Childhood Acute Lymphoblastic Leukemia Treatment & Symptoms

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.

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.

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.

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.

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.

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 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 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 chromosome-positive 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.

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.