Question: What are the treatments for Langerhans Cell Histiocytosis ?
Answer: Key Points - There are different types of treatment for patients with Langerhans cell histiocytosis (LCH). - Children with LCH should have their treatment planned by a team of health care providers who are experts in treating childhood cancer. - Some cancer treatments cause side effects months or years after treatment for childhood cancer has ended. - Nine types of standard treatment are used: - Chemotherapy - Surgery - Radiation therapy - Photodynamic therapy - Biologic therapy - Targeted therapy - Other drug therapy - Stem cell transplant - 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 treatment. - When treatment of LCH stops, new lesions may appear or old lesions may come back. - Follow-up tests may be needed. There are different types of treatment for patients with Langerhans cell histiocytosis (LCH). Different types of treatments are available for patients with LCH. 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. Whenever possible, patients should take part in a clinical trial in order to receive new types of treatment for LCH. Some clinical trials are open only to patients who have not started 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 treatment is a decision that ideally involves the patient, family, and health care team. Children with LCH should have their treatment planned by a team of health care providers who are experts in treating childhood cancer. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric healthcare providers who are experts in treating children with LCH and who specialize in certain areas of medicine. These may include the following specialists: - Pediatrician. - Primary care physician. - Pediatric surgeon. - Pediatric hematologist. - Radiation oncologist. - Neurologist. - Endocrinologist. - Pediatric nurse specialist. - Rehabilitation specialist. - Psychologist. - Social worker. Some cancer treatments cause side effects months or years after treatment for childhood cancer has ended. Side effects from cancer treatment that begin during or after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: - Slow growth and development. - Hearing loss. - Bone, tooth, liver, and lung problems. - Changes in mood, feeling, learning, thinking, or memory. - Second cancers, such as leukemia, retinoblastoma, Ewing sarcoma, brain or liver cancer. 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. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.) Many patients with multisystem LCH have late effects caused by treatment or by the disease itself. These patients often have long-term health problems that affect their quality of life. Nine types of standard treatment are used: 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 onto the skin or 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). Chemotherapy agents given by injection or by mouth are used to treat LCH. Chemotherapy agents include vinblastine, cytarabine, cladribine, and methotrexate. Nitrogen mustard is a drug that is put directly on the skin to treat small LCH lesions. Surgery Surgery may be used to remove LCH lesions and a small amount of nearby healthy tissue. Curettage is a type of surgery that uses a curette (a sharp, spoon-shaped tool) to scrape LCH cells from bone. When there is severe liver or lung damage, the entire organ may be removed and replaced with a healthy liver or lung from a donor. 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. External radiation therapy uses a machine outside the body to send radiation toward the cancer. In LCH, a special lamp may be used to send ultraviolet B (UVB) radiation toward LCH skin lesions. 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 LCH, laser light is aimed at the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients who have photodynamic therapy should not spend too much time in the sun. In one type of photodynamic therapy, called psoralen and ultraviolet A (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet A radiation is directed to the skin. 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. Interferon is a type of biologic therapy used to treat LCH of the skin. Immunomodulators are also a type of biologic therapy. Thalidomide is an immunomodulator used to treat LCH. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to find and attack LCH cells without harming normal cells. Imatinib mesylate is a type of targeted therapy called a tyrosine kinase inhibitor. It stops blood stem cells from turning into dendritic cells that may become cancer cells. Other types of kinase inhibitors that affect cells with mutations (changes) in the BRAF gene, such as dabrafenib and vemurafenib, are being studied in clinical trials for LCH. A family of genes, called ras genes, may cause cancer when they are mutated. Ras genes make proteins that are involved in cell signaling pathways, cell growth, and cell death. Ras pathway inhibitors are a type of targeted therapy being studied in clinical trials. They block the actions of a mutated ras gene or its protein and may stop the growth of cancer. Other drug therapy Other drugs used to treat LCH include the following: - Steroid therapy, such as prednisone, is used to treat LCH lesions. - Bisphosphonate therapy (such as pamidronate, zoledronate, or alendronate) is used to treat LCH lesions of the bone and to lessen bone pain. - Nonsteroidal anti-inflammatory drugs (NSAIDs) are drugs (such as aspirin and ibuprofen) that are commonly used to decrease fever, swelling, pain, and redness. Sometimes an NSAID called indomethacin is used to treat LCH. - Retinoids, such as isotretinoin, are drugs related to vitamin A that can slow the growth of LCH cells in the skin. The retinoids are taken by mouth. Stem cell transplant Stem cell transplant is a method of giving chemotherapy and replacing blood-forming cells destroyed by the LCH 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. Observation Observation is closely monitoring a patient's 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 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 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 for Childhood LCH and the Treatment Options for Adult LCH sections for links to current treatment clinical trials. These have been