be used: One of the following tests may be done on the samples of blood or bone marrow tissue that are removed: The prognosis and treatment options depend on the following: The extent or spread of cancer is usually described as stages. In chronic myelogenous leukemia (CML), the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The information from tests and procedures done to diagnose chronic myelogenous leukemia is also used to plan treatment. As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of signs or symptoms determine the phase of the disease. In chronic phase CML, fewer than 10% of the cells in the blood and bone marrow are blast cells. In accelerated phase CML, 10% to 19% of the cells in the blood and bone marrow are blast cells. In blastic phase CML, 20% or more of the cells in the blood or bone marrow are blast cells. When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis. In relapsed CML, the number of blast cells increases after a remission. Different types of treatment are available for patients with chronic myelogenous leukemia (CML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information about new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Chronic Myelogenous Leukemia for more information. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Chronic Myelogenous Leukemia for more information. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Donor lymphocyte infusion (DLI) is a cancer treatment that may be used after stem cell transplant. Lymphocytes (a type of white blood cell) from the stem cell transplant donor are removed from the donor’s blood and may be frozen for storage. The donor’s lymphocytes are thawed if they were frozen and then given to the patient through one or more infusions. The lymphocytes see the patient’s cancer cells as not belonging to the body and attack them. Splenectomy is surgery to remove the spleen. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of accelerated phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of blastic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. In relapsed CML, the number of blast cells increases after a remission. Treatment of relapsed chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myelogenous leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myelogenous leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myelogenous Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/cml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389183] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myelogenous Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Chronic Myelogenous Leukemia ?

Key Points - After chronic myelogenous leukemia has been diagnosed, tests are done to find out if the cancer has spread. - Chronic myelogenous leukemia has 3 phases. - Chronic phase - Accelerated phase - Blastic phase After chronic myelogenous leukemia has been diagnosed, tests are done to find out if the cancer has spread. Staging is the process used to find out how far the cancer has spread. There is no standard staging system for chronic myelogenous leukemia (CML). Instead, the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The information from tests and procedures done to detect (find) and diagnose chronic myelogenous leukemia is also used to plan treatment. Chronic myelogenous leukemia has 3 phases. As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of signs or symptoms determine the phase of the disease. Chronic phase In chronic phase CML, fewer than 10% of the cells in the blood and bone marrow are blast cells. Accelerated phase In accelerated phase CML, 10% to 19% of the cells in the blood and bone marrow are blast cells. Blastic phase In blastic phase CML, 20% or more of the cells in the blood or bone marrow are blast cells. When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis.

Chronic myelogenous leukemia (also called CML or chronic granulocytic leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age, and rarely occurs in children. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In CML, too many blood stem cells become a type of white blood cell called granulocytes. These granulocytes are abnormal and do not become healthy white blood cells. They are also called leukemia cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. This summary is about chronic myelogenous leukemia. See the following PDQ summaries for more information about leukemia: These and other signs and symptoms may be caused by CML or by other conditions. Check with your doctor if you have any of the following: Sometimes CML does not cause any symptoms at all. Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the “Philadelphia chromosome.” It results in the bone marrow making a protein, called tyrosine kinase, that causes too many stem cells to become white blood cells (granulocytes or blasts). The Philadelphia chromosome is not passed from parent to child. EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. The following tests and procedures may be used: One of the following tests may be done on the samples of blood or bone marrow tissue that are removed: The prognosis and treatment options depend on the following: The extent or spread of cancer is usually described as stages. In chronic myelogenous leukemia (CML), the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The information from tests and procedures done to diagnose chronic myelogenous leukemia is also used to plan treatment. As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of signs or symptoms determine the phase of the disease. In chronic phase CML, fewer than 10% of the cells in the blood and bone marrow are blast cells. In accelerated phase CML, 10% to 19% of the cells in the blood and bone marrow are blast cells. In blastic phase CML, 20% or more of the cells in the blood or bone marrow are blast cells. When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis. In relapsed CML, the number of blast cells increases after a remission. Different types of treatment are available for patients with chronic myelogenous leukemia (CML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information about new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Chronic Myelogenous Leukemia for more information. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Chronic Myelogenous Leukemia for more information. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Donor lymphocyte infusion (DLI) is a cancer treatment that may be used after stem cell transplant. Lymphocytes (a type of white blood cell) from the stem cell transplant donor are removed from the donor’s blood and may be frozen for storage. The donor’s lymphocytes are thawed if they were frozen and then given to the patient through one or more infusions. The lymphocytes see the patient’s cancer cells as not belonging to the body and attack them. Splenectomy is surgery to remove the spleen. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of accelerated phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of blastic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. In relapsed CML, the number of blast cells increases after a remission. Treatment of relapsed chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myelogenous leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myelogenous leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myelogenous Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/cml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389183] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myelogenous Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Chronic Myelogenous Leukemia ?

New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Chronic myelogenous leukemia (also called CML or chronic granulocytic leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age, and rarely occurs in children. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In CML, too many blood stem cells become a type of white blood cell called granulocytes. These granulocytes are abnormal and do not become healthy white blood cells. They are also called leukemia cells. The leukemia cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. This summary is about chronic myelogenous leukemia. See the following PDQ summaries for more information about leukemia: These and other signs and symptoms may be caused by CML or by other conditions. Check with your doctor if you have any of the following: Sometimes CML does not cause any symptoms at all. Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the “Philadelphia chromosome.” It results in the bone marrow making a protein, called tyrosine kinase, that causes too many stem cells to become white blood cells (granulocytes or blasts). The Philadelphia chromosome is not passed from parent to child. EnlargePhiladelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome. The following tests and procedures may be used: One of the following tests may be done on the samples of blood or bone marrow tissue that are removed: The prognosis and treatment options depend on the following: The extent or spread of cancer is usually described as stages. In chronic myelogenous leukemia (CML), the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The information from tests and procedures done to diagnose chronic myelogenous leukemia is also used to plan treatment. As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of signs or symptoms determine the phase of the disease. In chronic phase CML, fewer than 10% of the cells in the blood and bone marrow are blast cells. In accelerated phase CML, 10% to 19% of the cells in the blood and bone marrow are blast cells. In blastic phase CML, 20% or more of the cells in the blood or bone marrow are blast cells. When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis. In relapsed CML, the number of blast cells increases after a remission. Different types of treatment are available for patients with chronic myelogenous leukemia (CML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information about new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Chronic Myelogenous Leukemia for more information. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Chronic Myelogenous Leukemia for more information. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Donor lymphocyte infusion (DLI) is a cancer treatment that may be used after stem cell transplant. Lymphocytes (a type of white blood cell) from the stem cell transplant donor are removed from the donor’s blood and may be frozen for storage. The donor’s lymphocytes are thawed if they were frozen and then given to the patient through one or more infusions. The lymphocytes see the patient’s cancer cells as not belonging to the body and attack them. Splenectomy is surgery to remove the spleen. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of accelerated phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of blastic phase chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. In relapsed CML, the number of blast cells increases after a remission. Treatment of relapsed chronic myelogenous leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myelogenous leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myelogenous leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myelogenous Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/cml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389183] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myelogenous Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Chronic Myelogenous Leukemia ?

Key Points - There are different types of treatment for patients with chronic myelogenous leukemia. - Six types of standard treatment are used: - Targeted therapy - Chemotherapy - Biologic therapy - High-dose chemotherapy with stem cell transplant - Donor lymphocyte infusion (DLI) - Surgery - New types of treatment are being tested in clinical trials. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with chronic myelogenous leukemia. Different types of treatment are available for patients with chronic myelogenous leukemia (CML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information about new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Six types of standard treatment are used: Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Tyrosine kinase inhibitors are targeted therapy drugs used to treat chronic myelogenous leukemia. Imatinib mesylate, nilotinib, dasatinib, and ponatinib are tyrosine kinase inhibitors that are used to treat CML. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Biologic therapy Biologic therapy is a treatment that uses the patients immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the bodys natural defenses against cancer. This type of cancer treatment is also called biotherapy or immunotherapy. See Drugs Approved for Chronic Myelogenous Leukemia for more information. High-dose chemotherapy with stem cell transplant High-dose chemotherapy with stem cell transplant is a method of giving high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the bodys blood cells. See Drugs Approved for Chronic Myelogenous Leukemia for more information. Donor lymphocyte infusion (DLI) Donor lymphocyte infusion (DLI) is a cancer treatment that may be used after stem cell transplant. Lymphocytes (a type of white blood cell) from the stem cell transplant donor are removed from the donors blood and may be frozen for storage. The donors lymphocytes are thawed if they were frozen and then given to the patient through one or more infusions. The lymphocytes see the patients cancer cells as not belonging to the body and attack them. Surgery Splenectomy is surgery to remove the spleen. New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Chronic Myelogenous Leukemia Chronic Phase Chronic Myelogenous Leukemia Treatment of chronic phase chronic myelogenous leukemia may include the following: - Targeted therapy with a tyrosine kinase inhibitor. - High-dose chemotherapy with donor stem cell transplant. - Chemotherapy. - Splenectomy. - A clinical trial of lower-dose chemotherapy with donor stem cell transplant. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with chronic phase chronic myelogenous leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Accelerated Phase Chronic Myelogenous Leukemia Treatment of accelerated phase chronic myelogenous leukemia may include the following: - Donor stem cell transplant. - Targeted therapy with a tyrosine kinase inhibitor. - Tyrosine kinase inhibitor therapy followed by a donor stem cell transplant. - Biologic therapy (interferon) with or without chemotherapy. - High-dose chemotherapy. - Chemotherapy. - Transfusion therapy to replace red blood cells, platelets, and sometimes white blood cells, to relieve symptoms and improve quality of life. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with accelerated phase chronic myelogenous leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Blastic Phase Chronic Myelogenous Leukemia Treatment of blastic phase chronic myelogenous leukemia may include the following: - Targeted therapy with a tyrosine kinase inhibitor. - Chemotherapy using one or more drugs. - High-dose chemotherapy. - Donor stem cell transplant. - Chemotherapy as palliative therapy to relieve symptoms and improve quality of life. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with blastic phase chronic myelogenous leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Relapsed Chronic Myelogenous Leukemia Treatment of relapsed chronic myelogenous leukemia may include the following: - Targeted therapy with a tyrosine kinase inhibitor. - Donor stem cell transplant. - Chemotherapy. - Donor lymphocyte infusion. - Biologic therapy (interferon). - A clinical trial of new types or higher doses of targeted therapy or donor stem cell transplant. Check the list of NCI-supported cancer clinical trials that are now accepting patients with relapsing chronic myelogenous leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood stem cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A myeloid stem cell becomes a red blood cell, white blood cell, or platelet. A lymphoid stem cell becomes a lymphoblast and then one of three types of lymphocytes (white blood cells): In mycosis fungoides, T-cell lymphocytes become cancerous and affect the skin. When these lymphocytes occur in the blood, they are called Sézary cells. In Sézary syndrome, cancerous T-cell lymphocytes affect the skin and large numbers of Sézary cells are found in the blood. Mycosis fungoides and Sézary syndrome are the two most common types of cutaneous T-cell lymphoma (a type of non-Hodgkin lymphoma). For information about other types of skin cancer or non-Hodgkin lymphoma, see the following PDQ summaries: Mycosis fungoides may go through the following phases: Check with your doctor if you have any of these signs. Also, skin all over the body is reddened, itchy, peeling, and painful. There may also be patches, plaques, or tumors on the skin. It is not known if Sézary syndrome is an advanced form of mycosis fungoides or a separate disease. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Mycosis fungoides and Sézary syndrome are hard to cure. Treatment is usually palliative, to relieve symptoms and improve the quality of life. Patients with early stage disease may live many years. The process used to find out if cancer has spread from the skin to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if mycosis fungoides spreads to the liver, the cancer cells in the liver are actually mycosis fungoides cells. The disease is metastatic mycosis fungoides, not liver cancer. Stage I is divided into stages IA and IB as follows: There may be a low number of Sézary cells in the blood. Stage II is divided into stages IIA and IIB as follows: There may be a low number of Sézary cells in the blood. In stage III, 80% or more of the skin surface is reddened and may have patches, papules, plaques, or tumors. Lymph nodes may be abnormal, but they are not cancerous. There may be a low number of Sézary cells in the blood. When there is a high number of Sézary cells in the blood, the disease is called Sézary syndrome. Stage IV is divided into stages IVA1, IVA2, and IVB as follows: Mycosis fungoides and Sézary syndrome may come back in the skin or in other parts of the body, such as the spleen or liver. Different types of treatment are available for patients with mycosis fungoides and Sézary syndrome. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients undergoing photodynamic therapy will need to limit the amount of time spent in sunlight. There are different types of photodynamic therapy: Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Sometimes, total skin electron beam (TSEB) radiation therapy is used to treat mycosis fungoides and Sézary syndrome. This is a type of external radiation treatment in which a radiation therapy machine aims electrons (tiny, invisible particles) at the skin covering the whole body. External radiation therapy may also be used as palliative therapy to relieve symptoms and improve quality of life. Ultraviolet A (UVA) radiation therapy or ultraviolet B (UVB) radiation therapy may be given using a special lamp or laser that directs radiation at the skin. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Sometimes the chemotherapy is topical (put on the skin in a cream, lotion, or ointment). See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Topical corticosteroids are used to relieve red, swollen, and inflamed skin. They are a type of steroid. Topical corticosteroids may be in a cream, lotion, or ointment. Retinoids, such as bexarotene, are drugs related to vitamin A that can slow the growth of certain types of cancer cells. The retinoids may be taken by mouth or put on the skin. Lenalidomide is a drug that helps the immune system kill abnormal blood cells or cancer cells and may prevent the growth of new blood vessels that tumors need to grow. Vorinostat and romidepsin are two of the histone deacetylase (HDAC) inhibitors used to treat mycosis fungoides and Sézary syndrome. HDAC inhibitors cause a chemical change that stops tumor cells from dividing. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of monoclonal antibodies include: High doses of chemotherapy and sometimes radiation therapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and radiation therapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage I and stage II mycosis fungoides may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage III and stage IV mycosis fungoides including Sézary syndrome is palliative (to relieve symptoms and improve the quality of life) and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent mycosis fungoides including Sézary syndrome may be within a clinical trial and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about mycosis fungoides and Sézary syndrome, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of mycosis fungoides (including Sézary Syndrome). It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Mycosis Fungoides (Including Sézary Syndrome) Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/mycosis-fungoides-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389317] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Mycosis Fungoides (Including Sézary Syndrome) Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Mycosis Fungoides and the Szary Syndrome ?

