Question: What are the treatments for Childhood Acute Lymphoblastic Leukemia ?
Answer: Key Points - There are different types of treatment for childhood acute lymphoblastic leukemia (ALL). - Children with ALL should have their treatment planned by a team of doctors who are experts in treating childhood leukemia. - Children and adolescents may have treatment-related side effects that appear months or years after treatment for acute lymphoblastic leukemia. - The treatment of childhood ALL usually has three phases. - Four types of standard treatment are used: - Chemotherapy - Radiation therapy - Chemotherapy with stem cell transplant - Targeted therapy - Treatment is given to kill leukemia cells that have spread or may spread to the brain, spinal cord, or testicles. - New types of treatment are being tested in clinical trials. - Chimeric antigen receptor (CAR) T-cell 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 childhood acute lymphoblastic leukemia (ALL). Different types of treatment are available for children with acute lymphoblastic leukemia (ALL). 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 ALL should have their treatment planned by a team of doctors who are experts in treating childhood leukemia. 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 professionals 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. - Pathologist. - Radiologist. - Pediatric nurse specialist. - Social worker. - Rehabilitation specialist. - Psychologist. - Child-life specialist. Children and adolescents may have treatment-related side effects that appear months or years after treatment for acute lymphoblastic leukemia. Regular follow-up exams are very important. Treatment can cause side effects long after it has ended. These are called late effects. Late effects of cancer treatment may include: - Physical problems, including problems with the heart, blood vessels, liver, or bones, and fertility. When dexrazoxane is given with chemotherapy drugs called anthracyclines, the risk of late heart effects is lessened. - Changes in mood, feelings, thinking, learning, or memory. Children younger than 4 years who have received radiation therapy to the brain have a higher risk of these effects. - Second cancers (new types of cancer) or other conditions, such as brain tumors, thyroid cancer, acute myeloid leukemia, and myelodysplastic syndrome. 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. The treatment of childhood ALL usually has three phases. The treatment of childhood ALL is done in phases: - Remission induction: 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: This is the second phase of treatment. It begins once the leukemia is in remission. The goal of consolidation/intensification therapy is to kill any leukemia cells that remain in the body and may cause a relapse. - Maintenance: This is the third phase of treatment. The goal 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. Not taking medication as ordered by the doctor during maintenance therapy increases the chance the cancer will come back. This is also called the continuation therapy phase. Four 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 into the cerebrospinal fluid (intrathecal), 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 child's risk group. Children with high-risk ALL receive more anticancer drugs and higher doses of anticancer drugs than children with standard-risk ALL. Intrathecal chemotherapy may be used to treat childhood ALL that has spread, or may spread, to the brain and spinal cord. See Drugs Approved for Acute Lymphoblastic 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 cancer being treated. External radiation therapy may be used to treat childhood ALL that has spread, or may spread, to the brain, spinal cord, or testicles. It may also be used to prepare the bone marrow for a stem cell transplant. Chemotherapy with stem cell transplant Stem cell transplant is a method of giving high doses of chemotherapy and sometimes total-body irradiation, and then replacing the blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of a donor. After the patient receives treatment, the donor's stem cells are given to the patient through an infusion. These reinfused stem cells grow into (and restore) the patient's blood cells. The stem cell donor doesn't have to be related to the patient. Stem cell transplant is rarely used as initial treatment for children and adolescents with ALL. It is used more often as part of treatment for ALL that relapses (comes back after treatment). See Drugs Approved for Acute Lymphoblastic Leukemia for more information. Targeted therapy Targeted therapy is a treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Tyrosine kinase inhibitors (TKIs) are targeted therapy drugs that block the enzyme, tyrosine kinase, which causes stem cells to become more white blood cells or blasts than the body needs. Imatinib mesylate is a TKI used in the treatment of children with Philadelphia chromosome positive ALL. Dasatinib and ruxolitinib are TKIs that are being studied in the treatment of newly diagnosed high-risk ALL. 