retrieved from NCI's clinical trials database. When treatment of LCH stops, new lesions may appear or old lesions may come back. Many patients with LCH get better with treatment. However, when treatment stops, new lesions may appear or old lesions may come back. This is called reactivation (recurrence) and may occur within one year after stopping treatment. Patients with multisystem disease are more likely to have a reactivation. More common sites of reactivation are bone, ears, or skin. Diabetes insipidus also may develop. Less common sites of reactivation include lymph nodes, bone marrow, spleen, liver, or lung. Some patients may have more than one reactivation over a number of years. Follow-up tests may be needed. Some of the tests that were done to diagnose LCH may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include: - Physical exam. - Neurological exam. - Ultrasound exam. - MRI. - CT scan. - PET scan. Other tests that may be needed include: - Brain stem auditory evoked response (BAER) test: A test that measures the brain's response to clicking sounds or certain tones. - 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 a lung function test. - 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. 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 LCH in Children Treatment of Low-Risk Disease in Children Skin Lesions Treatment of childhood Langerhans cell histiocytosis (LCH) skin lesions may include the following: - Observation. When severe rashes, pain, ulceration, or bleeding occur, treatment may include the following: - Steroid therapy. - Chemotherapy, given by mouth. - Nitrogen mustard applied to the skin. - Photodynamic therapy with psoralen and ultraviolet A (PUVA) therapy. - UVB radiation therapy. Lesions in Bones or Other Low-Risk Organs Treatment of childhood LCH bone lesions in the front, sides, or back of the skull, or in any other single bone may include the following: - Surgery (curettage) with or without steroid therapy. - Low-dose radiation therapy for lesions that affect nearby organs. Treatment of childhood LCH lesions in bones around the ears or eyes is done to lower the risk of diabetes insipidus and other long-term problems. Treatment may include: - Chemotherapy and steroid therapy. - Surgery (curettage). Treatment of childhood LCH lesions of the spine or thigh bone lesions may include: - Observation. - Low-dose radiation therapy. - Chemotherapy, for lesions that spread from the spine into nearby tissue. - Surgery to strengthen the weakened bone by bracing or fusing the bones together. Treatment of two or more bone lesions may include: - Chemotherapy and steroid therapy. Treatment of two or more bone lesions combined with childhood LCH skin lesions, lymph node lesions, or diabetes insipidus may include: - Chemotherapy with or without steroid therapy. - Bisphosphonate therapy. Treatment of High-Risk Disease in Children Treatment of childhood LCH multisystem disease lesions in the spleen, liver, or bone marrow (with or without skin, bone, lymph node, lung, or pituitary gland lesions) may include: - Chemotherapy and steroid therapy. Higher doses of combination chemotherapy and steroid therapy may be given to patients whose tumors do not respond to initial chemotherapy. - A liver transplant for patients with severe liver damage. Treatment of childhood LCH central nervous system (CNS) lesions may include: - Chemotherapy with or without steroid therapy. - Steroid therapy. Treatment of LCH CNS neurodegenerative syndrome may include: - Retinoid therapy. - Chemotherapy. Treatment Options for Recurrent, Refractory, and Progressive Childhood LCH in Children Recurrent LCH is cancer that cannot be detected for some time after treatment and then comes back. Treatment of recurrent childhood LCH in the skin, bone, lymph nodes, gastrointestinal tract, pituitary gland, or central nervous system (low-risk organs) may include: - Chemotherapy with or without steroid therapy. - Bisphosphonate therapy. - Nonsteroidal anti-inflammatory drug (NSAID) therapy with indomethacin. - A clinical trial of a targeted therapy. Refractory LCH is cancer that does not get better with treatment. Treatment of refractory childhood LCH in high-risk organs and in multisystem low-risk organs may include high-dose chemotherapy. Treatment of childhood LCH in multisystem high-risk organs that did not respond to chemotherapy may include stem cell transplant. Progressive LCH is cancer that continues to grow during treatment. Treatment of progressive childhood LCH in patients with multisystem disease may include anticancer drugs that have not been given to the patient before. Treatment Options for LCH in Adults Treatment Options for LCH of the Lung in Adults Treatment for LCH of the lung in adults may include: - Quitting smoking for all patients who smoke. Lung damage will get worse over time in patients who do not quit smoking. In patients who quit smoking, lung damage may get better or it may get worse over time. - Chemotherapy. - Lung transplant for patients with severe lung damage. Sometimes LCH of the lung will go away or not get worse even if it's not treated. Treatment Options for LCH of the Bone in Adults Treatment for LCH that affects only the bone in adults may include: - Surgery with or without steroid therapy. - Chemotherapy with or without low-dose radiation therapy. - Radiation therapy. - Bisphosphonate therapy, for severe bone pain. Treatment Options for LCH of the Skin in Adults Treatment for LCH that affects only the skin in adults may include: - Surgery. - Steroid or other drug therapy applied or injected into the skin. - Photodynamic therapy with psoralen and ultraviolet A (PUVA) radiation. - UVB radiation therapy. - Chemotherapy or biologic therapy given by mouth, such as methotrexate, thalidomide, or interferon. - Retinoid therapy may be used if the skin lesions do not get better with other treatment. Treatment for LCH that affects the skin and other body systems in adults may include: - Chemotherapy. Treatment Options for Single-System and Multisystem LCH in Adults Treatment of single-system and multisystem disease in adults may include: - Chemotherapy with or without a drug given to weaken the immune system. - Bisphosphonate therapy, for severe bone pain. - A clinical trial of a targeted therapy.