Key Points - Mycosis fungoides and the Szary syndrome are diseases in which lymphocytes (a type of white blood cell) become malignant (cancerous) and affect the skin. - Mycosis fungoides and the Szary syndrome are types of cutaneous T-cell lymphoma. - A sign of mycosis fungoides is a red rash on the skin. - In the Szary syndrome, cancerous T-cells are found in the blood. - Tests that examine the skin and blood are used to detect (find) and diagnose mycosis fungoides and the Szary syndrome. - Certain factors affect prognosis (chance of recovery) and treatment options. Mycosis fungoides and the Szary syndrome are diseases in which lymphocytes (a type of white blood cell) become malignant (cancerous) and affect the skin. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood stem cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A myeloid stem cell becomes a red blood cell, white blood cell, or platelet. A lymphoid stem cell becomes a lymphoblast and then one of three types of lymphocytes (white blood cells): - B-cell lymphocytes that make antibodies to help fight infection. - T-cell lymphocytes that help B-lymphocytes make the antibodies that help fight infection. - Natural killer cells that attack cancer cells and viruses. In mycosis fungoides, T-cell lymphocytes become cancerous and affect the skin. In the Szary syndrome, cancerous T-cell lymphocytes affect the skin and are in the blood. Mycosis fungoides and the Szary syndrome are types of cutaneous T-cell lymphoma. Mycosis fungoides and the Szary syndrome are the two most common types of cutaneous T-cell lymphoma (a type of non-Hodgkin lymphoma). For information about other types of skin cancer or non-Hodgkin lymphoma, see the following PDQ summaries: - Adult Non-Hodgkin Lymphoma Treatment - Skin Cancer Treatment - Melanoma Treatment - Kaposi Sarcoma Treatment In the Szary syndrome, cancerous T-cells are found in the blood. Also, skin all over the body is reddened, itchy, peeling, and painful. There may also be patches, plaques, or tumors on the skin. It is not known if the Szary syndrome is an advanced form of mycosis fungoides or a separate disease.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood stem cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A myeloid stem cell becomes a red blood cell, white blood cell, or platelet. A lymphoid stem cell becomes a lymphoblast and then one of three types of lymphocytes (white blood cells): In mycosis fungoides, T-cell lymphocytes become cancerous and affect the skin. When these lymphocytes occur in the blood, they are called Sézary cells. In Sézary syndrome, cancerous T-cell lymphocytes affect the skin and large numbers of Sézary cells are found in the blood. Mycosis fungoides and Sézary syndrome are the two most common types of cutaneous T-cell lymphoma (a type of non-Hodgkin lymphoma). For information about other types of skin cancer or non-Hodgkin lymphoma, see the following PDQ summaries: Mycosis fungoides may go through the following phases: Check with your doctor if you have any of these signs. Also, skin all over the body is reddened, itchy, peeling, and painful. There may also be patches, plaques, or tumors on the skin. It is not known if Sézary syndrome is an advanced form of mycosis fungoides or a separate disease. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Mycosis fungoides and Sézary syndrome are hard to cure. Treatment is usually palliative, to relieve symptoms and improve the quality of life. Patients with early stage disease may live many years. The process used to find out if cancer has spread from the skin to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if mycosis fungoides spreads to the liver, the cancer cells in the liver are actually mycosis fungoides cells. The disease is metastatic mycosis fungoides, not liver cancer. Stage I is divided into stages IA and IB as follows: There may be a low number of Sézary cells in the blood. Stage II is divided into stages IIA and IIB as follows: There may be a low number of Sézary cells in the blood. In stage III, 80% or more of the skin surface is reddened and may have patches, papules, plaques, or tumors. Lymph nodes may be abnormal, but they are not cancerous. There may be a low number of Sézary cells in the blood. When there is a high number of Sézary cells in the blood, the disease is called Sézary syndrome. Stage IV is divided into stages IVA1, IVA2, and IVB as follows: Mycosis fungoides and Sézary syndrome may come back in the skin or in other parts of the body, such as the spleen or liver. Different types of treatment are available for patients with mycosis fungoides and Sézary syndrome. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients undergoing photodynamic therapy will need to limit the amount of time spent in sunlight. There are different types of photodynamic therapy: Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Sometimes, total skin electron beam (TSEB) radiation therapy is used to treat mycosis fungoides and Sézary syndrome. This is a type of external radiation treatment in which a radiation therapy machine aims electrons (tiny, invisible particles) at the skin covering the whole body. External radiation therapy may also be used as palliative therapy to relieve symptoms and improve quality of life. Ultraviolet A (UVA) radiation therapy or ultraviolet B (UVB) radiation therapy may be given using a special lamp or laser that directs radiation at the skin. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Sometimes the chemotherapy is topical (put on the skin in a cream, lotion, or ointment). See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Topical corticosteroids are used to relieve red, swollen, and inflamed skin. They are a type of steroid. Topical corticosteroids may be in a cream, lotion, or ointment. Retinoids, such as bexarotene, are drugs related to vitamin A that can slow the growth of certain types of cancer cells. The retinoids may be taken by mouth or put on the skin. Lenalidomide is a drug that helps the immune system kill abnormal blood cells or cancer cells and may prevent the growth of new blood vessels that tumors need to grow. Vorinostat and romidepsin are two of the histone deacetylase (HDAC) inhibitors used to treat mycosis fungoides and Sézary syndrome. HDAC inhibitors cause a chemical change that stops tumor cells from dividing. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of monoclonal antibodies include: High doses of chemotherapy and sometimes radiation therapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and radiation therapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage I and stage II mycosis fungoides may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage III and stage IV mycosis fungoides including Sézary syndrome is palliative (to relieve symptoms and improve the quality of life) and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent mycosis fungoides including Sézary syndrome may be within a clinical trial and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about mycosis fungoides and Sézary syndrome, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of mycosis fungoides (including Sézary Syndrome). It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Mycosis Fungoides (Including Sézary Syndrome) Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/mycosis-fungoides-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389317] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Mycosis Fungoides (Including Sézary Syndrome) Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Mycosis Fungoides and the Szary Syndrome ?

A sign of mycosis fungoides is a red rash on the skin. Mycosis fungoides may go through the following phases: - Premycotic phase: A scaly, red rash in areas of the body that usually are not exposed to the sun. This rash does not cause symptoms and may last for months or years. It is hard to diagnose the rash as mycosis fungoides during this phase. - Patch phase: Thin, reddened, eczema -like rash. - Plaque phase: Small raised bumps (papules) or hardened lesions on the skin, which may be reddened. - Tumor phase: Tumors form on the skin. These tumors may develop ulcers and the skin may get infected. Check with your doctor if you have any of these signs.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood stem cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A myeloid stem cell becomes a red blood cell, white blood cell, or platelet. A lymphoid stem cell becomes a lymphoblast and then one of three types of lymphocytes (white blood cells): In mycosis fungoides, T-cell lymphocytes become cancerous and affect the skin. When these lymphocytes occur in the blood, they are called Sézary cells. In Sézary syndrome, cancerous T-cell lymphocytes affect the skin and large numbers of Sézary cells are found in the blood. Mycosis fungoides and Sézary syndrome are the two most common types of cutaneous T-cell lymphoma (a type of non-Hodgkin lymphoma). For information about other types of skin cancer or non-Hodgkin lymphoma, see the following PDQ summaries: Mycosis fungoides may go through the following phases: Check with your doctor if you have any of these signs. Also, skin all over the body is reddened, itchy, peeling, and painful. There may also be patches, plaques, or tumors on the skin. It is not known if Sézary syndrome is an advanced form of mycosis fungoides or a separate disease. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Mycosis fungoides and Sézary syndrome are hard to cure. Treatment is usually palliative, to relieve symptoms and improve the quality of life. Patients with early stage disease may live many years. The process used to find out if cancer has spread from the skin to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if mycosis fungoides spreads to the liver, the cancer cells in the liver are actually mycosis fungoides cells. The disease is metastatic mycosis fungoides, not liver cancer. Stage I is divided into stages IA and IB as follows: There may be a low number of Sézary cells in the blood. Stage II is divided into stages IIA and IIB as follows: There may be a low number of Sézary cells in the blood. In stage III, 80% or more of the skin surface is reddened and may have patches, papules, plaques, or tumors. Lymph nodes may be abnormal, but they are not cancerous. There may be a low number of Sézary cells in the blood. When there is a high number of Sézary cells in the blood, the disease is called Sézary syndrome. Stage IV is divided into stages IVA1, IVA2, and IVB as follows: Mycosis fungoides and Sézary syndrome may come back in the skin or in other parts of the body, such as the spleen or liver. Different types of treatment are available for patients with mycosis fungoides and Sézary syndrome. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients undergoing photodynamic therapy will need to limit the amount of time spent in sunlight. There are different types of photodynamic therapy: Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Sometimes, total skin electron beam (TSEB) radiation therapy is used to treat mycosis fungoides and Sézary syndrome. This is a type of external radiation treatment in which a radiation therapy machine aims electrons (tiny, invisible particles) at the skin covering the whole body. External radiation therapy may also be used as palliative therapy to relieve symptoms and improve quality of life. Ultraviolet A (UVA) radiation therapy or ultraviolet B (UVB) radiation therapy may be given using a special lamp or laser that directs radiation at the skin. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Sometimes the chemotherapy is topical (put on the skin in a cream, lotion, or ointment). See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Topical corticosteroids are used to relieve red, swollen, and inflamed skin. They are a type of steroid. Topical corticosteroids may be in a cream, lotion, or ointment. Retinoids, such as bexarotene, are drugs related to vitamin A that can slow the growth of certain types of cancer cells. The retinoids may be taken by mouth or put on the skin. Lenalidomide is a drug that helps the immune system kill abnormal blood cells or cancer cells and may prevent the growth of new blood vessels that tumors need to grow. Vorinostat and romidepsin are two of the histone deacetylase (HDAC) inhibitors used to treat mycosis fungoides and Sézary syndrome. HDAC inhibitors cause a chemical change that stops tumor cells from dividing. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of monoclonal antibodies include: High doses of chemotherapy and sometimes radiation therapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and radiation therapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage I and stage II mycosis fungoides may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage III and stage IV mycosis fungoides including Sézary syndrome is palliative (to relieve symptoms and improve the quality of life) and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent mycosis fungoides including Sézary syndrome may be within a clinical trial and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about mycosis fungoides and Sézary syndrome, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of mycosis fungoides (including Sézary Syndrome). It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Mycosis Fungoides (Including Sézary Syndrome) Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/mycosis-fungoides-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389317] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Mycosis Fungoides (Including Sézary Syndrome) Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Mycosis Fungoides and the Szary Syndrome ?

