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Acute Myeloid Leukemia (AML)

What is Acute Myeloid Leukemia in Children and Teens?

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is the second most common childhood leukemia after acute lymphoblastic leukemia (ALL).

About 500 children are found to have AML in the U.S. each year. It is much more common in adults.

Childhood AML is most common during the first 2 years of life and during the teenage years.

AML affects blood cells called myeloid stem cells. Normally, the bone marrow makes blood-producing stem cells that either become myeloid stem cells or lymphoid stem cells. A myeloid stem cell becomes one of three types of mature blood cells:

  • Red blood cells
  • White blood cells called granulocytes
  • Platelets
Microscope image that shows normal bone marrow

This image shows how normal, healthy bone marrow appears through a microscope.

Microscope image shows bone marrow of a patient with acute myeloid leukemia

This is the bone marrow of a pediatric patient with acute myeloid leukemia.

What Causes Acute Myeloid Leukemia?

In leukemia, cancer cells grow rapidly in the bone marrow. These cancer cells are immature white blood cells called blasts. When this happens, healthy blood cells — white blood cells, red blood cells, and platelets — can’t do their jobs correctly.

Graphic showing the blood forming process and how it results in blast cells. The graphic begins with a blood stem cell. To the left, it branches off into myeloid stem cell, which branches into platelets, red blood cells, myeloblast, and monoblast. The myeloblast changes into white blood cells (also called granulocytes) and the monoblast changes into a monocyte. The right branch of blood stem cell goes to lymphoid stem cell, which branches into lymphoblasts (which changes into white blood cells) and blast cells.

AML affects blood cells called myeloid stem cells. Normally, the bone marrow makes blood-producing stem cells that either become myeloid stem cells or lymphoid stem cells.

AML affects blood cells called myeloid stem cells. Normally, the bone marrow makes blood-producing stem cells that either become myeloid stem cells or lymphoid stem cells. A myeloid stem cell becomes one of three types of mature blood cells:

  • Red blood cells
  • White blood cells called granulocytes
  • Platelets

Risk factors for childhood AML include having:

  • A brother or sister, especially a twin, with leukemia
  • Past treatment with chemotherapy or radiation
  • Myelodysplastic syndrome
  • Certain inherited disorders

Having certain inherited disorders, such as:

  • Diamond-Blackfan syndrome
  • Familial platelet disorder
  • Fanconi anemia
  • Li-Fraumeni syndrome
  • Mismatch repair syndromes
  • MonoMAC syndrome
  • Rothmund-Thomson syndrome
  • Schwachman-Diamond syndrome

Acute Myeloid Leukemia Symptoms

In AML, signs and symptoms may include:

  • Fever
  • Fatigue
  • Infections
  • Easy bruising and bleeding
  • Frequent nosebleeds
  • Pain in bones or joints
  • Pain or feeling of fullness under the rib cage
  • Swollen lymph nodes
  • Poor appetite

Acute Myeloid Leukemia Diagnosis

Bone marrow tests are usually required to diagnose leukemia. Doctors may begin to suspect leukemia after conducting a physical exam, taking a medical history, and looking at the results of blood tests. Children with leukemia usually have a high number of immature white blood cells in their blood.

  1. During the exam and history, the provider will:

    • Check general signs of health, including signs of disease, such as lumps or anything else that is unusual.
    • Examine the eyes, mouth, skin, and ears. The provider will feel the patient’s abdomen for signs of an enlarged spleen or liver. In boys, the provider may also examine the testes.
    • Ask about other medical conditions the patient may have and illnesses that relatives such as parents, siblings, and grandparents may have had. The provider is looking for possible inherited conditions that have an increased risk of cancer.
  2. Complete blood count

    Doctors will order a blood test called a complete blood count. A sample of blood is drawn and checked for the:

    • Number of red blood cells and platelets
    • Number and type of white blood cells
    • Amount of hemoglobin in the red blood cells
    • Portion of the blood sample made up of red blood cells

    In leukemia, the blood may have a high number of white blood cells. Many of these cells can be blasts, an early form of cell that is usually found only in the bone marrow in healthy children.

    Blood chemistry studies

    A blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease.

    A young patient sits on an exam table with her parents nearby while a nurse draws a blood sample.

    Doctors may begin to suspect leukemia after conducting a physical exam, taking a medical history, and looking at the results of blood tests.

If cancer is determined, more tests will be performed to pinpoint the subtype of the cancer. These tests include:

  1. Immunophenotyping is used to diagnose specific types of leukemia by comparing the cancer cells to normal cells of the immune system.

    Immunohistochemistry and flow cytometry are the laboratory tests.

    • Immunohistochemistry is a test that uses antibodies to show specific proteins in a sample of tissue. The complexes of proteins and antibodies are stained brown or red and can be seen under a microscope.
    • In flow cytometry, cells are stained with a light-sensitive dye, placed in a fluid, and passed in a stream before a laser or other type of light. The test measures the number of cells, the percentage of live cells, and certain characteristics of cells, such as size, shape, and the presence of tumor markers on the cell surface.
  2. Cytogenetic analysis involves laboratory tests in which pathologists look for certain changes in the chromosomes.

