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Acute Myeloid Leukemia (AML)

What is Acute Myeloid Leukemia?

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. In leukemia, cancer cells crowd out healthy blood cells, which can cause fever, fatigue, easy bruising, bleeding, infections, and other problems. Acute leukemia means that symptoms typically worsen over a short period of time. Children may become sick very quickly and need medical attention right away.

  • About 500 children are found to have AML in the U.S. each year. (It is much more common in adults.) 
  • AML is the second most common childhood leukemia after acute lymphoblastic leukemia (ALL). 
  • Childhood AML is most common during the first 2 years of life and during the teenage years.
  • The number of children with AML is about equal between boys and girls and between people of European and African descent.
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.

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

AML Subtypes

There are several subtypes of AML. This is important because the type of AML determines prognosis and treatment.

Knowing the subtype of AML allows doctors to classify AML cases into low-risk or high-risk, which helps them select the most appropriate treatment. Treatment for AML is usually chemotherapy and sometimes bone marrow transplant. Prescribing treatment based on risk category (risk-adapted therapy) has led to increased cancer survival rates. Patients with higher risk forms of cancer may receive the most aggressive therapy while patients with a lower-risk case may receive a lower-intensity treatment that has fewer side effects. For more information about AML subtype, ask your physician.

Subtypes of acute myeloid leukemia (AML) and related tumors seen in children (World Health Organization, 2016):

  • AML with recurrent genetic abnormalities
  • AML with translocation between chromosomes 8 and 21
  • AML with translocation or inversion in chromosome 16
  • APL with PML-RARA (acute promyelocytic leukemia)
  • AML with translocation between chromosomes 9 and 11
  • AML with translocation of chromosomes 6 and 9
  • AML with translocation or inversion of chromosome 3
  • AML (megakaryoblastic) with translocation of between chromosomes 1 and 22
  • AML with mutated NPM1
  • AML with biallelic mutations of CEBPA
  • Provisional entity: AML with mutated RUNX1
  • AML with myelodysplasia-related changes
  • Therapy-related myeloid neoplasms
  • AML, not otherwise specified 
  • AML with minimal differentiation 
  • AML without maturation 
  • AML with maturation 
  • Acute myelomonocytic leukemia 
  • Acute monoblastic/monocytic leukemia 
  • Pure erythroid leukemia 
  • Acute megakaryoblastic leukemia 
  • Myeloid sarcoma 
  • Myeloid proliferations related to Down syndrome 
  • Transient abnormal myelopoiesis (TAM) 
  • Myeloid leukemia associated with Down syndrome

Signs and Symptoms of Acute Myeloid Leukemia

In AML, signs and symptoms may include:

  • Fever
  • Feeling very tired
  • Infections
  • Easy bruising and bleeding
  • Frequent nosebleeds
  • Bleeding that is hard to stop, even from a small cut
  • Pain in bones or joints
  • Pain or feeling of fullness under the rib cage
  • Swollen lymph nodes
  • Poor appetite

Diagnosis of Acute Myeloid Leukemia

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. At first, a physician will perform a physical exam and look at the patient’s medical history. During the physical exam, the doctor will check general signs of health, including signs of disease, such as lumps or anything else that seems unusual. The eyes, mouth, skin, and ears will be looked at carefully. A nervous system exam may be done. The doctor will feel the patient’s abdomen for signs of an enlarged spleen or liver.  

    In looking at the patient's medical history, the doctor will look to see if there are any medical conditions or patterns of cancer in the family that could indicate that the cancer may be inherited. Only 8-10 percent of childhood cancers is inherited. 

    Risk factors for childhood AML include:

    • Having a brother or sister, especially a twin, with leukemia.
    • Past treatment with chemotherapy or radiation therapy.
    • Having myelodyplastic syndrome.
    • Having certain inherited disorders, such as:
      • Diamond-Blackfan syndrome
      • Down syndrome
      • Fanconi anemia
      • Li-Fraumeni syndrome
      • Mismatch repair syndromes
      • Neurofibromatosis type 1
      • Noonan syndrome
      • Schwachman-Diamond syndrome
  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 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.

  3. Bone marrow tests like bone marrow aspiration and biopsy will confirm a diagnosis of cancer and pinpoint the type of cancer. Most children are asleep (sedated) during the procedure. If awake during the procedure, patients are given appropriate pain medicine.

    Bone marrow aspiration: Doctors will obtain a bone marrow sample by inserting a thin, hollow needle into the hipbone. A pathologist will view the bone marrow under a microscope to look for signs of cancer.

    Bone marrow biopsy:  Doctors will remove a small piece of bone tissue to determine how much the cancer has spread in the bone marrow. The biopsy is typically performed right before or after the aspiration.  

    How is a bone marrow aspiration / biopsy performed?

    • Patients will lie on their side on the bed or, less commonly, sit up with legs bent and crossed at the ankles. 
    • The doctor or nurse practitioner will feel the patient’s lower back to find the right spot for the test. 
    • Next, the health care provider will put on gloves and clean the patient’s back with a germ-killing soap. Then, the health care provider might place plastic towels over the back, leaving only a small area of skin showing. 
    • If the patient is going to be awake during the aspirate or biopsy, numbing cream will be placed on the spot where the bone marrow aspirate or biopsy will be taken about one hour before the procedure. Liquid numbing medicine might be injected at the procedure site. 
    • The health care provider will insert a needle through the skin into the bone marrow. If both an aspiration and biopsy are done, a separate needle will be used for each procedure.
    • The health care provider will take out the needle, clean the spot with alcohol, and put on a bandage.
    A pediatric cancer patient is positioned for a bone marrow biopsy with a clinician's hands performing the procedure.

    Bone marrow tests are usually required to diagnose leukemia.

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. 

Treatment of Acute Myeloid Leukemia

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.

1. Induction

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 drug, most commonly daunorubicin, in combination with etoposide and/or thioguanine. 

2. Consolidation/intensification/post-induction

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 are capable of detecting 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.

Prognosis for Acute Myeloid Leukemia

The five-year survival rates 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

A late effect is a health problem that occurs months or years after a disease is diagnosed or after treatment has ended. Late effects may be caused by cancer or cancer treatment. They may include physical, mental, and social problems and second cancers.

Late effects may include:

  • Recurrence of the AML
  • Second cancers such as skin, brain cancer, bone, breast, soft tissue, and thyroid
  • Heart and lung problems
  • Bone problems
  • Endocrine conditions
  • Gastrointestinal issues
  • Difficulties with mental flexibility, reasoning, planning, and organization
  • Depression
  • Sleep problems

Focus of Current Acute Myeloid Leukemia Research

Current research focuses on developing more effective treatments for children whose cancer doesn’t respond to the original therapy, developing more therapies that target the cancer cells without harming surrounding healthy cells, and designing effective therapies with fewer side effects.

Reviewed: June 2018