Tests that examine the skin and blood are used to detect (find) and diagnose mycosis fungoides and the Szary syndrome. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps, the number and type of skin lesions, or anything else that seems unusual. Pictures of the skin and a history of the patients health habits and past illnesses and treatments will also be taken. - Complete blood count with differential : A procedure in which a sample of blood is drawn and checked for the following: - The number of red blood cells and platelets. - The number and type of white blood cells. - The amount of hemoglobin (the protein that carries oxygen) in the red blood cells. - The portion of the blood sample made up of red blood cells. - Peripheral blood smear : A procedure in which a sample of blood is viewed under a microscope to count different circulating blood cells (red blood cells, white blood cells, platelets, etc.) and see whether the cells look normal. - Skin biopsy : The removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. The doctor may remove a growth from the skin, which will be examined by a pathologist. More than one skin biopsy may be needed to diagnose mycosis fungoides. - Immunophenotyping : A process used to identify cells, based on the types of antigens or markers on the surface of the cell. This process may include special staining of the blood cells. It is used to diagnose specific types of leukemia and lymphoma by comparing the cancer cells to normal cells of the immune system. - T-cell receptor (TCR) gene rearrangement test: A laboratory test in which cells in a sample of tissue are checked to see if there is a certain change in the genes. This gene change can lead to too many of one kind of T-cells (white blood cells that fight infection) to be made. - Flow cytometry : A laboratory test that measures the number of cells in a sample of blood, the percentage of live cells in a sample, and certain characteristics of cells, such as size, shape, and the presence of tumor markers on the cell surface. The cells are stained with a light-sensitive dye, placed in a fluid, and passed in a stream before a laser or other type of light. The measurements are based on how the light-sensitive dye reacts to the light.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood stem cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A myeloid stem cell becomes a red blood cell, white blood cell, or platelet. A lymphoid stem cell becomes a lymphoblast and then one of three types of lymphocytes (white blood cells): In mycosis fungoides, T-cell lymphocytes become cancerous and affect the skin. When these lymphocytes occur in the blood, they are called Sézary cells. In Sézary syndrome, cancerous T-cell lymphocytes affect the skin and large numbers of Sézary cells are found in the blood. Mycosis fungoides and Sézary syndrome are the two most common types of cutaneous T-cell lymphoma (a type of non-Hodgkin lymphoma). For information about other types of skin cancer or non-Hodgkin lymphoma, see the following PDQ summaries: Mycosis fungoides may go through the following phases: Check with your doctor if you have any of these signs. Also, skin all over the body is reddened, itchy, peeling, and painful. There may also be patches, plaques, or tumors on the skin. It is not known if Sézary syndrome is an advanced form of mycosis fungoides or a separate disease. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Mycosis fungoides and Sézary syndrome are hard to cure. Treatment is usually palliative, to relieve symptoms and improve the quality of life. Patients with early stage disease may live many years. The process used to find out if cancer has spread from the skin to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if mycosis fungoides spreads to the liver, the cancer cells in the liver are actually mycosis fungoides cells. The disease is metastatic mycosis fungoides, not liver cancer. Stage I is divided into stages IA and IB as follows: There may be a low number of Sézary cells in the blood. Stage II is divided into stages IIA and IIB as follows: There may be a low number of Sézary cells in the blood. In stage III, 80% or more of the skin surface is reddened and may have patches, papules, plaques, or tumors. Lymph nodes may be abnormal, but they are not cancerous. There may be a low number of Sézary cells in the blood. When there is a high number of Sézary cells in the blood, the disease is called Sézary syndrome. Stage IV is divided into stages IVA1, IVA2, and IVB as follows: Mycosis fungoides and Sézary syndrome may come back in the skin or in other parts of the body, such as the spleen or liver. Different types of treatment are available for patients with mycosis fungoides and Sézary syndrome. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients undergoing photodynamic therapy will need to limit the amount of time spent in sunlight. There are different types of photodynamic therapy: Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Sometimes, total skin electron beam (TSEB) radiation therapy is used to treat mycosis fungoides and Sézary syndrome. This is a type of external radiation treatment in which a radiation therapy machine aims electrons (tiny, invisible particles) at the skin covering the whole body. External radiation therapy may also be used as palliative therapy to relieve symptoms and improve quality of life. Ultraviolet A (UVA) radiation therapy or ultraviolet B (UVB) radiation therapy may be given using a special lamp or laser that directs radiation at the skin. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Sometimes the chemotherapy is topical (put on the skin in a cream, lotion, or ointment). See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Topical corticosteroids are used to relieve red, swollen, and inflamed skin. They are a type of steroid. Topical corticosteroids may be in a cream, lotion, or ointment. Retinoids, such as bexarotene, are drugs related to vitamin A that can slow the growth of certain types of cancer cells. The retinoids may be taken by mouth or put on the skin. Lenalidomide is a drug that helps the immune system kill abnormal blood cells or cancer cells and may prevent the growth of new blood vessels that tumors need to grow. Vorinostat and romidepsin are two of the histone deacetylase (HDAC) inhibitors used to treat mycosis fungoides and Sézary syndrome. HDAC inhibitors cause a chemical change that stops tumor cells from dividing. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of monoclonal antibodies include: High doses of chemotherapy and sometimes radiation therapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and radiation therapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage I and stage II mycosis fungoides may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage III and stage IV mycosis fungoides including Sézary syndrome is palliative (to relieve symptoms and improve the quality of life) and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent mycosis fungoides including Sézary syndrome may be within a clinical trial and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about mycosis fungoides and Sézary syndrome, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of mycosis fungoides (including Sézary Syndrome). It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Mycosis Fungoides (Including Sézary Syndrome) Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/mycosis-fungoides-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389317] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Mycosis Fungoides (Including Sézary Syndrome) Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Mycosis Fungoides and the Szary Syndrome ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options depend on the following: - The stage of the cancer. - The type of lesion (patches, plaques, or tumors). Mycosis fungoides and the Szary syndrome are hard to cure. Treatment is usually palliative, to relieve symptoms and improve the quality of life. Patients with early stage disease may live many years.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood stem cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A myeloid stem cell becomes a red blood cell, white blood cell, or platelet. A lymphoid stem cell becomes a lymphoblast and then one of three types of lymphocytes (white blood cells): In mycosis fungoides, T-cell lymphocytes become cancerous and affect the skin. When these lymphocytes occur in the blood, they are called Sézary cells. In Sézary syndrome, cancerous T-cell lymphocytes affect the skin and large numbers of Sézary cells are found in the blood. Mycosis fungoides and Sézary syndrome are the two most common types of cutaneous T-cell lymphoma (a type of non-Hodgkin lymphoma). For information about other types of skin cancer or non-Hodgkin lymphoma, see the following PDQ summaries: Mycosis fungoides may go through the following phases: Check with your doctor if you have any of these signs. Also, skin all over the body is reddened, itchy, peeling, and painful. There may also be patches, plaques, or tumors on the skin. It is not known if Sézary syndrome is an advanced form of mycosis fungoides or a separate disease. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Mycosis fungoides and Sézary syndrome are hard to cure. Treatment is usually palliative, to relieve symptoms and improve the quality of life. Patients with early stage disease may live many years. The process used to find out if cancer has spread from the skin to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if mycosis fungoides spreads to the liver, the cancer cells in the liver are actually mycosis fungoides cells. The disease is metastatic mycosis fungoides, not liver cancer. Stage I is divided into stages IA and IB as follows: There may be a low number of Sézary cells in the blood. Stage II is divided into stages IIA and IIB as follows: There may be a low number of Sézary cells in the blood. In stage III, 80% or more of the skin surface is reddened and may have patches, papules, plaques, or tumors. Lymph nodes may be abnormal, but they are not cancerous. There may be a low number of Sézary cells in the blood. When there is a high number of Sézary cells in the blood, the disease is called Sézary syndrome. Stage IV is divided into stages IVA1, IVA2, and IVB as follows: Mycosis fungoides and Sézary syndrome may come back in the skin or in other parts of the body, such as the spleen or liver. Different types of treatment are available for patients with mycosis fungoides and Sézary syndrome. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients undergoing photodynamic therapy will need to limit the amount of time spent in sunlight. There are different types of photodynamic therapy: Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Sometimes, total skin electron beam (TSEB) radiation therapy is used to treat mycosis fungoides and Sézary syndrome. This is a type of external radiation treatment in which a radiation therapy machine aims electrons (tiny, invisible particles) at the skin covering the whole body. External radiation therapy may also be used as palliative therapy to relieve symptoms and improve quality of life. Ultraviolet A (UVA) radiation therapy or ultraviolet B (UVB) radiation therapy may be given using a special lamp or laser that directs radiation at the skin. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Sometimes the chemotherapy is topical (put on the skin in a cream, lotion, or ointment). See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Topical corticosteroids are used to relieve red, swollen, and inflamed skin. They are a type of steroid. Topical corticosteroids may be in a cream, lotion, or ointment. Retinoids, such as bexarotene, are drugs related to vitamin A that can slow the growth of certain types of cancer cells. The retinoids may be taken by mouth or put on the skin. Lenalidomide is a drug that helps the immune system kill abnormal blood cells or cancer cells and may prevent the growth of new blood vessels that tumors need to grow. Vorinostat and romidepsin are two of the histone deacetylase (HDAC) inhibitors used to treat mycosis fungoides and Sézary syndrome. HDAC inhibitors cause a chemical change that stops tumor cells from dividing. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of monoclonal antibodies include: High doses of chemotherapy and sometimes radiation therapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and radiation therapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage I and stage II mycosis fungoides may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage III and stage IV mycosis fungoides including Sézary syndrome is palliative (to relieve symptoms and improve the quality of life) and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent mycosis fungoides including Sézary syndrome may be within a clinical trial and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about mycosis fungoides and Sézary syndrome, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of mycosis fungoides (including Sézary Syndrome). It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Mycosis Fungoides (Including Sézary Syndrome) Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/mycosis-fungoides-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389317] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Mycosis Fungoides (Including Sézary Syndrome) Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Mycosis Fungoides and the Szary Syndrome ?

Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood stem cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A myeloid stem cell becomes a red blood cell, white blood cell, or platelet. A lymphoid stem cell becomes a lymphoblast and then one of three types of lymphocytes (white blood cells): In mycosis fungoides, T-cell lymphocytes become cancerous and affect the skin. When these lymphocytes occur in the blood, they are called Sézary cells. In Sézary syndrome, cancerous T-cell lymphocytes affect the skin and large numbers of Sézary cells are found in the blood. Mycosis fungoides and Sézary syndrome are the two most common types of cutaneous T-cell lymphoma (a type of non-Hodgkin lymphoma). For information about other types of skin cancer or non-Hodgkin lymphoma, see the following PDQ summaries: Mycosis fungoides may go through the following phases: Check with your doctor if you have any of these signs. Also, skin all over the body is reddened, itchy, peeling, and painful. There may also be patches, plaques, or tumors on the skin. It is not known if Sézary syndrome is an advanced form of mycosis fungoides or a separate disease. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Mycosis fungoides and Sézary syndrome are hard to cure. Treatment is usually palliative, to relieve symptoms and improve the quality of life. Patients with early stage disease may live many years. The process used to find out if cancer has spread from the skin to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if mycosis fungoides spreads to the liver, the cancer cells in the liver are actually mycosis fungoides cells. The disease is metastatic mycosis fungoides, not liver cancer. Stage I is divided into stages IA and IB as follows: There may be a low number of Sézary cells in the blood. Stage II is divided into stages IIA and IIB as follows: There may be a low number of Sézary cells in the blood. In stage III, 80% or more of the skin surface is reddened and may have patches, papules, plaques, or tumors. Lymph nodes may be abnormal, but they are not cancerous. There may be a low number of Sézary cells in the blood. When there is a high number of Sézary cells in the blood, the disease is called Sézary syndrome. Stage IV is divided into stages IVA1, IVA2, and IVB as follows: Mycosis fungoides and Sézary syndrome may come back in the skin or in other parts of the body, such as the spleen or liver. Different types of treatment are available for patients with mycosis fungoides and Sézary syndrome. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients undergoing photodynamic therapy will need to limit the amount of time spent in sunlight. There are different types of photodynamic therapy: Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Sometimes, total skin electron beam (TSEB) radiation therapy is used to treat mycosis fungoides and Sézary syndrome. This is a type of external radiation treatment in which a radiation therapy machine aims electrons (tiny, invisible particles) at the skin covering the whole body. External radiation therapy may also be used as palliative therapy to relieve symptoms and improve quality of life. Ultraviolet A (UVA) radiation therapy or ultraviolet B (UVB) radiation therapy may be given using a special lamp or laser that directs radiation at the skin. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Sometimes the chemotherapy is topical (put on the skin in a cream, lotion, or ointment). See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Topical corticosteroids are used to relieve red, swollen, and inflamed skin. They are a type of steroid. Topical corticosteroids may be in a cream, lotion, or ointment. Retinoids, such as bexarotene, are drugs related to vitamin A that can slow the growth of certain types of cancer cells. The retinoids may be taken by mouth or put on the skin. Lenalidomide is a drug that helps the immune system kill abnormal blood cells or cancer cells and may prevent the growth of new blood vessels that tumors need to grow. Vorinostat and romidepsin are two of the histone deacetylase (HDAC) inhibitors used to treat mycosis fungoides and Sézary syndrome. HDAC inhibitors cause a chemical change that stops tumor cells from dividing. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of monoclonal antibodies include: High doses of chemotherapy and sometimes radiation therapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and radiation therapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage I and stage II mycosis fungoides may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage III and stage IV mycosis fungoides including Sézary syndrome is palliative (to relieve symptoms and improve the quality of life) and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent mycosis fungoides including Sézary syndrome may be within a clinical trial and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about mycosis fungoides and Sézary syndrome, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of mycosis fungoides (including Sézary Syndrome). It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Mycosis Fungoides (Including Sézary Syndrome) Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/mycosis-fungoides-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389317] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Mycosis Fungoides (Including Sézary Syndrome) Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Mycosis Fungoides and the Szary Syndrome ?

