In Treatment

Chemotherapy is the primary treatment for AML. It is known that few children, if any, will be cured with just one course of chemotherapy, even if no leukemia is found in the bone marrow (also called remission) after the first course. Multiple chemotherapy treatments are required for a chance at long-term cure. The length of chemotherapy treatment is usually 4-5 months. Some patients may receive a bone marrow transplant following initial chemotherapy.

The drugs selected for treatment are based on the specific cancer diagnosis. Chemotherapy for AML may be given by mouth or injected into a vein, muscle or the central nervous system. Chemotherapy for AML is usually divided into phases of treatment.

Induction: The first part of treatment. The goal of this phase is to kill the leukemia cells and allow normal blood cells to return. As the “targets” are the abnormal mutated white blood cells, the chemotherapy usually reduces all of the white blood cells (good and mutated) to zero, and it takes several weeks for healthy bone marrow to regrow. Infection is a major risk during this period, and patients remain in the hospital until there is evidence of a regrowth of healthy white blood cells. A bone marrow aspirate is performed at the end of this phase. The bone marrow is looked at under a microscope, and is expected to show only normal cells. This is called remission. This does not mean that the child is cured, because without further treatment the disease will return. This phase of treatment usually lasts about 4 weeks, but may be longer depending upon the length of recovery of normal blood cells.

Consolidation: The second part of treatment to kill any leukemia cells remaining in the body. This phase usually lasts about 4-6 weeks. Again, infection is a major risk and patients may need to be hospitalized until there are enough healthy white blood cells to fight infection.

Intensification: Additional chemotherapy to kill remaining leukemia cells. This phase usually lasts about 8-12 weeks.

In most children, the signs and symptoms of the disease are absent after induction, and most children will be in remission after induction. Approximately 50-60% of children will be cured of AML with current modern therapy

Blood and Bone Marrow Transplant (BMT)

If there is a family member (brother, sister, parent) who is a tissue type match, a bone marrow transplant may be recommended in first remission unless there are low risk mutations found. The chances of staying in remission after a BMTare higher than with chemotherapy alone. Siblings of a patient (and sometimes parents) can have their blood tested to see if they are a tissue type match.

If a patient has not achieved remission with the initial chemotherapy, a blood or marrow transplant from an unrelated donor may be recommended. The risks of unrelated donor blood or marrow transplant may be higher than with a matched family member, so this procedure is considered in first remission only for patients at high risk for relapse.

With the following exceptions, patients all receive the same treatment.
  • Down Syndrome: children with Down syndrome generally require less intensive chemotherapy to achieve a cure.
  • Acute Promyelocytic Leukemia: these children receive different chemotherapy with the special medication, all-trans retinoic acid. These patients respond well to this unique therapy and generally do not receive a bone marrow transplant in first remission.
  • Specific AML Risk Groups including:
    • High Risk types of AML that do not respond well to standard chemotherapy. These patients often receive a blood or marrow transplant in first remission.
    • Low Risk types of AML that respond well to chemotherapy. Patients may not be recommended for bone marrow transplant in first remission, even if a family member is a tissue type match.
  • Central nervous system disease at diagnosis (blasts in the cerebrospinal fluid). Some children may have blast cells in the fluid collected at the first spinal tap. They receive extra chemotherapy given with spinal taps to kill these cells.


If leukemia returns (relapses) it usually happens within the first two years following diagnosis. Additional chemotherapy may be given to get the child back into remission. There may be new therapies in research studies that may be beneficial to AML patients; your doctor may discuss these with you if they are available. Upon achieving a new remission, a bone marrow transplant will be considered to maintain remission. If a related donor is not available, then an unrelated donor may be considered.

Causes of AML

We currently DO NOT know what causes AML. Only a few risk factors for AML are known for sure.

Genetic conditions
Children with some genetic syndromes are more likely than others to have their myeloid cells develop additional mutations that lead to AML. These are children with Down syndrome, Fanconi anemia, neurofibromatosis type 1, Bloom syndrome, Schwachman syndrome, familial monosomy 7 and Kostmann granulocytopenia. Children with these genetic conditions are more at risk for AML, but most children with AML do not have one of these conditions. These syndromes usually require medical care, so you would know if your child has one of them.

Ionizing radiation (x-rays)
Early studies showed there was an increased risk when the baby was x-rayed while in its mother's womb. Today, however, researchers believe that x-rays during pregnancy cause few if any cases of AML because pregnant women are not x-rayed often and the amount of radiation used in x-rays is much, much lower than it was years ago.

Children who receive x-rays to diagnose a medical condition, such as a broken bone or tooth decay, are NOT at increased risk - much higher, cumulative, doses of radiation are required to cause AML.

Chemotherapy for a previous cancer
Treatment with certain chemotherapy medications increases the risk of AML, although the overall risk is low.

Research Studies

The majority of children with cancer participate in research studies. This high rate of participation has been essential to improving the cure rates for children’s cancer.

Researchers design various studies to improve treatment and advance the understanding of cancer and its causes. Clinical trials are carefully reviewed and must be approved through a formal scientific process before anyone can be enrolled. If there is a research study “open” that your child is “eligible for,” you may be asked to allow your child to participate. It is also possible that your child will be asked to participate in more than one study.

Whether an individual is eligible for a particular study may depend on age, location of the cancer, the extent of the disease and other information. Researchers usually must limit their study to some of these characteristics to have a scientifically valid study. Further, researchers must follow exactly the same restrictions throughout the study.

If your child is eligible to participate in one or more study, your doctor will discuss these with you during an initial treatment conference (also called informed consent conference). The doctor will describe the study, potential risks of participation, and other information you need to decide whether or not you would like your child to participate in the study. You always have the choice to participate or not in research studies.

If you do choose to have your child participate in a study, you doctor will explain what type of information you will receive about the results of the study. The overall results of the research study will be published to inform the public and other researchers. No study will publish any information that identifies an individual.

Visit the Clinical Trials section of this website to learn more about the various kinds of research studies.

Last updated July, 2011

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