Expand Content

Acute Myeloid Leukemia

Childhood acute myeloid leukemia (AML)

Childhood acute myeloid leukemia (AML) is a cancer of the blood-forming tissue, primarily the bone marrow. AML is also called acute nonlymphocytic leukemia or acute myelogenous leukemia. There are several subtypes of AML. It is less common than acute lymphocytic leukemia (also called acute lymphoblastic leukemia or ALL), another leukemia that occurs in children and adolescents. Children with Down Syndrome have an increased risk of developing acute myeloid leukemia during the first three years of life.

Blood cells are produced by the bone marrow. The bone marrow is the "spongy tissue" inside the large bones of the body. The bone marrow makes red blood cells (which carry oxygen and other materials to all tissues of the body), white blood cells (which fight infection), and platelets (which help the blood to clot).

The bone marrow continually produces new normal cells. In leukemia, the bone marrow starts producing large numbers of abnormal cells of one type, usually immature white cells. These abnormal, immature cells are called blasts. Blasts may flood the blood stream and lymph system, as well as invade vital organs such as the brain, testes, ovaries, or skin. Acute myeloid leukemia blast cells can rarely appear as a solid tumor, called an isolated granulocytic sarcoma or chloroma.

Leukemia in children can be acute (progressing quickly with many immature blasts) or chronic (progressing slowly with more mature-looking cancerous blast cells). Acute myeloid leukemia progresses quickly, and can occur in both children and adults. Children and adolescents are treated differently than adults.

Early signs and/or symptoms of acute myeloid leukemia may include fever, feeling weak or tired, aching joints or bones (the child may limp), chills, bleeding, easy bruising or swollen lymph nodes. If your child has any of these symptoms, your child's doctor will order blood tests to evaluate the different kinds of blood cells. If the results of the blood tests are not normal, your child may be referred to a pediatric oncologist who will perform a bone marrow biopsy and aspiration. During this test, a needle is inserted into a bone in the hip and a small amount of bone marrow is removed and examined under a microscope, enabling the doctor to determine if your child has leukemia and what kind of leukemia your child may have. Chromosomal analysis on the bone marrow cells will be performed. The best treatment plan for your child will then be determined.

Stages of childhood acute myeloid leukemia

There is no staging for acute myeloid leukemia in children. Acute myeloid leukemia is spread throughout the bloodstream at the time of diagnosis. It may also be within other body tissues.

In remission

Remission means that curative treatment has been given and that the number of white blood cells and other cells in the blood and bone marrow are approaching normal. There are no obvious signs or symptoms of leukemia. The bone marrow report will say "less than 5% blasts."


Recurrent means that the leukemia has come back (recurred) after going into remission. Refractory means that the leukemia failed to go into remission following treatment designed to induce remission.

How childhood acute myeloid leukemia (AML) is treated

There are treatments for all subtypes of childhood acute myeloid leukemia in children. This treatment should be decided upon and administered by a multidisciplinary team of cancer specialists, headed by a pediatric oncologist who is trained and experienced in the treatment of childhood and adolescent cancers. The primary treatment for acute myeloid leukemia is chemotherapy, sometimes followed by bone marrow transplantation. Radiation therapy may be used in certain cases. Biological therapy is being studied in clinical trials.

Chemotherapy is the use of drugs to kill cancer cells. Chemotherapy drugs may be taken by mouth or injected into a vein (intravenous injection ) or a muscle (intramuscular injection). Chemotherapy is called a systemic treatment because the drug enters the bloodstream, travels through the body, to kill cancer cells. Chemotherapy will also be injected into the fluid that surrounds the brain and spinal cord (intrathecal chemotherapy).

Radiation therapy uses x-rays or other high-energy rays to kill cancer cells and shrink tumors. Radiation for acute myeloid leukemia in children is also called external beam radiation therapy.

If there is a brother or sister match, bone marrow transplantation is used to replace the patient's bone marrow with healthy bone marrow. This is done after remission is achieved. First, high doses of chemotherapy with or without radiation therapy are given to destroy the bone marrow. Healthy marrow is then taken from another person (a donor) whose tissue type is the same (or almost the same) as the patient's. The donor may be an identical twin (the best match), a brother or sister, or person not related (this tends to be reserved for the situation of relapsed AML). The healthy marrow is given to the patient through a needle in a vein, and the healthy marrow replaces the marrow that was destroyed. A bone marrow transplant using marrow from a relative or person not related is called an allogeneic bone marrow transplant. Another type of bone marrow transplant, called autologous bone marrow transplant, is being studied in clinical trials. During this procedure, bone marrow is taken from the patient and treated with drugs to kill any cancer cells. The marrow is then frozen. Next, the patient is given high-dose chemotherapy, with or without radiation therapy, to destroy all of the remaining bone marrow. The saved frozen marrow is then thawed and returned to the patient to replace the marrow that was destroyed.