Key Points - After mycosis fungoides and the Szary syndrome have been diagnosed, tests are done to find out if cancer cells have spread from the skin to other parts of the body. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - The following stages are used for mycosis fungoides and the Szary syndrome: - Stage I Mycosis Fungoides - Stage II Mycosis Fungoides - Stage III Mycosis Fungoides - Stage IV Mycosis Fungoides - Stage IV Szary Syndrome After mycosis fungoides and the Szary syndrome have been diagnosed, tests are done to find out if cancer cells have spread from the skin to other parts of the body. The process used to find out if cancer has spread from the skin to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: - Chest x-ray : An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the lymph nodes, chest, abdomen, and pelvis, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. - Lymph node biopsy : The removal of all or part of a lymph node. A pathologist views the tissue under a microscope to look for cancer cells. - Bone marrow aspiration and biopsy : The removal of bone marrow and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. A pathologist views the bone marrow and bone under a microscope to look for signs of cancer. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if mycosis fungoides spreads to the liver, the cancer cells in the liver are actually mycosis fungoides cells. The disease is metastatic mycosis fungoides, not liver cancer. The following stages are used for mycosis fungoides and the Szary syndrome: Stage I Mycosis Fungoides Stage I is divided into stage IA and stage IB as follows: - Stage IA: Less than 10% of the skin surface is covered with patches, papules, and/or plaques. - Stage IB: Ten percent or more of the skin surface is covered with patches, papules, and/or plaques. There may be abnormal lymphocytes in the blood but they are not cancerous. Stage II Mycosis Fungoides Stage II is divided into stage IIA and stage IIB as follows: - Stage IIA: Any amount of the skin surface is covered with patches, papules, and/or plaques. Lymph nodes are enlarged but cancer has not spread to them. - Stage IIB: One or more tumors that are 1 centimeter or larger are found on the skin. Lymph nodes may be enlarged but cancer has not spread to them. There may be abnormal lymphocytes in the blood but they are not cancerous. Stage III Mycosis Fungoides In stage III, nearly all of the skin is reddened and may have patches, papules, plaques, or tumors. Lymph nodes may be enlarged but cancer has not spread to them. There may be abnormal lymphocytes in the blood but they are not cancerous. Stage IV Mycosis Fungoides Stage IV is divided into stage IVA and stage IVB as follows: - Stage IVA: Most of the skin is reddened and any amount of the skin surface is covered with patches, papules, plaques, or tumors, and either: - cancer has spread to lymph nodes and there may be cancerous lymphocytes in the blood; or - there are cancerous lymphocytes in the blood and lymph nodes may be enlarged, but cancer has not spread to them. - Stage IVB: Most of the skin is reddened and any amount of the skin surface is covered with patches, papules, plaques, or tumors. Cancer has spread to other organs in the body. Lymph nodes may be enlarged and cancer may have spread to them. There may be cancerous lymphocytes in the blood. Stage IV Szary Syndrome In stage IV: - Most of the skin is reddened and covered with patches, papules, plaques, or tumors; and - There is a high level of cancerous lymphocytes in the blood; and - Lymph nodes may be enlarged and cancer may have spread to them.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood stem cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A myeloid stem cell becomes a red blood cell, white blood cell, or platelet. A lymphoid stem cell becomes a lymphoblast and then one of three types of lymphocytes (white blood cells): In mycosis fungoides, T-cell lymphocytes become cancerous and affect the skin. When these lymphocytes occur in the blood, they are called Sézary cells. In Sézary syndrome, cancerous T-cell lymphocytes affect the skin and large numbers of Sézary cells are found in the blood. Mycosis fungoides and Sézary syndrome are the two most common types of cutaneous T-cell lymphoma (a type of non-Hodgkin lymphoma). For information about other types of skin cancer or non-Hodgkin lymphoma, see the following PDQ summaries: Mycosis fungoides may go through the following phases: Check with your doctor if you have any of these signs. Also, skin all over the body is reddened, itchy, peeling, and painful. There may also be patches, plaques, or tumors on the skin. It is not known if Sézary syndrome is an advanced form of mycosis fungoides or a separate disease. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Mycosis fungoides and Sézary syndrome are hard to cure. Treatment is usually palliative, to relieve symptoms and improve the quality of life. Patients with early stage disease may live many years. The process used to find out if cancer has spread from the skin to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if mycosis fungoides spreads to the liver, the cancer cells in the liver are actually mycosis fungoides cells. The disease is metastatic mycosis fungoides, not liver cancer. Stage I is divided into stages IA and IB as follows: There may be a low number of Sézary cells in the blood. Stage II is divided into stages IIA and IIB as follows: There may be a low number of Sézary cells in the blood. In stage III, 80% or more of the skin surface is reddened and may have patches, papules, plaques, or tumors. Lymph nodes may be abnormal, but they are not cancerous. There may be a low number of Sézary cells in the blood. When there is a high number of Sézary cells in the blood, the disease is called Sézary syndrome. Stage IV is divided into stages IVA1, IVA2, and IVB as follows: Mycosis fungoides and Sézary syndrome may come back in the skin or in other parts of the body, such as the spleen or liver. Different types of treatment are available for patients with mycosis fungoides and Sézary syndrome. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients undergoing photodynamic therapy will need to limit the amount of time spent in sunlight. There are different types of photodynamic therapy: Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Sometimes, total skin electron beam (TSEB) radiation therapy is used to treat mycosis fungoides and Sézary syndrome. This is a type of external radiation treatment in which a radiation therapy machine aims electrons (tiny, invisible particles) at the skin covering the whole body. External radiation therapy may also be used as palliative therapy to relieve symptoms and improve quality of life. Ultraviolet A (UVA) radiation therapy or ultraviolet B (UVB) radiation therapy may be given using a special lamp or laser that directs radiation at the skin. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Sometimes the chemotherapy is topical (put on the skin in a cream, lotion, or ointment). See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Topical corticosteroids are used to relieve red, swollen, and inflamed skin. They are a type of steroid. Topical corticosteroids may be in a cream, lotion, or ointment. Retinoids, such as bexarotene, are drugs related to vitamin A that can slow the growth of certain types of cancer cells. The retinoids may be taken by mouth or put on the skin. Lenalidomide is a drug that helps the immune system kill abnormal blood cells or cancer cells and may prevent the growth of new blood vessels that tumors need to grow. Vorinostat and romidepsin are two of the histone deacetylase (HDAC) inhibitors used to treat mycosis fungoides and Sézary syndrome. HDAC inhibitors cause a chemical change that stops tumor cells from dividing. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and Sézary syndrome are types of non-Hodgkin lymphoma.) Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of monoclonal antibodies include: High doses of chemotherapy and sometimes radiation therapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and radiation therapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage I and stage II mycosis fungoides may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed stage III and stage IV mycosis fungoides including Sézary syndrome is palliative (to relieve symptoms and improve the quality of life) and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent mycosis fungoides including Sézary syndrome may be within a clinical trial and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about mycosis fungoides and Sézary syndrome, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of mycosis fungoides (including Sézary Syndrome). It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Mycosis Fungoides (Including Sézary Syndrome) Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/mycosis-fungoides-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389317] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Mycosis Fungoides (Including Sézary Syndrome) Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Mycosis Fungoides and the Szary Syndrome ?

Key Points - There are different types of treatment for patients with mycosis fungoides and the Szary syndrome cancer. - Six types of standard treatment are used: - Photodynamic therapy - Radiation therapy - Chemotherapy - Other drug therapy - Biologic therapy - Targeted therapy - New types of treatment are being tested in clinical trials. - High-dose chemotherapy and radiation therapy with stem cell transplant - Treatment for mycosis fungoides and the Szary syndrome may cause side effects. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with mycosis fungoides and the Szary syndrome cancer. Different types of treatment are available for patients with mycosis fungoides and the Szary syndrome. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Six types of standard treatment are used: Photodynamic therapy Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients undergoing photodynamic therapy will need to limit the amount of time spent in sunlight. In one type of photodynamic therapy, called psoralen and ultraviolet A (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet radiation is directed to the skin. In another type of photodynamic therapy, called extracorporeal photochemotherapy, the patient is given drugs and then some blood cells are taken from the body, put under a special ultraviolet A light, and put back into the body. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy is used to treat mycosis fungoides and the Szary syndrome, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Sometimes, total skin electron beam (TSEB) radiation therapy is used to treat mycosis fungoides and the Szary syndrome. This is a type of external radiation treatment in which a radiation therapy machine aims electrons (tiny, invisible particles) at the skin covering the whole body. Ultraviolet B (UVB) radiation therapy uses a special lamp or laser that directs UVB radiation at the skin. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Sometimes the chemotherapy is topical (put on the skin in a cream, lotion, or ointment). The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and the Szary syndrome are types of non-Hodgkin lymphoma.) Other drug therapy Topical corticosteroids are used to relieve red, swollen, and inflamed skin. They are a type of steroid. Topical corticosteroids may be in a cream, lotion, or ointment. Retinoids, such as bexarotene, are drugs related to vitamin A that can slow the growth of certain types of cancer cells. The retinoids may be taken by mouth or put on the skin. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and the Szary syndrome are types of non-Hodgkin lymphoma.) Biologic therapy Biologic therapy is a treatment that uses the patient&apos;s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body&apos;s natural defenses against cancer. This type of cancer treatment is also called biotherapy or immunotherapy. Interferon is a type of biologic therapy used to treat mycosis fungoides and the Szary syndrome. It interferes with the division of cancer cells and can slow tumor growth. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and the Szary syndrome are types of non-Hodgkin lymphoma.) Targeted therapy Targeted therapy is a treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Monoclonal antibody therapy and other types of targeted therapies are used to treat mycosis fungoides and the Szary syndrome. Alemtuzumab is a monoclonal antibody used to treat mycosis fungoides and the Szary syndrome. Monoclonal antibody therapy uses antibodies made in the laboratory, from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Other types of monoclonal antibody therapy are being studied in clinical trials to treat mycosis fungoides and the Szary syndrome. Vorinostat and romidepsin are two of the histone deacetylase (HDAC) inhibitors used to treat mycosis fungoides and the Szary syndrome. HDAC inhibitors cause a chemical change that stops tumor cells from dividing. Pralatrexate is a dihydrofolate reductase (DHFR) inhibitor used to treat mycosis fungoides and the Szary syndrome. It builds up in cancer cells and stops them from using folate, a nutrient needed for cells to divide. Pralatrexate may slow the growth of tumors and kill cancer cells. See Drugs Approved for Non-Hodgkin Lymphoma for more information. (Mycosis fungoides and the Szary syndrome are types of non-Hodgkin lymphoma.) New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy and radiation therapy with stem cell transplant This treatment is a method of giving high doses of chemotherapy and radiation therapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the bone marrow or blood of the patient or a donor and are frozen and stored. After therapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body&apos;s blood cells. Treatment for mycosis fungoides and the Szary syndrome may cause side effects. For information about side effects caused by treatment for cancer, see our Side Effects page. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options by Stage Stage I Mycosis Fungoides Treatment of stage I mycosis fungoides may include the following: - PUVA therapy. Biologic therapy may also be given. - Radiation therapy. In some cases, radiation therapy is given to skin lesions, as palliative therapy to reduce tumor size or relieve symptoms and improve quality of life. - Topical corticosteroid therapy. - Retinoid therapy. - Topical or systemic chemotherapy. - Biologic therapy. Topical chemotherapy may also be given. - Targeted therapy. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I mycosis fungoides/Sezary syndrome. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage II Mycosis Fungoides Treatment of stage II mycosis fungoides is palliative (to relieve symptoms and improve the quality of life) and may include the following: - PUVA therapy. Biologic therapy may also be given. - Radiation therapy. - Topical corticosteroid therapy. - Retinoid therapy. - Topical or systemic chemotherapy. - Biologic therapy. Topical chemotherapy may also be given. - Targeted therapy. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage II mycosis fungoides/Sezary syndrome. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage III Mycosis Fungoides Treatment of stage III mycosis fungoides is palliative (to relieve symptoms and improve the quality of life) and may include the following: - PUVA therapy. Systemic chemotherapy or biologic therapy may also be given. - Extracorporeal photochemotherapy. - Radiation therapy. - Topical corticosteroid therapy. - Retinoid therapy. - Systemic chemotherapy with one or more drugs. Topical chemotherapy or radiation therapy may also be given. - Topical chemotherapy. - Biologic therapy. Topical chemotherapy may also be given. - Targeted therapy. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage III mycosis fungoides/Sezary syndrome. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IV Mycosis Fungoides and the Szary Syndrome Treatment of stage IV mycosis fungoides and stage IV Szary syndrome is palliative (to relieve symptoms and improve the quality of life) and may include the following: - PUVA therapy. Systemic chemotherapy or biologic therapy may also be given. - Extracorporeal photochemotherapy. Radiation therapy may also be given. - Radiation therapy. - Topical corticosteroid therapy. - Retinoid therapy. - Systemic chemotherapy with one or more drugs, or topical chemotherapy. - Biologic therapy. Topical chemotherapy may also be given. - Targeted therapy. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage IV mycosis fungoides/Sezary syndrome. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases. The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative neoplasms include the following 6 types: These types are described below. Chronic myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made. The following tests and procedures may be used: Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment. In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells, white blood cells, or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis. Polycythemia vera often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may occur as the number of blood cells increases. Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of the following: In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made). In polycythemia vera, the erythropoietin level would be lower than normal because the body does not need to make more red blood cells. The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells. Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following: Prognosis depends on the following: Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow. Essential thrombocythemia often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by essential thrombocythemia or by other conditions. Check with your doctor if you have any of the following: Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack. Prognosis and treatment options depend on the following: Chronic neutrophilic leukemia is a disease in which too many blood stem cells become a type of white blood cell called neutrophils. Neutrophils are infection-fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia. Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia. Chronic eosinophilic leukemia may not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for chronic myeloproliferative neoplasms. It is important to know the type of myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with chronic myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative neoplasms. Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream. Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Myeloproliferative Neoplasms for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body, such as the spleen, with cancer. Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis. Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots. Thalidomide, lenalidomide, and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells. Erythropoietic growth factors are used to stimulate the bone marrow to make red blood cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Other types of targeted therapies are being studied in clinical trials. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of essential thrombocythemia in patients younger than 60 years who have no signs or symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic neutrophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic eosinophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. 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What is (are) Chronic Myeloproliferative Neoplasms ?