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. Blinatumomab and inotuzumab are monoclonal antibodies being studied in the treatment of refractory childhood ALL. New kinds of targeted therapies are also being studied in the treatment of childhood ALL. See Drugs Approved for Acute Lymphoblastic Leukemia for more information. Treatment is given to kill leukemia cells that have spread or may spread to the brain, spinal cord, or testicles. Treatment to kill leukemia cells or prevent the spread of leukemia cells to the brain and spinal cord (central nervous system; CNS) is called CNS-directed therapy. Chemotherapy may be used to treat leukemia cells that have spread, or may spread, to the brain and spinal cord. Because standard doses of chemotherapy may not reach leukemia cells in the CNS, the cells are able to hide in the CNS. Systemic chemotherapy given in high doses or intrathecal chemotherapy (into the cerebrospinal fluid) is able to reach leukemia cells in the CNS. Sometimes external radiation therapy to the brain is also given. These treatments are given in addition to treatment that is used to kill leukemia cells in the rest of the body. All children with ALL receive CNS-directed therapy as part of induction therapy and consolidation/intensification therapy and sometimes during maintenance therapy. If the leukemia cells spread to the testicles, treatment includes high doses of systemic chemotherapy and sometimes radiation therapy. 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. Chimeric antigen receptor (CAR) T-cell therapy CAR T-cell therapy is a type of immunotherapy that changes the patient's T cells (a type of immune system cell) so they will attack certain proteins on the surface of cancer cells. T cells are taken from the patient and special receptors are added to their surface in the laboratory. The changed cells are called chimeric antigen receptor (CAR) T cells. The CAR T cells are grown in the laboratory and given to the patient by infusion. The CAR T cells multiply in the patient's blood and attack cancer cells. CAR T-cell therapy is being studied in the treatment of childhood ALL that has relapsed (come back) a second time. 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. Bone marrow aspiration and biopsy is done during all phases of treatment to see how well the treatment is working. Treatment Options for Childhood Acute Lymphoblastic Leukemia Newly Diagnosed Childhood Acute Lymphoblastic Leukemia (Standard Risk) The treatment of standard-risk childhood acute lymphoblastic leukemia (ALL) during the remission induction, consolidation /intensification, and maintenance phases always includes combination chemotherapy. When children are in remission after remission induction therapy, a stem cell transplant using stem cells from a donor may be done. When children are not in remission after remission induction therapy, further treatment is usually the same treatment given to children with high-risk ALL. Intrathecal chemotherapy is given to prevent the spread of leukemia cells to the brain and spinal cord. Treatments being studied in clinical trials for standard-risk ALL include new chemotherapy regimens. Check the list of NCI-supported cancer clinical trials that are now accepting patients with untreated childhood acute lymphoblastic 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. Newly Diagnosed Childhood Acute Lymphoblastic Leukemia (High Risk) The treatment of high-risk childhood acute lymphoblastic leukemia (ALL) during the remission induction, consolidation /intensification, and maintenance phases always includes combination chemotherapy. Children in the high-risk ALL group are given more anticancer drugs and higher doses of anticancer drugs, especially during the consolidation/intensification phase, than children in the standard-risk group. Intrathecal and systemic chemotherapy are given to prevent or treat the spread of leukemia cells to the brain and spinal cord. Sometimes radiation therapy to the brain is also given. Treatments being studied in clinical trials for high-risk ALL include new chemotherapy regimens with or without targeted therapy or stem cell transplant. Check the list of NCI-supported cancer clinical trials that are now accepting patients with untreated childhood acute lymphoblastic 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. Newly Diagnosed Childhood Acute Lymphoblastic Leukemia (Very High Risk) The treatment of very highrisk childhood acute lymphoblastic leukemia (ALL) during the remission induction, consolidation /intensification, and maintenance phases always includes combination chemotherapy. Children in the very highrisk ALL group are given more anticancer drugs than children in the high-risk group. It is not clear whether a stem cell transplant during first remission will help the child live longer. Intrathecal and systemic chemotherapy are given to prevent