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Home > Health Library > Childhood Acute Lymphoblastic Leukemia Treatment (PDQ®): Treatment - Patient Information [NCI]
This information is produced and provided by the National Cancer Institute (NCI). The information in this topic may have changed since it was written. For the most current information, contact the National Cancer Institute via the Internet web site at http://cancer.gov or call 1-800-4-CANCER.
Childhood acute lymphoblastic leukemia (ALL) is a type of cancer in which the bone marrow makes too many immature lymphocytes (a type of white blood cell).
Childhood acute lymphoblastic leukemia (also called ALL or acute lymphocytic leukemia) is a cancer of the blood and bone marrow. This type of cancer usually gets worse quickly if it is not treated.
Anatomy of the bone. The bone is made up of compact bone, spongy bone, and bone marrow. Compact bone makes up the outer layer of the bone. Spongy bone is found mostly at the ends of bones and contains red marrow. Bone marrow is found in the center of most bones and has many blood vessels. There are two types of bone marrow: red and yellow. Red marrow contains blood stem cells that can become red blood cells, white blood cells, or platelets. Yellow marrow is made mostly of fat.
ALL is the most common type of cancer in children.
Leukemia may affect red blood cells, white blood cells, and platelets.
In a healthy child, 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 myeloid stem cell becomes one of three types of mature blood cells:
A lymphoid stem cell becomes a lymphoblast cell and then one of three types of lymphocytes (white blood cells):
Blood cell development. A blood stem cell goes through several steps to become a red blood cell, platelet, or white blood cell.
In a child with ALL, too many stem cells become lymphoblasts, B lymphocytes, or T lymphocytes. The cells do not work like normal lymphocytes and are not able to fight infection very well. These cells are cancer (leukemia) cells. Also, as the number of leukemia cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may lead to infection, anemia, and easy bleeding.
This summary is about acute lymphoblastic leukemia in children, adolescents, and young adults. See the following PDQ summaries for information about other types of leukemia:
Past treatment for cancer and certain genetic conditions affect the risk of having childhood ALL.
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.
Possible risk factors for ALL include the following:
Signs of childhood ALL include fever and bruising.
These and other signs and symptoms may be caused by childhood ALL or by other conditions. Check with your child's doctor if your child has any of the following:
Tests that examine the blood and bone marrow are used to detect (find) and diagnose childhood ALL.
The following tests and procedures may be used to diagnose childhood ALL and find out if leukemia cells have spread to other parts of the body such as the brain or testicles:
The following tests are done on blood or the bone marrow tissue that is removed:
This procedure is done after leukemia is diagnosed to find out if leukemia cells have spread to the brain and spinal cord. Intrathecal chemotherapy is given after the sample of fluid is removed to treat any leukemia cells that may have spread to the brain and spinal cord.
Certain factors affect prognosis (chance of recovery) and treatment options.
The prognosis (chance of recovery) depends on:
Treatment options depend on:
For leukemia that relapses (comes back) after treatment, the prognosis and treatment options depend partly on the following:
In childhood ALL, risk groups are used to plan treatment.
There are three risk groups in childhood ALL. They are described as:
Other factors that affect the risk group include the following:
It is important to know the risk group in order to plan treatment. Children with high-risk or very high–risk ALL usually receive more anticancer drugs and/or higher doses of anticancer drugs than children with standard-risk ALL.
Relapsed childhood ALL is cancer that has come back after it has been treated.
The leukemia may come back in the blood and bone marrow, brain, spinal cord, testicles, or other parts of the body.
Refractory childhood ALL is cancer that does not respond to treatment.
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:
Treatment for childhood acute lymphoblastic leukemia may cause side effects.
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 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:
Four types of standard treatment are used:
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 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 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
Chemotherapy and sometimes total-body irradiation 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.
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).
Stem cell transplant. (Step 1): Blood is taken from a vein in the arm of 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.
Targeted therapy is a treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. There are different types of targeted therapy:
New kinds of targeted therapies are also being studied in the treatment of childhood ALL.
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.
Intrathecal 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.
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.
CAR T-cell therapy. A type of treatment in which a patient's T cells (a type of immune cell) are changed in the laboratory so they will bind to cancer cells and kill them. Blood from a vein in the patient's arm flows through a tube to an apheresis machine (not shown), which removes the white blood cells, including the T cells, and sends the rest of the blood back to the patient. Then, the gene for a special receptor called a chimeric antigen receptor (CAR) is inserted into the T cells in the laboratory. Millions of the CAR T cells are grown in the laboratory and then given to the patient by infusion. The CAR T cells are able to bind to an antigen on the cancer cells and kill them.
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. 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.
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.
For information about the treatments listed below, see the Treatment Option Overview section.
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.
Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available.
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.
Newly Diagnosed Childhood Acute Lymphoblastic Leukemia (Very High Risk)
The treatment of very high–risk childhood acute lymphoblastic leukemia (ALL) during the remission induction, consolidation /intensification, and maintenance phases always includes combination chemotherapy. Children in the very high–risk 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.
Treatments being studied in clinical trials for very high–risk ALL include new chemotherapy regimens with or without targeted therapy.
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 chemotherapy for infants with a certain gene change.
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.
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 chromosome–positive ALL
The treatment of Philadelphia chromosome –positive childhood ALL during the remission induction, consolidation /intensification, and maintenance phases may include the following:
Treatments being studied in clinical trials for Philadelphia chromosome–positive childhood ALL include a new regimen of targeted therapy (imatinib mesylate) and combination chemotherapy with or without a stem cell transplant.
Refractory Childhood Acute Lymphoblastic Leukemia
There is no standard treatment for the treatment of refractory childhood acute lymphoblastic leukemia (ALL).
The treatment of refractory childhood ALL may include the following:
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:
Standard treatment of relapsed childhood acute lymphoblastic leukemia (ALL) that comes back outside the bone marrow may include the following:
Some of the treatments being studied in clinical trials for relapsed childhood ALL include:
For more information from the National Cancer Institute about childhood acute lymphoblastic leukemia, 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.
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Purpose of This Summary
This PDQ cancer information summary has current information about the treatment of childhood acute lymphoblastic 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.
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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.
Clinical Trial Information
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).
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PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Acute Lymphoblastic Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/child-all-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389385]
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Last Revised: 2019-07-23
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