Key Points - Myeloproliferative neoplasms are a group of diseases in which the bone marrow makes too many red blood cells, white blood cells, or platelets. - There are 6 types of chronic myeloproliferative neoplasms. - Tests that examine the blood and bone marrow are used to detect (find) and diagnose chronic myeloproliferative neoplasms. Myeloproliferative neoplasms are a group of diseases in which the bone marrow makes too many red blood cells, white blood cells, or platelets. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: - Red blood cells that carry oxygen and other substances to all tissues of the body. - White blood cells that fight infection and disease. - Platelets that form blood clots to stop bleeding. In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases. There are 6 types of chronic myeloproliferative neoplasms. The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative neoplasms include the following 6 types: - Chronic myelogenous leukemia. - Polycythemia vera. - Primary myelofibrosis (also called chronic idiopathic myelofibrosis). - Essential thrombocythemia. - Chronic neutrophilic leukemia. - Chronic eosinophilic leukemia. These types are described below. Chronic myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases. The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative neoplasms include the following 6 types: These types are described below. Chronic myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made. The following tests and procedures may be used: Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment. In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells, white blood cells, or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis. Polycythemia vera often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may occur as the number of blood cells increases. Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of the following: In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made). In polycythemia vera, the erythropoietin level would be lower than normal because the body does not need to make more red blood cells. The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells. Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following: Prognosis depends on the following: Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow. Essential thrombocythemia often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by essential thrombocythemia or by other conditions. Check with your doctor if you have any of the following: Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack. Prognosis and treatment options depend on the following: Chronic neutrophilic leukemia is a disease in which too many blood stem cells become a type of white blood cell called neutrophils. Neutrophils are infection-fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia. Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia. Chronic eosinophilic leukemia may not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for chronic myeloproliferative neoplasms. It is important to know the type of myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with chronic myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative neoplasms. Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream. Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Myeloproliferative Neoplasms for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body, such as the spleen, with cancer. Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis. Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots. Thalidomide, lenalidomide, and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells. Erythropoietic growth factors are used to stimulate the bone marrow to make red blood cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Other types of targeted therapies are being studied in clinical trials. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of essential thrombocythemia in patients younger than 60 years who have no signs or symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic neutrophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic eosinophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/chronic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389435] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Chronic Myeloproliferative Neoplasms ?

Tests that examine the blood and bone marrow are used to detect (find) and diagnose chronic myeloproliferative neoplasms. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Complete blood count (CBC) with differential : A procedure in which a sample of blood is drawn and checked for the following: - The number of red blood cells and platelets. - The number and type of white blood cells. - The amount of hemoglobin (the protein that carries oxygen) in the red blood cells. - The portion of the blood sample made up of red blood cells. - Peripheral blood smear : A procedure in which a sample of blood is checked for the following: - Whether there are red blood cells shaped like teardrops. - The number and kinds of white blood cells. - The number of platelets. - Whether there are blast cells. - Blood chemistry studies : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease. - Bone marrow aspiration and biopsy : The removal of bone marrow, blood, and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for abnormal cells. - Cytogenetic analysis : A test in which cells in a sample of blood or bone marrow are viewed under a microscope to look for certain changes in the chromosomes. Certain diseases or disorders may be diagnosed or ruled out based on the chromosomal changes. - Gene mutation test: A laboratory test done on a bone marrow or blood sample to check for mutations in JAK2 , MPL , or CALR genes. A JAK2 gene mutation is often found in patients with polycythemia vera, essential thrombocythemia, or primary myelofibrosis. MPL or CALR gene mutations are found in patients with essential thrombocythemia or primary myelofibrosis.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases. The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative neoplasms include the following 6 types: These types are described below. Chronic myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made. The following tests and procedures may be used: Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment. In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells, white blood cells, or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis. Polycythemia vera often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may occur as the number of blood cells increases. Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of the following: In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made). In polycythemia vera, the erythropoietin level would be lower than normal because the body does not need to make more red blood cells. The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells. Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following: Prognosis depends on the following: Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow. Essential thrombocythemia often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by essential thrombocythemia or by other conditions. Check with your doctor if you have any of the following: Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack. Prognosis and treatment options depend on the following: Chronic neutrophilic leukemia is a disease in which too many blood stem cells become a type of white blood cell called neutrophils. Neutrophils are infection-fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia. Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia. Chronic eosinophilic leukemia may not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for chronic myeloproliferative neoplasms. It is important to know the type of myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with chronic myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative neoplasms. Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream. Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Myeloproliferative Neoplasms for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body, such as the spleen, with cancer. Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis. Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots. Thalidomide, lenalidomide, and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells. Erythropoietic growth factors are used to stimulate the bone marrow to make red blood cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Other types of targeted therapies are being studied in clinical trials. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of essential thrombocythemia in patients younger than 60 years who have no signs or symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic neutrophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic eosinophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/chronic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389435] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Chronic Myeloproliferative Neoplasms ?

Key Points - There is no standard staging system for chronic myeloproliferative neoplasms. There is no standard staging system for chronic myeloproliferative neoplasms. Staging is the process used to find out how far the cancer has spread. There is no standard staging system for chronic myeloproliferative neoplasms. Treatment is based on the type of myeloproliferative neoplasm the patient has. It is important to know the type in order to plan treatment.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases. The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative neoplasms include the following 6 types: These types are described below. Chronic myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made. The following tests and procedures may be used: Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment. In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells, white blood cells, or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis. Polycythemia vera often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may occur as the number of blood cells increases. Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of the following: In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made). In polycythemia vera, the erythropoietin level would be lower than normal because the body does not need to make more red blood cells. The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells. Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following: Prognosis depends on the following: Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow. Essential thrombocythemia often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by essential thrombocythemia or by other conditions. Check with your doctor if you have any of the following: Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack. Prognosis and treatment options depend on the following: Chronic neutrophilic leukemia is a disease in which too many blood stem cells become a type of white blood cell called neutrophils. Neutrophils are infection-fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia. Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia. Chronic eosinophilic leukemia may not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for chronic myeloproliferative neoplasms. It is important to know the type of myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with chronic myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative neoplasms. Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream. Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Myeloproliferative Neoplasms for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body, such as the spleen, with cancer. Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis. Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots. Thalidomide, lenalidomide, and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells. Erythropoietic growth factors are used to stimulate the bone marrow to make red blood cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Other types of targeted therapies are being studied in clinical trials. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of essential thrombocythemia in patients younger than 60 years who have no signs or symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic neutrophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic eosinophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/chronic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389435] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Chronic Myeloproliferative Neoplasms ?

Key Points - There are different types of treatment for patients with chronic myeloproliferative neoplasms. - Eleven types of standard treatment are used: - Watchful waiting - Phlebotomy - Platelet apheresis - Transfusion therapy - Chemotherapy - Radiation therapy - Other drug therapy - Surgery - Biologic therapy - Targeted therapy - High-dose chemotherapy with stem cell transplant - New types of treatment are being tested in clinical trials. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with chronic myeloproliferative neoplasms. Different types of treatments are available for patients with chronic myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Eleven types of standard treatment are used: Watchful waiting Watchful waiting is closely monitoring a patients condition without giving any treatment until signs or symptoms appear or change. Phlebotomy Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative neoplasms. Platelet apheresis Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patients bloodstream. Transfusion therapy Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Myeloproliferative Neoplasms for more information. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type of cancer being treated. External radiation therapy is used to treat chronic myeloproliferative neoplasms, and is usually directed at the spleen. Other drug therapy Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis. Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots. Thalidomide, lenalidomide, and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Surgery Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged. Biologic therapy Biologic therapy is a treatment that uses the patient&apos;s immune system to fight cancer or other diseases. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body&apos;s natural defenses against disease. This type of treatment is also called biotherapy or immunotherapy. Interferon alfa and pegylated interferon alpha are biologic agents commonly used to treat some chronic myeloproliferative neoplasms. Erythropoietic growth factors are also biologic agents. They are used to stimulate the bone marrow to make red blood cells. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Tyrosine kinase inhibitors are targeted therapy drugs that block signals needed for tumors to grow. Ruxolitinib is a tyrosine kinase inhibitor used to treat certain types of myelofibrosis. See Drugs Approved for Myeloproliferative Neoplasms for more information. Other types of targeted therapies are being studied in clinical trials. High-dose chemotherapy with stem cell transplant High-dose chemotherapy with stem cell transplant is a method of giving high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body&apos;s blood cells. New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Chronic Myeloproliferative Neoplasms Chronic Myelogenous Leukemia See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information. Polycythemia Vera The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following: - Phlebotomy. - Chemotherapy with or without phlebotomy. - Biologic therapy using interferon alfa or pegylated interferon alpha. - Low-dose aspirin. Check the list of NCI-supported cancer clinical trials that are now accepting patients with polycythemia vera. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Primary Myelofibrosis Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: - Erythropoietic growth factors. - Prednisone. - Danazol. - Thalidomide, lenalidomide, or pomalidomide, with or without prednisone. Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: - Targeted therapy with ruxolitinib. - Chemotherapy. - Donor stem cell transplant. - Thalidomide, lenalidomide, or pomalidomide. - Splenectomy. - Radiation therapy to the spleen, lymph nodes, or other areas outside the bone marrow where blood cells are forming. - Biologic therapy using interferon alfa or erythropoietic growth factors. - A clinical trial of other targeted therapy drugs. Check the list of NCI-supported cancer clinical trials that are now accepting patients with primary myelofibrosis. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Essential Thrombocythemia Treatment of essential thrombocythemia in patients younger than 60 years who have no signs or symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following: - Chemotherapy. - Anagrelide therapy. - Biologic therapy using interferon alfa or pegylated interferon alpha. - Platelet apheresis. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with essential thrombocythemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Chronic Neutrophilic Leukemia Treatment of chronic neutrophilic leukemia may include the following: - Donor bone marrow transplant. - Chemotherapy. - Biologic therapy using interferon alfa. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with chronic neutrophilic leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Chronic Eosinophilic Leukemia Treatment of chronic eosinophilic leukemia may include the following: - Bone marrow transplant. - Biologic therapy using interferon alfa. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with chronic eosinophilic leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

The rectum is part of the body’s digestive system. The digestive system takes in nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The digestive system is made up of the esophagus, stomach, and the small and large intestines. The colon (large bowel) is the first part of the large intestine and is about 5 feet long. Together, the rectum and anal canal make up the last part of the large intestine and are 6 to 8 inches long. The anal canal ends at the anus (the opening of the large intestine to the outside of the body).EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. For more information about rectal cancer, see: Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk to your doctor if you think you may be at risk for colorectal cancer. Risk factors for colorectal cancer include the following: Older age is a main risk factor for most cancers. The chance of getting cancer increases as you get older. These and other signs and symptoms may be caused by rectal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out whether cancer has spread within the rectum or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if rectal cancer spreads to the lung, the cancer cells in the lung are actually rectal cancer cells. The disease is metastatic rectal cancer, not lung cancer. In stage 0 rectal cancer, abnormal cells are found in the mucosa (innermost layer) of the rectum wall. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. In stage I rectal cancer, cancer has formed in the mucosa (innermost layer) of the rectum wall and has spread to the submucosa (layer of tissue next to the mucosa) or to the muscle layer of the rectum wall. Stage II rectal cancer is divided into stages IIA, IIB, and IIC. Stage III rectal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IIIA, cancer has spread: In stage IIIB, cancer has spread: In stage IIIC, cancer has spread: Stage IV rectal cancer is divided into stages IVA, IVB, and IVC. The cancer may come back in the rectum or in other parts of the body, such as the colon, pelvis, liver, or lungs. Different types of treatment are available for patients with rectal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for all stages of rectal cancer. The cancer is removed using one of the following types of surgery: After the cancer is removed, the surgeon will either: Radiation therapy and/or chemotherapy may be given before surgery to shrink the tumor, make it easier to remove the cancer, and help with bowel control after surgery. Treatment given before surgery is called neoadjuvant therapy. After all the cancer that can be seen at the time of the surgery is removed, some patients may be given radiation therapy and/or chemotherapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Short-course preoperative radiation therapy is used in some types of rectal cancer. This treatment uses fewer and lower doses of radiation than standard treatment, followed by surgery several days after the last dose. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly in the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Chemoembolization of the hepatic artery is a type of regional chemotherapy that may be used to treat cancer that has spread to the liver. This is done by blocking the hepatic artery (the main artery that supplies blood to the liver) and injecting anticancer drugs between the blockage and the liver. The liver’s arteries then carry the drugs into the liver. Only a small amount of the drug reaches other parts of the body. The blockage may be temporary or permanent, depending on what is used to block the artery. The liver continues to receive some blood from the hepatic portal vein, which carries blood from the stomach and intestine. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Colon and Rectal Cancer. Active surveillance is closely following a patient's condition without giving any treatment unless there are changes in test results. It is used to find early signs that the condition is getting worse. In active surveillance, patients are given certain exams and tests to check if the cancer is growing. When the cancer begins to grow, treatment is given to cure the cancer. Tests include the following: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapies used in the treatment of rectal cancer include the following: There are different types of monoclonal antibody therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy: Immune checkpoint inhibitors block proteins called checkpoints that are made by some types of immune system cells, such as T cells, and some cancer cells. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. They are used to treat some patients with metastatic colorectal cancer. There are two types of immune checkpoint inhibitor therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. After treatment for rectal cancer, a blood test to measure amounts of carcinoembryonic antigen (a substance in the blood that may be increased when cancer is present) may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II and stage III rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV and recurrent rectal cancer may include the following: Treatment of rectal cancer that has spread to other organs depends on where the cancer has spread. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about rectal cancer, see: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of rectal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Rectal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/colorectal/patient/rectal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389378] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Rectal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Rectal Cancer ?