Biological therapy attempts to stimulate or restore the ability of the patient's immune system to fight cancer. It uses substances that are normally produced by the patient and that are now made in a laboratory to boost, direct, or restore the body's natural defenses against disease. Biological therapy is sometimes called biological response modifier therapy or immunotherapy. It is most commonly used after chemotherapy has reduced the number of leukemia cells in the body.

There are two or three phases of treatment for acute myeloid leukemia.

The first phase, remission induction therapy, uses chemotherapy to kill as many of the leukemia cells as possible and cause the leukemia to go into remission. Approximately 80% of children with AML achieve remission following induction chemotherapy. Once the leukemia goes into remission and there are no signs of leukemia, postremission therapy is given. The purpose of postremission therapy is to kill any remaining leukemia cells. Postremission therapy is divided into two phases, consolidation and intensification. Your child may receive either or both phases of postremission therapy. The five-year leukemia free survival rate for patients with acute myeloid leukemia is approximately 50%.

As both treatment and preventive therapy, your child will also receive chemotherapy to the central nervous system (CNS). This therapy consists of intrathecal and/or high-dose systemic chemotherapy to kill any leukemia cells present in the central nervous system (CNS) and/or to prevent the spread of cancer cells to the brain and spinal cord. Radiation therapy to the brain and spine may also be given, in addition to chemotherapy, for this purpose.

Unwanted side effects can result from treatment both during therapy as well as after therapy ends, so it is important that your child continue to see his or her doctor. Chemotherapy may lead to heart, kidney, and hearing problems. Radiation therapy may lead to problems with growth, development, and sight (e.g. cataracts).

Treatment on clinical trial

Treatment for childhood acute myeloid leukemia in children depends on the type of leukemia and whether the patient has been previously treated for acute myeloid leukemia. The best treatment is given by cancer doctors with experience in treating children with leukemia at hospitals where pediatric cancer patients are treated.  The current clinical trial being tested nationally is available for children and teens through age 18.

Your child may receive treatment that is considered standard therapy based on its effectiveness in treating patients in past clinical trials, or you may choose to have your child take part in a clinical trial. Clinical trials are designed to test new potentially better treatments against current standard therapy in hopes of finding better ways to treat pediatric cancer patients. The good news is that Children's Oncology Group clinical trials are closely overseen by a data monitoring committee, so that in real time, if one treatment is found to be better than another, the treating physicians are notified immediately, and your child's treatment will be changed right away if necessary.

Special circumstances

The majority of children with Down Syndrome and acute myeloid leukemia can be cured of their leukemia using less intensive chemotherapy regimens than current intensive timing AML therapy. Bone marrow transplantation is usually not indicated in Down Syndrome patients.

The majority of children with Down Syndrome and acute myeloid leukemia can be cured of their leukemia using less intensive chemotherapy regimens than current intensive timing AML therapy. Bone marrow transplantation is usually not indicated in Down Syndrome patients.

Sometimes the diagnosis is Myelodysplastic Syndrome (MDS). Some forms of MDS are a transition to acute myeloid leukemia and are treated the same as acute myeloid leukemia, while other forms are observed before being treated.

A variant of AML called Acute Promyelocytic Leukemia (APL) is sometimes diagnosed. The treatment is different than described above, but it my involved being treated on a clinical trial. Treatment may include arsenic combined with chemotherapy.

Recurrent childhood acute myeloid leukemia

Treatment depends on the type of treatment your child previously received. Treatments currently being studied in clinical trials include new chemotherapy drugs, bone marrow/stem cell transplantation, and biological therapy (e.g. arsenic trioxide, all-trans retinoic acid).

Cleveland Clinic Children's

Here at Cleveland Clinic Children's, we specialize in the care of children and young adults with cancer. We use a team approach that cares for all members of the family, recognizing that this is a very difficult time for all of them. Our goal is to have our patients get through therapy with as normal a life as possible, and to emerge from therapy knowledgeable about how to maintain their health.

Should you have more questions about the child or young adult in your life with cancer, please contact Cleveland Clinic Cancer Answer line at 216.444.5517 or 800.223.2273, extension 4-5517. Your question will be forwarded to the Chair of the Pediatric Oncology Department.

We wish you and your child well.

For other websites regarding childhood cancer:

Source: National Institutes of Health, National Cancer Institute, Children's Oncology Group Last Reviewed: 6/14/05

© 1995-2009 The Cleveland Clinic Foundation. All rights reserved.

This information is provided by the Cleveland Clinic Health System and is not intended to replace the medical advice of your doctor or health care provider. Please consult your health care provider for advice about a specific medical condition.
Rev. 6/05