    One such test is FISH (fluorescence in situ hybridization). This test looks at genes or chromosomes in cells and tissues. Pieces of DNA that contain a fluorescent dye are made in the laboratory and added to cells or tissues on a glass slide. When these pieces of DNA attach to certain genes or areas of chromosomes on the slide, they light up.

  3. The doctor may recommend running laboratory tests to identify specific genes, proteins, and other factors involved in the leukemia. This examination is important because cancer is caused by mistakes (mutations) in the cell’s genes.

    Identifying these mistakes helps diagnose the specific subtype of leukemia. Based on that information, doctors can choose treatment options tailored to the individual case. Children whose leukemia shows mutations associated with a good outcome may be prescribed less-toxic treatments. On the other hand, doctors may prescribe more intensive treatments for patients with a leukemia with mutations associated with poorer outcomes. Mutations may be identified for which a treatment targeted to that specific mutation are available.

Tests to determine if the cancer has spread include:

  1. A lumbar puncture is performed to collect a sample of cerebrospinal fluid from the spinal column to determine if the cancer has spread to the central nervous system. This procedure is also called an LP or spinal tap.

    A needle is placed between two bones in the spine and into the fluid around the spinal cord. A sample of fluid is removed. It is checked under a microscope for signs that leukemia cells have spread to the brain and spinal cord. Our bodies make spinal fluid constantly, so the body quickly replaces the small amount an LP takes.

  2. An X-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body on a computer screen or special film. The chest X-ray is done to see if leukemia cells have formed a mass in the middle of the chest.

Acute Myeloid Leukemia Treatment

Treatment depends on the type of AML. Three forms of AML — acute promyelocytic leukemia (APL), AML in children with Down syndrome, and FLT3-mutated AML — are treated differently from other forms of AML.

Chemotherapy is the primary AML treatment. Bone marrow transplant may also be an option.


The goal of the induction phase is to kill leukemia cells in the blood and bone marrow and bring the disease into remission. Since AML patients are vulnerable to infection, supportive therapy with antibiotics is also given.

Central nervous system (CNS) sanctuary therapy (also called CNS prophylaxis) may also be given during this time to kill leukemia cells that remain in the brain and spinal cord. Medications are injected into the fluid-filled space between the thin layers of tissue that cover the brain and spinal cord (intrathecal).

Induction therapy typically includes a combination of drugs such as cytarabine and an anthracycline, most commonly daunorubicin. Etoposide, thioguanine, or gemtuzumab ozogamicin may also be given during induction therapy.


The goal of this phase is to kill any remaining leukemia cells that could grow and cause the cancer to relapse. Cancer centers can perform tests that can detect a single AML cell among 1,000 normal cells. Children who have more than one cell in 1,000 after completing the induction phase are at the greatest risk of relapsing.

This consolidation phase begins after the patient is in remission. It includes 2-4 cycles of chemotherapy and lasts for 4 to 6 months. Such therapy includes some of the drugs used in induction while also introducing non-cross–resistant drugs and commonly high-dose cytarabine.

Hematopoietic cell transplant 

A hematopoietic cell transplant (also known as a bone marrow transplant or stem cell transplant) may be recommended for children who are at high risk for relapse or whose AML is resistant to treatment. Doctors sometimes look at how well induction chemotherapy worked to decide whether a bone marrow transplant is needed.

AML patients may receive an allogeneic transplant.

In an allogeneic bone marrow transplant, children receive blood cell-producing cells from a healthy donor. Patients must have a suitable donor to be eligible for a transplant. Before receiving the donor cells, the patient’s existing blood cells in the bone marrow are destroyed by chemotherapy and sometimes radiation. The patient receives the healthy donor blood and marrow cells through an infusion. If successful, these new donor cells will grow into and replace the patient’s blood and marrow cells. As a result, the patient’s bone marrow should start to produce healthy blood cells.

Acute Myeloid Leukemia Prognosis

The five-year survival rate for childhood AML is about 70 percent.

About 90 percent of children with AML have no cancer cells in their blood after initial treatment. About 30 percent of children with AML relapse or have disease that is resistant to treatment (refractory).

Late Effects of Acute Myeloid Leukemia

Some AML patients may have late effects. A late effect is a health problem that occurs months or years after treatment has ended.

Cancer patients should continue to be followed by their treatment center care team and/or a primary care provider in the community after cancer treatment. Late effects can often be treated or, in some cases, prevented.

Different treatments may have different late effects. Not all patients will have late effects. Patients who had the exact same treatment may experience different late effects.

AML patients may be at risk for:

  • Recurrence of the AML
  • Second cancers such as skin, brain cancer, bone, breast, soft tissue, and thyroid
  • Heart problems
  • Liver problems
  • Fertility problems
  • Cognitive problems

Patients who undergo a hematopoietic cell transplant may be at risk for certain late effects.

Focus of Current Acute Myeloid Leukemia Research

Researchers are testing new drugs to treat AML. These include:

  • Selinexor
  • Venetoclax
  • Flotetuzumab
  • Vyxeos

Reviewed: June 2020