Key Points - Rectal cancer is a disease in which malignant (cancer) cells form in the tissues of the rectum. - Health history affects the risk of developing rectal cancer. - Signs of rectal cancer include a change in bowel habits or blood in the stool. - Tests that examine the rectum and colon are used to detect (find) and diagnose rectal cancer. - Certain factors affect prognosis (chance of recovery) and treatment options. Rectal cancer is a disease in which malignant (cancer) cells form in the tissues of the rectum. The rectum is part of the bodys digestive system. The digestive system takes in nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The digestive system is made up of the esophagus, stomach, and the small and large intestines. The colon (large bowel) is the first part of the large intestine and is about 5 feet long. Together, the rectum and anal canal make up the last part of the large intestine and are 6-8 inches long. The anal canal ends at the anus (the opening of the large intestine to the outside of the body). See the following PDQ summaries for more information about rectal cancer: - Unusual Cancers of Childhood Treatment (see Colorectal Cancer section) - Colorectal Cancer Prevention - Colorectal Cancer Screening - Gastrointestinal Stromal Tumors Treatment - Genetics of Colorectal Cancer

The rectum is part of the body’s digestive system. The digestive system takes in nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The digestive system is made up of the esophagus, stomach, and the small and large intestines. The colon (large bowel) is the first part of the large intestine and is about 5 feet long. Together, the rectum and anal canal make up the last part of the large intestine and are 6 to 8 inches long. The anal canal ends at the anus (the opening of the large intestine to the outside of the body).EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. For more information about rectal cancer, see: Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk to your doctor if you think you may be at risk for colorectal cancer. Risk factors for colorectal cancer include the following: Older age is a main risk factor for most cancers. The chance of getting cancer increases as you get older. These and other signs and symptoms may be caused by rectal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out whether cancer has spread within the rectum or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if rectal cancer spreads to the lung, the cancer cells in the lung are actually rectal cancer cells. The disease is metastatic rectal cancer, not lung cancer. In stage 0 rectal cancer, abnormal cells are found in the mucosa (innermost layer) of the rectum wall. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. In stage I rectal cancer, cancer has formed in the mucosa (innermost layer) of the rectum wall and has spread to the submucosa (layer of tissue next to the mucosa) or to the muscle layer of the rectum wall. Stage II rectal cancer is divided into stages IIA, IIB, and IIC. Stage III rectal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IIIA, cancer has spread: In stage IIIB, cancer has spread: In stage IIIC, cancer has spread: Stage IV rectal cancer is divided into stages IVA, IVB, and IVC. The cancer may come back in the rectum or in other parts of the body, such as the colon, pelvis, liver, or lungs. Different types of treatment are available for patients with rectal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for all stages of rectal cancer. The cancer is removed using one of the following types of surgery: After the cancer is removed, the surgeon will either: Radiation therapy and/or chemotherapy may be given before surgery to shrink the tumor, make it easier to remove the cancer, and help with bowel control after surgery. Treatment given before surgery is called neoadjuvant therapy. After all the cancer that can be seen at the time of the surgery is removed, some patients may be given radiation therapy and/or chemotherapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Short-course preoperative radiation therapy is used in some types of rectal cancer. This treatment uses fewer and lower doses of radiation than standard treatment, followed by surgery several days after the last dose. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly in the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Chemoembolization of the hepatic artery is a type of regional chemotherapy that may be used to treat cancer that has spread to the liver. This is done by blocking the hepatic artery (the main artery that supplies blood to the liver) and injecting anticancer drugs between the blockage and the liver. The liver’s arteries then carry the drugs into the liver. Only a small amount of the drug reaches other parts of the body. The blockage may be temporary or permanent, depending on what is used to block the artery. The liver continues to receive some blood from the hepatic portal vein, which carries blood from the stomach and intestine. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Colon and Rectal Cancer. Active surveillance is closely following a patient's condition without giving any treatment unless there are changes in test results. It is used to find early signs that the condition is getting worse. In active surveillance, patients are given certain exams and tests to check if the cancer is growing. When the cancer begins to grow, treatment is given to cure the cancer. Tests include the following: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapies used in the treatment of rectal cancer include the following: There are different types of monoclonal antibody therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy: Immune checkpoint inhibitors block proteins called checkpoints that are made by some types of immune system cells, such as T cells, and some cancer cells. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. They are used to treat some patients with metastatic colorectal cancer. There are two types of immune checkpoint inhibitor therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. After treatment for rectal cancer, a blood test to measure amounts of carcinoembryonic antigen (a substance in the blood that may be increased when cancer is present) may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II and stage III rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV and recurrent rectal cancer may include the following: Treatment of rectal cancer that has spread to other organs depends on where the cancer has spread. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about rectal cancer, see: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of rectal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Rectal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/colorectal/patient/rectal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389378] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Rectal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Rectal Cancer? ?

Health history affects the risk of developing rectal cancer. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesnt mean that you will not get cancer. Talk to your doctor if you think you may be at risk for colorectal cancer. Risk factors for colorectal cancer include the following: - Having a family history of colon or rectal cancer in a first-degree relative (parent, sibling, or child). - Having a personal history of cancer of the colon, rectum, or ovary. - Having a personal history of high-risk adenomas (colorectal polyps that are 1 centimeter or larger in size or that have cells that look abnormal under a microscope). - Having inherited changes in certain genes that increase the risk of familial adenomatous polyposis (FAP) or Lynch syndrome (hereditary nonpolyposis colorectal cancer). - Having a personal history of chronic ulcerative colitis or Crohn disease for 8 years or more. - Having three or more alcoholic drinks per day. - Smoking cigarettes. - Being black. - Being obese. Older age is a main risk factor for most cancers. The chance of getting cancer increases as you get older.

The rectum is part of the body’s digestive system. The digestive system takes in nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The digestive system is made up of the esophagus, stomach, and the small and large intestines. The colon (large bowel) is the first part of the large intestine and is about 5 feet long. Together, the rectum and anal canal make up the last part of the large intestine and are 6 to 8 inches long. The anal canal ends at the anus (the opening of the large intestine to the outside of the body).EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. For more information about rectal cancer, see: Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk to your doctor if you think you may be at risk for colorectal cancer. Risk factors for colorectal cancer include the following: Older age is a main risk factor for most cancers. The chance of getting cancer increases as you get older. These and other signs and symptoms may be caused by rectal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out whether cancer has spread within the rectum or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if rectal cancer spreads to the lung, the cancer cells in the lung are actually rectal cancer cells. The disease is metastatic rectal cancer, not lung cancer. In stage 0 rectal cancer, abnormal cells are found in the mucosa (innermost layer) of the rectum wall. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. In stage I rectal cancer, cancer has formed in the mucosa (innermost layer) of the rectum wall and has spread to the submucosa (layer of tissue next to the mucosa) or to the muscle layer of the rectum wall. Stage II rectal cancer is divided into stages IIA, IIB, and IIC. Stage III rectal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IIIA, cancer has spread: In stage IIIB, cancer has spread: In stage IIIC, cancer has spread: Stage IV rectal cancer is divided into stages IVA, IVB, and IVC. The cancer may come back in the rectum or in other parts of the body, such as the colon, pelvis, liver, or lungs. Different types of treatment are available for patients with rectal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for all stages of rectal cancer. The cancer is removed using one of the following types of surgery: After the cancer is removed, the surgeon will either: Radiation therapy and/or chemotherapy may be given before surgery to shrink the tumor, make it easier to remove the cancer, and help with bowel control after surgery. Treatment given before surgery is called neoadjuvant therapy. After all the cancer that can be seen at the time of the surgery is removed, some patients may be given radiation therapy and/or chemotherapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Short-course preoperative radiation therapy is used in some types of rectal cancer. This treatment uses fewer and lower doses of radiation than standard treatment, followed by surgery several days after the last dose. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly in the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Chemoembolization of the hepatic artery is a type of regional chemotherapy that may be used to treat cancer that has spread to the liver. This is done by blocking the hepatic artery (the main artery that supplies blood to the liver) and injecting anticancer drugs between the blockage and the liver. The liver’s arteries then carry the drugs into the liver. Only a small amount of the drug reaches other parts of the body. The blockage may be temporary or permanent, depending on what is used to block the artery. The liver continues to receive some blood from the hepatic portal vein, which carries blood from the stomach and intestine. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Colon and Rectal Cancer. Active surveillance is closely following a patient's condition without giving any treatment unless there are changes in test results. It is used to find early signs that the condition is getting worse. In active surveillance, patients are given certain exams and tests to check if the cancer is growing. When the cancer begins to grow, treatment is given to cure the cancer. Tests include the following: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapies used in the treatment of rectal cancer include the following: There are different types of monoclonal antibody therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy: Immune checkpoint inhibitors block proteins called checkpoints that are made by some types of immune system cells, such as T cells, and some cancer cells. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. They are used to treat some patients with metastatic colorectal cancer. There are two types of immune checkpoint inhibitor therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. After treatment for rectal cancer, a blood test to measure amounts of carcinoembryonic antigen (a substance in the blood that may be increased when cancer is present) may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II and stage III rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV and recurrent rectal cancer may include the following: Treatment of rectal cancer that has spread to other organs depends on where the cancer has spread. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about rectal cancer, see: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of rectal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Rectal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/colorectal/patient/rectal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389378] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Rectal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Rectal Cancer ?

Signs of rectal cancer include a change in bowel habits or blood in the stool. These and other signs and symptoms may be caused by rectal cancer or by other conditions. Check with your doctor if you have any of the following: - Blood (either bright red or very dark) in the stool. - A change in bowel habits. - Diarrhea. - Constipation. - Feeling that the bowel does not empty completely. - Stools that are narrower or have a different shape than usual. - General abdominal discomfort (frequent gas pains, bloating, fullness, or cramps). - Change in appetite. - Weight loss for no known reason. - Feeling very tired.