or treat the spread of leukemia cells to the brain and spinal cord. Sometimes radiation therapy to the brain is also given. Treatments being studied in clinical trials for very highrisk ALL include new chemotherapy regimens with or without targeted therapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with untreated childhood acute lymphoblastic 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. Newly Diagnosed Childhood Acute Lymphoblastic Leukemia (Special Groups) T-cell childhood acute lymphoblastic leukemia The treatment of T-cell childhood acute lymphoblastic leukemia (ALL) during the remission induction, consolidation /intensification, and maintenance phases always includes combination chemotherapy. Children with T-cell ALL are given more anticancer drugs and higher doses of anticancer drugs than children in the newly diagnosed standard-risk group. Intrathecal and systemic chemotherapy are given to prevent the spread of leukemia cells to the brain and spinal cord. Sometimes radiation therapy to the brain is also given. Treatments being studied in clinical trials for T-cell ALL include new anticancer agents and chemotherapy regimens with or without targeted therapy. Infants with ALL The treatment of infants with ALL during the remission induction, consolidation /intensification, and maintenance phases always includes combination chemotherapy. Infants with ALL are given different anticancer drugs and higher doses of anticancer drugs than children 1 year and older in the standard-risk group. It is not clear whether a stem cell transplant during first remission will help the child live longer. Intrathecal and systemic chemotherapy are given to prevent the spread of leukemia cells to the brain and spinal cord. Treatments being studied in clinical trials for infants with ALL include the following: - A clinical trial of chemotherapy followed by a donor stem cell transplant for infants with certain gene changes. Children 10 years and older and adolescents with ALL The treatment of ALL in children and adolescents (10 years and older) during the remission induction, consolidation /intensification, and maintenance phases always includes combination chemotherapy. Children 10 years and older and adolescents with ALL are given more anticancer drugs and higher doses of anticancer drugs than children in the standard-risk group. Intrathecal and systemic chemotherapy are given to prevent the spread of leukemia cells to the brain and spinal cord. Sometimes radiation therapy to the brain is also given. Treatments being studied in clinical trials for children 10 years and older and adolescents with ALL include new anticancer agents and chemotherapy regimens with or without targeted therapy. Philadelphia chromosomepositive ALL The treatment of Philadelphia chromosome positive childhood ALL during the remission induction, consolidation /intensification, and maintenance phases may include the following: - Combination chemotherapy and targeted therapy with a tyrosine kinase inhibitor (imatinib mesylate) with or without a stem cell transplant using stem cells from a donor. Check the list of NCI-supported cancer clinical trials that are now accepting patients with T-cell childhood acute lymphoblastic leukemia and Philadelphia chromosome positive childhood precursor acute lymphoblastic 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. Refractory Childhood Acute Lymphoblastic Leukemia There is no standard treatment for the treatment of refractory childhood acute lymphoblastic leukemia (ALL). Some of the treatments being studied in clinical trials for refractory childhood ALL include: - Targeted therapy (blinatumomab or inotuzumab). - Chimeric antigen receptor (CAR) T-cell therapy. Relapsed Childhood Acute Lymphoblastic Leukemia Standard treatment of relapsed childhood acute lymphoblastic leukemia (ALL) that comes back in the bone marrow may include the following: - Combination chemotherapy. - Chemotherapy with or without total-body irradiation followed by a stem cell transplant, using stem cells from a donor. Standard treatment of relapsed childhood acute lymphoblastic leukemia (ALL) that comes back outside the bone marrow may include the following: - Systemic chemotherapy and intrathecal chemotherapy with radiation therapy to the brain and/or spinal cord for cancer that comes back in the brain and spinal cord only. - Combination chemotherapy and radiation therapy for cancer that comes back in the testicles only. - Stem cell transplant for cancer that has recurred in the brain and/or spinal cord. Some of the treatments being studied in clinical trials for relapsed childhood ALL include: - New anticancer drugs and new combination chemotherapy treatments. - Combination chemotherapy and new kinds of targeted therapies (blinatumomab or inotuzumab). - Chimeric antigen receptor (CAR) T-cell therapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with recurrent childhood acute lymphoblastic 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.