Acute Myeloid Leukemia & Similar Malignancies

General Information | Treatment Options | Resources

Treatment
  • Overview
  • Standard Treatment
  • Clinical Trials
  • Treatment By Stage

There are different types of treatment for children with acute myeloid leukemia (AML), chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), transient myeloproliferative disorder (TMD), or myelodysplastic syndromes.

Different types of treatment are available for children with AML, CML, JMML, TMD, or myelodysplastic syndromes. 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 AML, CML, JMML, TMD, or myelodysplastic syndromes should have their treatment planned by a team of doctors with expertise 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 pediatric doctors who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists:

  • Hematologist.
  • Medical oncologist.
  • Pediatric surgeon.
  • Radiation oncologist.
  • Neurologist.
  • Neuropathologist.
  • Neuroradiologist.
  • Pediatric nurse specialist.
  • Social worker.
  • Rehabilitation specialist.
  • Psychologist.

Regular follow-up exams are very important. Some cancer treatments cause side effects that continue or appear years after cancer treatment has ended. These are called late effects. Late effects of cancer treatment may include the following:

  • Physical problems.
  • Changes in mood, feelings, thinking, learning, or memory.
  • Having a new type 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 possible late effects caused by some treatments. See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.

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. This is sometimes called re-staging.

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.

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. Its purpose 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 purpose of postremission 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 chemotherapy that is given by mouth or injected into a vein 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 and radiation therapy are able to reach and kill leukemia cells in the CNS and prevent the cancer from recurring (coming back). CNS sanctuary therapy is also called CNS prophylaxis.

Six types of standard treatment are used for childhood AML, childhood CML, JMML, TMD, or myelodysplastic syndromes.

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 spinal column (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. Anticancer drugs given by mouth or vein to treat AML cannot cross the blood-brain barrier and enter the fluid that surrounds the brain and spinal cord. Instead, an anticancer drug is injected into the fluid-filled space to kill leukemia cells that may have spread there. This is called intrathecal chemotherapy.

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. External radiation therapy may be used to treat childhood AML that has spread, or may spread, to the brain and spinal cord. When used this way, it is called central nervous system (CNS) sanctuary therapy or CNS prophylaxis.

Stem cell transplantation

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.

Other drug therapy

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 a subtype of AML called acute promyelocytic leukemia (APL).

Imatinib (Gleevec) is a type of anticancer drug called a tyrosine kinase inhibitor. It blocks the enzyme, tyrosine kinase, that causes stem cells to develop into more white blood cells (granulocytes or blasts) than the body needs.

Watchful waiting

Watchful waiting is closely monitoring a patient's condition without giving any treatment until symptoms appear or change. It is sometimes used to treat myelodysplastic syndromes 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 person earlier and stored until needed.
  • Drug therapy, such as antibiotics.
  • 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 Web site.

Biologic therapy

Biologic therapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This type of cancer treatment is also called biotherapy or immunotherapy.

Monoclonal antibody therapy is a certain type of biologic therapy. 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.

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 clinical trials database.

A link to a list of current clinical trials is included for each treatment section. For some types or stages of cancer, there may not be any trials listed. Check with your doctor for clinical trials that are not listed here but may be right for you.

Newly Diagnosed Childhood Acute Myeloid Leukemia

Treatment of newly diagnosed childhood acute myeloid leukemia (AML) is combination chemotherapy. CNS sanctuary therapy is intrathecal chemotherapy with or without radiation therapy to the brain.

Treatment of newly diagnosed childhood acute leukemia with a granulocytic sarcoma (chloroma) may include chemotherapy with or without radiation therapy.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with untreated childhood acute myeloid leukemia and other myeloid malignancies.

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.
  • Stem cell transplant.
  • A clinical trial of chemotherapy with or without a monoclonal antibody.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with childhood acute myeloid leukemia in remission.

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 clinical trial of a new anticancer drug.
  • A clinical trial of a new monoclonal antibody.
  • A clinical trial of stem cell transplant using different sources of stem cells.

Treatment of recurrent acute promyelocytic leukemia may include all-trans retinoic acid (ATRA) or arsenic trioxide therapy

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with recurrent childhood acute myeloid leukemia.

Acute Promyelocytic Leukemia

Treatment of acute promyelocytic leukemia may include the following:

  • All-trans retinoic acid (ATRA) plus chemotherapy.
  • Arsenic trioxide therapy.
  • Combination chemotherapy.
  • A clinical trial of chemotherapy and ATRA with or without arsenic trioxide.
  • A clinical trial of a monoclonal antibody with ATRA.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with childhood acute promyelocytic leukemia (M3).

Children with Down Syndrome and AML

Treatment of AML in children who have Down syndrome may include the following:

  • Combination chemotherapy.
  • A clinical trial of lower-dose chemotherapy.

Childhood Chronic Myelogenous Leukemia

Treatment for childhood chronic myelogenous leukemia may include the following:

  • Stem cell transplant.
  • Drug therapy with Gleevec.
  • A clinical trial of Gleevec therapy with interferon or chemotherapy.
  • A clinical trial of stem cell transplant using lower doses of chemotherapy.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with childhood chronic myelogenous leukemia.

Juvenile Myelomonocytic Leukemia

Treatment of juvenile myelomonocytic leukemia may include the following:

  • Stem cell transplant.
  • A clinical trial of new combinations of chemotherapy and stem cell transplant.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with juvenile myelomonocytic leukemia.

Transient Myeloproliferative Disorder

Transient myeloproliferative disorder (TMD) 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 for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with acute myeloid leukemia/transient myeloproliferative disorder.

Myelodysplastic Syndromes

Treatment of myelodysplastic syndromes may include the following:

  • Stem cell transplant.
  • Combination chemotherapy.
  • A clinical trial of a new anticancer drug.

Supportive care treatments are used to manage problems caused by the disease, such as infection, bleeding, and anemia.

If the myelodysplastic syndrome progresses to acute myeloid leukemia (AML), treatment will be the same as treatment for the newly diagnosed patient with AML.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with childhood myelodysplastic syndromes.

Cancer information from the NCI PDQ service