The rectum is part of the body’s digestive system. The digestive system takes in nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The digestive system is made up of the esophagus, stomach, and the small and large intestines. The colon (large bowel) is the first part of the large intestine and is about 5 feet long. Together, the rectum and anal canal make up the last part of the large intestine and are 6 to 8 inches long. The anal canal ends at the anus (the opening of the large intestine to the outside of the body).EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. For more information about rectal cancer, see: Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk to your doctor if you think you may be at risk for colorectal cancer. Risk factors for colorectal cancer include the following: Older age is a main risk factor for most cancers. The chance of getting cancer increases as you get older. These and other signs and symptoms may be caused by rectal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out whether cancer has spread within the rectum or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if rectal cancer spreads to the lung, the cancer cells in the lung are actually rectal cancer cells. The disease is metastatic rectal cancer, not lung cancer. In stage 0 rectal cancer, abnormal cells are found in the mucosa (innermost layer) of the rectum wall. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. In stage I rectal cancer, cancer has formed in the mucosa (innermost layer) of the rectum wall and has spread to the submucosa (layer of tissue next to the mucosa) or to the muscle layer of the rectum wall. Stage II rectal cancer is divided into stages IIA, IIB, and IIC. Stage III rectal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IIIA, cancer has spread: In stage IIIB, cancer has spread: In stage IIIC, cancer has spread: Stage IV rectal cancer is divided into stages IVA, IVB, and IVC. The cancer may come back in the rectum or in other parts of the body, such as the colon, pelvis, liver, or lungs. Different types of treatment are available for patients with rectal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for all stages of rectal cancer. The cancer is removed using one of the following types of surgery: After the cancer is removed, the surgeon will either: Radiation therapy and/or chemotherapy may be given before surgery to shrink the tumor, make it easier to remove the cancer, and help with bowel control after surgery. Treatment given before surgery is called neoadjuvant therapy. After all the cancer that can be seen at the time of the surgery is removed, some patients may be given radiation therapy and/or chemotherapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Short-course preoperative radiation therapy is used in some types of rectal cancer. This treatment uses fewer and lower doses of radiation than standard treatment, followed by surgery several days after the last dose. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly in the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Chemoembolization of the hepatic artery is a type of regional chemotherapy that may be used to treat cancer that has spread to the liver. This is done by blocking the hepatic artery (the main artery that supplies blood to the liver) and injecting anticancer drugs between the blockage and the liver. The liver’s arteries then carry the drugs into the liver. Only a small amount of the drug reaches other parts of the body. The blockage may be temporary or permanent, depending on what is used to block the artery. The liver continues to receive some blood from the hepatic portal vein, which carries blood from the stomach and intestine. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Colon and Rectal Cancer. Active surveillance is closely following a patient's condition without giving any treatment unless there are changes in test results. It is used to find early signs that the condition is getting worse. In active surveillance, patients are given certain exams and tests to check if the cancer is growing. When the cancer begins to grow, treatment is given to cure the cancer. Tests include the following: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapies used in the treatment of rectal cancer include the following: There are different types of monoclonal antibody therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy: Immune checkpoint inhibitors block proteins called checkpoints that are made by some types of immune system cells, such as T cells, and some cancer cells. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. They are used to treat some patients with metastatic colorectal cancer. There are two types of immune checkpoint inhibitor therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. After treatment for rectal cancer, a blood test to measure amounts of carcinoembryonic antigen (a substance in the blood that may be increased when cancer is present) may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II and stage III rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV and recurrent rectal cancer may include the following: Treatment of rectal cancer that has spread to other organs depends on where the cancer has spread. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about rectal cancer, see: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of rectal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Rectal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/colorectal/patient/rectal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389378] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Rectal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Rectal Cancer ?

Tests that examine the rectum and colon are used to detect (find) and diagnose rectal cancer. Tests used to diagnose rectal cancer include the following: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Digital rectal exam (DRE): An exam of the rectum. The doctor or nurse inserts a lubricated, gloved finger into the lower part of the rectum to feel for lumps or anything else that seems unusual. In women, the vagina may also be examined. - Colonoscopy : A procedure to look inside the rectum and colon for polyps (small pieces of bulging tissue), abnormal areas, or cancer. A colonoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove polyps or tissue samples, which are checked under a microscope for signs of cancer. - Biopsy : The removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. Tumor tissue that is removed during the biopsy may be checked to see if the patient is likely to have the gene mutation that causes HNPCC. This may help to plan treatment. The following tests may be used: - Reverse transcriptionpolymerase chain reaction (RTPCR) test: A laboratory test in which cells in a sample of tissue are studied using chemicals to look for certain changes in the structure or function of genes. - Immunohistochemistry : A test that uses antibodies to check for certain antigens in a sample of tissue. The antibody is usually linked to a radioactive substance or a dye that causes the tissue to light up under a microscope. This type of test may be used to tell the difference between different types of cancer. - Carcinoembryonic antigen (CEA) assay : A test that measures the level of CEA in the blood. CEA is released into the bloodstream from both cancer cells and normal cells. When found in higher than normal amounts, it can be a sign of rectal cancer or other conditions.

The rectum is part of the body’s digestive system. The digestive system takes in nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The digestive system is made up of the esophagus, stomach, and the small and large intestines. The colon (large bowel) is the first part of the large intestine and is about 5 feet long. Together, the rectum and anal canal make up the last part of the large intestine and are 6 to 8 inches long. The anal canal ends at the anus (the opening of the large intestine to the outside of the body).EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. For more information about rectal cancer, see: Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk to your doctor if you think you may be at risk for colorectal cancer. Risk factors for colorectal cancer include the following: Older age is a main risk factor for most cancers. The chance of getting cancer increases as you get older. These and other signs and symptoms may be caused by rectal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out whether cancer has spread within the rectum or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if rectal cancer spreads to the lung, the cancer cells in the lung are actually rectal cancer cells. The disease is metastatic rectal cancer, not lung cancer. In stage 0 rectal cancer, abnormal cells are found in the mucosa (innermost layer) of the rectum wall. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. In stage I rectal cancer, cancer has formed in the mucosa (innermost layer) of the rectum wall and has spread to the submucosa (layer of tissue next to the mucosa) or to the muscle layer of the rectum wall. Stage II rectal cancer is divided into stages IIA, IIB, and IIC. Stage III rectal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IIIA, cancer has spread: In stage IIIB, cancer has spread: In stage IIIC, cancer has spread: Stage IV rectal cancer is divided into stages IVA, IVB, and IVC. The cancer may come back in the rectum or in other parts of the body, such as the colon, pelvis, liver, or lungs. Different types of treatment are available for patients with rectal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for all stages of rectal cancer. The cancer is removed using one of the following types of surgery: After the cancer is removed, the surgeon will either: Radiation therapy and/or chemotherapy may be given before surgery to shrink the tumor, make it easier to remove the cancer, and help with bowel control after surgery. Treatment given before surgery is called neoadjuvant therapy. After all the cancer that can be seen at the time of the surgery is removed, some patients may be given radiation therapy and/or chemotherapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Short-course preoperative radiation therapy is used in some types of rectal cancer. This treatment uses fewer and lower doses of radiation than standard treatment, followed by surgery several days after the last dose. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly in the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Chemoembolization of the hepatic artery is a type of regional chemotherapy that may be used to treat cancer that has spread to the liver. This is done by blocking the hepatic artery (the main artery that supplies blood to the liver) and injecting anticancer drugs between the blockage and the liver. The liver’s arteries then carry the drugs into the liver. Only a small amount of the drug reaches other parts of the body. The blockage may be temporary or permanent, depending on what is used to block the artery. The liver continues to receive some blood from the hepatic portal vein, which carries blood from the stomach and intestine. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Colon and Rectal Cancer. Active surveillance is closely following a patient's condition without giving any treatment unless there are changes in test results. It is used to find early signs that the condition is getting worse. In active surveillance, patients are given certain exams and tests to check if the cancer is growing. When the cancer begins to grow, treatment is given to cure the cancer. Tests include the following: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapies used in the treatment of rectal cancer include the following: There are different types of monoclonal antibody therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy: Immune checkpoint inhibitors block proteins called checkpoints that are made by some types of immune system cells, such as T cells, and some cancer cells. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. They are used to treat some patients with metastatic colorectal cancer. There are two types of immune checkpoint inhibitor therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. After treatment for rectal cancer, a blood test to measure amounts of carcinoembryonic antigen (a substance in the blood that may be increased when cancer is present) may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II and stage III rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV and recurrent rectal cancer may include the following: Treatment of rectal cancer that has spread to other organs depends on where the cancer has spread. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about rectal cancer, see: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of rectal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Rectal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/colorectal/patient/rectal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389378] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Rectal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Rectal Cancer ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options depend on the following: - The stage of the cancer (whether it affects the inner lining of the rectum only, involves the whole rectum, or has spread to lymph nodes, nearby organs, or other places in the body). - Whether the tumor has spread into or through the bowel wall. - Where the cancer is found in the rectum. - Whether the bowel is blocked or has a hole in it. - Whether all of the tumor can be removed by surgery. - The patients general health. - Whether the cancer has just been diagnosed or has recurred (come back).

The rectum is part of the body’s digestive system. The digestive system takes in nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The digestive system is made up of the esophagus, stomach, and the small and large intestines. The colon (large bowel) is the first part of the large intestine and is about 5 feet long. Together, the rectum and anal canal make up the last part of the large intestine and are 6 to 8 inches long. The anal canal ends at the anus (the opening of the large intestine to the outside of the body).EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. For more information about rectal cancer, see: Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk to your doctor if you think you may be at risk for colorectal cancer. Risk factors for colorectal cancer include the following: Older age is a main risk factor for most cancers. The chance of getting cancer increases as you get older. These and other signs and symptoms may be caused by rectal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out whether cancer has spread within the rectum or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if rectal cancer spreads to the lung, the cancer cells in the lung are actually rectal cancer cells. The disease is metastatic rectal cancer, not lung cancer. In stage 0 rectal cancer, abnormal cells are found in the mucosa (innermost layer) of the rectum wall. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. In stage I rectal cancer, cancer has formed in the mucosa (innermost layer) of the rectum wall and has spread to the submucosa (layer of tissue next to the mucosa) or to the muscle layer of the rectum wall. Stage II rectal cancer is divided into stages IIA, IIB, and IIC. Stage III rectal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IIIA, cancer has spread: In stage IIIB, cancer has spread: In stage IIIC, cancer has spread: Stage IV rectal cancer is divided into stages IVA, IVB, and IVC. The cancer may come back in the rectum or in other parts of the body, such as the colon, pelvis, liver, or lungs. Different types of treatment are available for patients with rectal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for all stages of rectal cancer. The cancer is removed using one of the following types of surgery: After the cancer is removed, the surgeon will either: Radiation therapy and/or chemotherapy may be given before surgery to shrink the tumor, make it easier to remove the cancer, and help with bowel control after surgery. Treatment given before surgery is called neoadjuvant therapy. After all the cancer that can be seen at the time of the surgery is removed, some patients may be given radiation therapy and/or chemotherapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Short-course preoperative radiation therapy is used in some types of rectal cancer. This treatment uses fewer and lower doses of radiation than standard treatment, followed by surgery several days after the last dose. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly in the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Chemoembolization of the hepatic artery is a type of regional chemotherapy that may be used to treat cancer that has spread to the liver. This is done by blocking the hepatic artery (the main artery that supplies blood to the liver) and injecting anticancer drugs between the blockage and the liver. The liver’s arteries then carry the drugs into the liver. Only a small amount of the drug reaches other parts of the body. The blockage may be temporary or permanent, depending on what is used to block the artery. The liver continues to receive some blood from the hepatic portal vein, which carries blood from the stomach and intestine. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Colon and Rectal Cancer. Active surveillance is closely following a patient's condition without giving any treatment unless there are changes in test results. It is used to find early signs that the condition is getting worse. In active surveillance, patients are given certain exams and tests to check if the cancer is growing. When the cancer begins to grow, treatment is given to cure the cancer. Tests include the following: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapies used in the treatment of rectal cancer include the following: There are different types of monoclonal antibody therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy: Immune checkpoint inhibitors block proteins called checkpoints that are made by some types of immune system cells, such as T cells, and some cancer cells. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. They are used to treat some patients with metastatic colorectal cancer. There are two types of immune checkpoint inhibitor therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. After treatment for rectal cancer, a blood test to measure amounts of carcinoembryonic antigen (a substance in the blood that may be increased when cancer is present) may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II and stage III rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV and recurrent rectal cancer may include the following: Treatment of rectal cancer that has spread to other organs depends on where the cancer has spread. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about rectal cancer, see: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of rectal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Rectal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/colorectal/patient/rectal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389378] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Rectal Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Rectal Cancer ?

Key Points - After rectal cancer has been diagnosed, tests are done to find out if cancer cells have spread within the rectum or to other parts of the body. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - The following stages are used for rectal cancer: - Stage 0 (Carcinoma in Situ) - Stage I - Stage II - Stage III - Stage IV After rectal cancer has been diagnosed, tests are done to find out if cancer cells have spread within the rectum or to other parts of the body. The process used to find out whether cancer has spread within the rectum or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: - Chest x-ray : An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. - Colonoscopy : A procedure to look inside the rectum and colon for polyps (small pieces of bulging tissue). abnormal areas, or cancer. A colonoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove polyps or tissue samples, which are checked under a microscope for signs of cancer. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the abdomen, pelvis, or chest, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). - PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. - Endorectal ultrasound : A procedure used to examine the rectum and nearby organs. An ultrasound transducer (probe) is inserted into the rectum and used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The doctor can identify tumors by looking at the sonogram. This procedure is also called transrectal ultrasound. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if rectal cancer spreads to the lung, the cancer cells in the lung are actually rectal cancer cells. The disease is metastatic rectal cancer, not lung cancer. The following stages are used for rectal cancer: Stage 0 (Carcinoma in Situ) In stage 0, abnormal cells are found in the mucosa (innermost layer) of the rectum wall. These abnormal cells may become cancer and spread. Stage 0 is also called carcinoma in situ. Stage I In stage I, cancer has formed in the mucosa (innermost layer) of the rectum wall and has spread to the submucosa (layer of tissue under the mucosa). Cancer may have spread to the muscle layer of the rectum wall. Stage II Stage II rectal cancer is divided into stage IIA, stage IIB, and stage IIC. - Stage IIA: Cancer has spread through the muscle layer of the rectum wall to the serosa (outermost layer) of the rectum wall. - Stage IIB: Cancer has spread through the serosa (outermost layer) of the rectum wall but has not spread to nearby organs. - Stage IIC: Cancer has spread through the serosa (outermost layer) of the rectum wall to nearby organs. Stage III Stage III rectal cancer is divided into stage IIIA, stage IIIB, and stage IIIC. In stage IIIA: - Cancer has spread through the mucosa (innermost layer) of the rectum wall to the submucosa (layer of tissue under the mucosa) and may have spread to the muscle layer of the rectum wall. Cancer has spread to at least one but not more than 3 nearby lymph nodes or cancer cells have formed in tissues near the lymph nodes; or - Cancer has spread through the mucosa (innermost layer) of the rectum wall to the submucosa (layer of tissue under the mucosa). Cancer has spread to at least 4 but not more than 6 nearby lymph nodes. In stage IIIB: - Cancer has spread through the muscle layer of the rectum wall to the serosa (outermost layer) of the rectum wall or has spread through the serosa but not to nearby organs. Cancer has spread to at least one but not more than 3 nearby lymph nodes or cancer cells have formed in tissues near the lymph nodes; or - Cancer has spread to the muscle layer of the rectum wall or to the serosa (outermost layer) of the rectum wall. Cancer has spread to at least 4 but not more than 6 nearby lymph nodes; or - Cancer has spread through the mucosa (innermost layer) of the rectum wall to the submucosa (layer of tissue under the mucosa) and may have spread to the muscle layer of the rectum wall. Cancer has spread to 7 or more nearby lymph nodes. In stage IIIC: - Cancer has spread through the serosa (outermost layer) of the rectum wall but has not spread to nearby organs. Cancer has spread to at least 4 but not more than 6 nearby lymph nodes; or - Cancer has spread through the muscle layer of the rectum wall to the serosa (outermost layer) of the rectum wall or has spread through the serosa but has not spread to nearby organs. Cancer has spread to 7 or more nearby lymph nodes; or - Cancer has spread through the serosa (outermost layer) of the rectum wall and has spread to nearby organs. Cancer has spread to one or more nearby lymph nodes or cancer cells have formed in tissues near the lymph nodes. Stage IV Stage IV rectal cancer is divided into stage IVA and stage IVB. - Stage IVA: Cancer may have spread through the rectum wall and may have spread to nearby organs or lymph nodes. Cancer has spread to one organ that is not near the rectum, such as the liver, lung, or ovary, or to a distant lymph node. - Stage IVB: Cancer may have spread through the rectum wall and may have spread to nearby organs or lymph nodes. Cancer has spread to more than one organ that is not near the rectum or into the lining of the abdominal wall.

