Acute Myeloid Leukemia

Acute myeloid leukemia (AML) develops when immature myeloid cells are damaged or mutated (changed) and multiply rapidly. This rapid growth leads to an accumulation of abnormal myeloid cells, which overcrowds cells in the bone marrow and prevents the production of healthy blood cells.

Diagnosing AML

In addition to a physical exam, several tests may be used to diagnose AML.

  • Bone marrow aspiration and biopsy are often done at the same time to remove bone marrow tissue samples for examination. A bone marrow biopsy involves removing a sample of marrow from within the bone. For bone marrow aspiration, liquid bone marrow is removed.
  • Lumbar puncture, also called a spinal tap, is a procedure in which the cerebral spinal fluid (CSF) is checked for leukemia cells. CSF is the fluid that surrounds the brain and spinal cord.

Blood Tests

  • Complete blood count (CBC) measures the levels of white blood cells, red blood cells (including hematocrit and hemoglobin levels) and platelets in the blood.
  • Flow cytometry, also called immunophenotyping, is a specialized test that counts blood cells and analyzes cell characteristics that help to classify the subtype of leukemia.
  • Peripheral blood smear is the study of the blood under a microscope to check for blast cells, abnormalities in the blood cells, and the number and types of white blood cells and platelets.

Genetic Tests

  • Cytogenetic testing uses the study of the characteristics of chromosomes (genetic strands) under a microscope to find changes or alterations in the leukemia cells. Cytogenetic testing with fluorescence in situ hybridization (FISH) is a technique that combines cytogenetic testing and fluorescent dyes to detect changes in the chromosomes.
  • Polymerase chain reaction (PCR) can be used to look for genetic changes.

Imaging tests, such as computed tomography (CT), magnetic resonance imaging (MRI), ultrasound and X-ray, are not necessary to diagnose AML but may help determine the extent of the disease.

Classifying AML

Staging is how physicians determine the extent of cancer. Knowing a cancer’s stage helps the doctor recommend the best treatment plan. Doctors use the World Health Organization (WHO) classification system to classify AML into different subtypes based on the appearance of the leukemia cells, as well as the presence or absence of certain mutations or chromosomal changes. The WHO classification system also takes into account factors that can affect prognosis (outlook).

Treating AML

Leukemias with specific genetic changes may respond better to treatments which include a chemotherapy drug that targets that mutation. It is important for your doctor to know all of the mutations and gene changes in your leukemia before you get started with chemotherapy. Clinical trials may offer access to leading-edge treatments that aren’t yet widely available. As you consider treatment options, talk to your doctor about the possibility of participating in a clinical trial (see Forms of Leukemia).


Chemotherapy is the main treatment for AML. Chemotherapy drugs are used to stop the growth of cancer, either by killing cancer cells or by preventing them from growing and dividing. This form of treatment is known as systemic therapy, meaning the drugs travel through the bloodstream and affect cells throughout the entire body. Chemotherapy for AML is usually given in phases.

Induction is the first phase and is designed to kill leukemia cells, clear the blood of them and put the disease in complete remission. Remission is defined as having blood counts that are back to normal, the absence of leukemia cells in blood samples that are examined under microscope and no signs or symptoms of disease.

In the second phase, called post-remission therapy or consolidation therapy, chemotherapy is given to kill the few remaining leukemia cells that cannot be detected because their level is low. The type of consolidation therapy depends on age and the risk of relapse of the individual patient and may consist of short rounds of high-dose chemotherapy or chemotherapy combined with stem cell transplantation.

Targeted Therapy

Targeted therapy uses drugs to block the growth of leukemia cells by targeting specific parts of leukemia cells. For example, certain drugs target the proteins created by specific chromosomal abnormalities found in some leukemia cells. These proteins help the leukemia cells grow. By targeting the proteins, these targeted therapy drugs stop the proteins from working, which helps stop the leukemia cells from growing. This allows your medical team to control the disease while limiting damage to healthy cells.

Stem Cell Transplantation

Stem cell transplantation, also known as bone marrow transplantation, is an infusion of healthy stem cells into the body, typically after high-dose chemotherapy is given. In allogeneic stem cell transplantation, the stem cells come from a volunteer donor whose tissue type closely matches that of the patient. These stem cells can be collected from umbilical cord blood, a family member or another donor. If available, a sister or brother offers the best chance of a close match. If a sibling or another family member is not a good match, an unrelated volunteer donor may be found through a national registry.

People receiving allogeneic transplantation may experience graft-versus-host disease (GvHD) as a side effect. GvHD can occur when white blood cells from the donor (the graft) recognize cells in the body (the host) as foreign and attack them. This problem can cause damage to the skin, liver, intestines and other organs. GvHD can be treated with steroids or other drugs that suppress your immune system.

Radiation Therapy

Radiation therapy is rarely used to treat AML, but it may be used if the cancer has spread to the brain, spinal fluid or testicles.

Clinical trials

Clinical trials may offer access to cutting-edge treatments that are not yet widely available. Many advances in cancer treatment are helping save lives today because of the research conducted in clinical trials. Ask your doctor if a trial might be right for you.

Refractory AML

AML that does not result in complete remission after treatment is called refractory AML. If you receive this diagnosis, talk with your medical team about options, which could include consulting with a specialist who has experience treating refractory AML, getting a second opinion, trying a new treatment strategy or a clinical trial.

Additional Resources


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