Question: What are the treatments for Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies ?
Answer: 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'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'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'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'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'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'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'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'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'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'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'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's doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website.