The rectum is part of the body’s digestive system. The digestive system takes in nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The digestive system is made up of the esophagus, stomach, and the small and large intestines. The colon (large bowel) is the first part of the large intestine and is about 5 feet long. Together, the rectum and anal canal make up the last part of the large intestine and are 6 to 8 inches long. The anal canal ends at the anus (the opening of the large intestine to the outside of the body).EnlargeAnatomy of the lower gastrointestinal (digestive) system showing the colon, rectum, and anus. Other organs that make up the digestive system are also shown. For more information about rectal cancer, see: Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk to your doctor if you think you may be at risk for colorectal cancer. Risk factors for colorectal cancer include the following: Older age is a main risk factor for most cancers. The chance of getting cancer increases as you get older. These and other signs and symptoms may be caused by rectal cancer or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out whether cancer has spread within the rectum or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if rectal cancer spreads to the lung, the cancer cells in the lung are actually rectal cancer cells. The disease is metastatic rectal cancer, not lung cancer. In stage 0 rectal cancer, abnormal cells are found in the mucosa (innermost layer) of the rectum wall. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. In stage I rectal cancer, cancer has formed in the mucosa (innermost layer) of the rectum wall and has spread to the submucosa (layer of tissue next to the mucosa) or to the muscle layer of the rectum wall. Stage II rectal cancer is divided into stages IIA, IIB, and IIC. Stage III rectal cancer is divided into stages IIIA, IIIB, and IIIC. In stage IIIA, cancer has spread: In stage IIIB, cancer has spread: In stage IIIC, cancer has spread: Stage IV rectal cancer is divided into stages IVA, IVB, and IVC. The cancer may come back in the rectum or in other parts of the body, such as the colon, pelvis, liver, or lungs. Different types of treatment are available for patients with rectal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for all stages of rectal cancer. The cancer is removed using one of the following types of surgery: After the cancer is removed, the surgeon will either: Radiation therapy and/or chemotherapy may be given before surgery to shrink the tumor, make it easier to remove the cancer, and help with bowel control after surgery. Treatment given before surgery is called neoadjuvant therapy. After all the cancer that can be seen at the time of the surgery is removed, some patients may be given radiation therapy and/or chemotherapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Short-course preoperative radiation therapy is used in some types of rectal cancer. This treatment uses fewer and lower doses of radiation than standard treatment, followed by surgery several days after the last dose. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly in the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Chemoembolization of the hepatic artery is a type of regional chemotherapy that may be used to treat cancer that has spread to the liver. This is done by blocking the hepatic artery (the main artery that supplies blood to the liver) and injecting anticancer drugs between the blockage and the liver. The liver’s arteries then carry the drugs into the liver. Only a small amount of the drug reaches other parts of the body. The blockage may be temporary or permanent, depending on what is used to block the artery. The liver continues to receive some blood from the hepatic portal vein, which carries blood from the stomach and intestine. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Colon and Rectal Cancer. Active surveillance is closely following a patient's condition without giving any treatment unless there are changes in test results. It is used to find early signs that the condition is getting worse. In active surveillance, patients are given certain exams and tests to check if the cancer is growing. When the cancer begins to grow, treatment is given to cure the cancer. Tests include the following: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapies used in the treatment of rectal cancer include the following: There are different types of monoclonal antibody therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Immune checkpoint inhibitor therapy: Immune checkpoint inhibitors block proteins called checkpoints that are made by some types of immune system cells, such as T cells, and some cancer cells. These checkpoints help keep immune responses from being too strong and sometimes can keep T cells from killing cancer cells. When these checkpoints are blocked, T cells can kill cancer cells better. They are used to treat some patients with metastatic colorectal cancer. There are two types of immune checkpoint inhibitor therapy: For more information, see Drugs Approved for Colon and Rectal Cancer. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. After treatment for rectal cancer, a blood test to measure amounts of carcinoembryonic antigen (a substance in the blood that may be increased when cancer is present) may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage 0 may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II and stage III rectal cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage IV and recurrent rectal cancer may include the following: Treatment of rectal cancer that has spread to other organs depends on where the cancer has spread. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about rectal cancer, see: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of rectal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Rectal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/colorectal/patient/rectal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389378] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. 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What are the treatments for Rectal Cancer ?

Key Points - There are different types of treatment for patients with rectal cancer. - Five types of standard treatment are used: - Surgery - Radiation therapy - Chemotherapy - Active surveillance - Targeted therapy - Other types of treatment are being tested in clinical trials. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with rectal cancer. Different types of treatment are available for patients with rectal cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Five types of standard treatment are used: Surgery Surgery is the most common treatment for all stages of rectal cancer. The cancer is removed using one of the following types of surgery: - Polypectomy: If the cancer is found in a polyp (a small piece of bulging tissue), the polyp is often removed during a colonoscopy. - Local excision: If the cancer is found on the inside surface of the rectum and has not spread into the wall of the rectum, the cancer and a small amount of surrounding healthy tissue is removed. - Resection: If the cancer has spread into the wall of the rectum, the section of the rectum with cancer and nearby healthy tissue is removed. Sometimes the tissue between the rectum and the abdominal wall is also removed. The lymph nodes near the rectum are removed and checked under a microscope for signs of cancer. - Radiofrequency ablation: The use of a special probe with tiny electrodes that kill cancer cells. Sometimes the probe is inserted directly through the skin and only local anesthesia is needed. In other cases, the probe is inserted through an incision in the abdomen. This is done in the hospital with general anesthesia. - Cryosurgery: A treatment that uses an instrument to freeze and destroy abnormal tissue. This type of treatment is also called cryotherapy. - Pelvic exenteration: If the cancer has spread to other organs near the rectum, the lower colon, rectum, and bladder are removed. In women, the cervix, vagina, ovaries, and nearby lymph nodes may be removed. In men, the prostate may be removed. Artificial openings (stoma) are made for urine and stool to flow from the body to a collection bag. After the cancer is removed, the surgeon will either: - do an anastomosis (sew the healthy parts of the rectum together, sew the remaining rectum to the colon, or sew the colon to the anus); or - make a stoma (an opening) from the rectum to the outside of the body for waste to pass through. This procedure is done if the cancer is too close to the anus and is called a colostomy. A bag is placed around the stoma to collect the waste. Sometimes the colostomy is needed only until the rectum has healed, and then it can be reversed. If the entire rectum is removed, however, the colostomy may be permanent. Radiation therapy and/or chemotherapy may be given before surgery to shrink the tumor, make it easier to remove the cancer, and help with bowel control after surgery. Treatment given before surgery is called neoadjuvant therapy. Even if all the cancer that can be seen at the time of the operation is removed, some patients may be given radiation therapy and/or chemotherapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy is used to treat rectal cancer. Short-course preoperative radiation therapy is used in some types of rectal cancer. This treatment uses fewer and lower doses of radiation than standard treatment, followed by surgery several days after the last dose. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly in the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Chemoembolization of the hepatic artery is a type of regional chemotherapy that may be used to treat cancer that has spread to the liver. This is done by blocking the hepatic artery (the main artery that supplies blood to the liver) and injecting anticancer drugs between the blockage and the liver. The livers arteries then carry the drugs into the liver. Only a small amount of the drug reaches other parts of the body. The blockage may be temporary or permanent, depending on what is used to block the artery. The liver continues to receive some blood from the hepatic portal vein, which carries blood from the stomach and intestine. The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Rectal Cancer for more information. Active surveillance Active surveillance is closely following a patient&apos;s condition without giving any treatment unless there are changes in test results. It is used to find early signs that the condition is getting worse. In active surveillance, patients are given certain exams and tests to check if the cancer is growing. When the cancer begins to grow, treatment is given to cure the cancer. Tests include the following: - Digital rectal exam. - MRI. - Endoscopy. - Sigmoidoscopy. - CT scan. - Carcinoembryonic antigen (CEA) assay. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Types of targeted therapies used in the treatment of rectal cancer include the following: - Monoclonal antibodies: Monoclonal antibody therapy is a type of targeted therapy being used for the treatment of rectal cancer. Monoclonal antibody therapy uses antibodies made in the laboratory from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. - Bevacizumab and ramucirumab are types of monoclonal antibodies that bind to a protein called vascular endothelial growth factor (VEGF). This may prevent the growth of new blood vessels that tumors need to grow. - Cetuximab and panitumumab are types of monoclonal antibodies that bind to a protein called epidermal growth factor receptor (EGFR) on the surface of some types of cancer cells. This may stop cancer cells from growing and dividing. - Angiogenesis inhibitors: Angiogenesis inhibitors stop the growth of new blood vessels that tumors need to grow. - Ziv-aflibercept is a vascular endothelial growth factor trap that blocks an enzyme needed for the growth of new blood vessels in tumors. - Regorafenib is used to treat colorectal cancer that has spread to other parts of the body and has not gotten better with other treatment. It blocks the action of certain proteins, including vascular endothelial growth factor. This may help keep cancer cells from growing and may kill them. It may also prevent the growth of new blood vessels that tumors need to grow. See Drugs Approved for Rectal Cancer for more information. Other types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. After treatment for rectal cancer, a blood test to measure amounts of carcinoembryonic antigen (a substance in the blood that may be increased when cancer is present) may be done to see if the cancer has come back. Treatment Options by Stage Stage 0 (Carcinoma in Situ) Treatment of stage 0 may include the following: - Simple polypectomy. - Local excision. - Resection (when the tumor is too large to remove by local excision). Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage 0 rectal cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage I Rectal Cancer Treatment of stage I rectal cancer may include the following: - Local excision. - Resection. - Resection with radiation therapy and chemotherapy after surgery. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I rectal cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stages II and III Rectal Cancer Treatment of stage II and stage III rectal cancer may include the following: - Surgery. - Chemotherapy combined with radiation therapy, followed by surgery. - Short-course radiation therapy followed by surgery and chemotherapy. - Resection followed by chemotherapy combined with radiation therapy. - Chemotherapy combined with radiation therapy, followed by active surveillance. Surgery may be done if the cancer recurs (comes back). - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage II rectal cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IV and Recurrent Rectal Cancer Treatment of stage IV and recurrent rectal cancer may include the following: - Surgery with or without chemotherapy or radiation therapy. - Systemic chemotherapy with or without targeted therapy (a monoclonal antibody or angiogenesis inhibitor). - Chemotherapy to control the growth of the tumor. - Radiation therapy, chemotherapy, or a combination of both, as palliative therapy to relieve symptoms and improve the quality of life. - Placement of a stent to help keep the rectum open if it is partly blocked by the tumor, as palliative therapy to relieve symptoms and improve the quality of life. - A clinical trial of a new anticancer drug. Treatment of rectal cancer that has spread to other organs depends on where the cancer has spread. - Treatment for areas of cancer that have spread to the liver includes the following: - Surgery to remove the tumor. Chemotherapy may be given before surgery, to shrink the tumor. - Cryosurgery or radiofrequency ablation. - Chemoembolization and/or systemic chemotherapy. - A clinical trial of chemoembolization combined with radiation therapy to the tumors in the liver. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage IV rectal cancer and recurrent rectal cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.