Acute Myeloid Leukemia

AML begins in the bone marrow, the soft inner part of certain bones, where new blood cells are made. Changes happen in some of these cells that make them grow out of control. AML usually grows and moves into the blood quickly.

Talking About AML

You will hear many words used to describe AML. Some key ones to know are:

Develops quickly. If not treated immediately, will progress rapidly and likely be fatal.

Short for “myeloblasts.” These are the immature blood-forming cells that normally appear in bone marrow. AML occurs when a blast is “stuck” in its immature state and multiplies.

A cancer of the blood cells, which grow out of control and crowd out healthy cells.

Describes the type of cells that would normally turn into more specific, mature kinds of white blood cells, red blood cells, or platelets.

When leukemia reappears in the marrow after treatment.

Risk Factors, Signs & Symptoms

There isn’t a known cause for most cases of AML, but research has shown that some things may increase your risk. These risk factors include: exposure to certain chemicals (such as benzene), smoking, and some genetic disorders or family history of leukemia. AML is sometimes a secondary cancer caused by radiation or chemotherapy for other cancers.

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If your doctor suspects AML, they will order tests, including:

Blood tests — specialized test to determine how many white blood cells you have and to see if these cells look abnormal. For information on normal results for Complete Blood Count (CBC) tests, visit

Bone marrow biopsy/aspiration — These tests take both solid and liquid portions of the marrow, usually from the pelvis. If 20 percent of the cells in the bone marrow are immature “blast” cells, the diagnosis is usually AML. In normal bone marrow, the blast count is 5 percent or less.

Biomarker Tests

Biomarker tests can be used to diagnose the specific subtype of AML you have, if any. The results can help your doctors decide which treatments may work best against your specific type of AML. The three main types of tests that look at changes (“mutations”) in the cancer’s genes are:

  • Cytogenetics (also called chromosome tests)
  • Fluorescence in situ hybridization (FISH)
  • Other molecular/genomic tests, such as:
    • Polymerase chain reaction (PCR)
    • DNA sequencing and microarray technologies
    • Next generation sequencing – looks for a number of specific genetic changes to the cancer cells at once

Your doctors use these tests to look for mutations in the AML cells (changes in the cancer cell’s DNA). Nearly two-thirds of people with AML have these changes.

Biomarkers that can help your doctors decide your AML treatment include:

  • FLT3 (pronounced “flit3”)
  • IDH1 and IDH2
  • p53 (TP53)
  • NPM1

Ask your doctor if you have been tested for these and other, newer biomarkers that may help decide which treatment is best for you.


AML is not just one disease. It is a set of diseases that are divided into subtypes. This information helps doctors to estimate how the disease will progress and what treatment will work best. Your doctor should tell you which subtype you have and which treatment is recommended.

Treatment & Side Effects

After you find out you have AML, your doctor will tell you which treatments are best for you. Your doctor may ask you what you want to do. This is called Treatment Planning. Due to the acute nature of AML, treatment may start as soon as you are diagnosed. There are often more treatment choices to be made after your first treatment is finished. For example, once you are in remission, you may have the option to get a stem cell transplant.

Some of the factors that help your doctor and health team decide what treatment to recommend include:

  • Your values, goals, and preferences
  • Your age
  • Your overall health
  • Your medical history
  • The subtype of AML you have

AML is an aggressive cancer. If possible, you should have your treatments at a major cancer center or university hospital to find the most up-to-date care. Usually, the first goal of treatment is to get the patient into complete remission (CR). This means that most of the AML has been eliminated from the patient’s blood or bone marrow. The long-term goal is to cure the disease. A cure means that the cancer will never return. This can be difficult to determine, because AML can return after many years. A complete remission (CR) is the first step towards a possible cure. The main types of treatment for AML are:

  • Clinical trials
  • Chemotherapy
  • Targeted therapy
  • Stem cell transplant


Clinical Trials

Researchers are finding new ways to treat AML. This means that the rates of cure are improving. Still, much more research is needed to fully understand the disease and how best to treat it. A clinical trial may be the only way to get certain treatments, including some that are very promising.

Key things to know

Clinical trials for AML are done to test new drugs, combinations of drugs, or different ways of doing stem cell transplant. A doctor experienced in treating AML should be able to recommend specific trials. The U.S. Food and Drug Administration and local review boards oversee all U.S. clinical trials to keep patients safe. If you join a clinical trial, you can leave at any time. Most often, the trial pays the costs of the drug being studied. Then your health insurance and your copay cover “standard” treatment costs. Be sure to ask what costs you may incur.



Most people with AML will receive chemotherapy treatment. The first phase of chemotherapy is induction therapy. It will usually begin right away. The goal of induction therapy is to bring on (“induce”) a complete remission (CR). This means: you have less than 5 percent blast cells in your bone marrow, your blood counts have returned to normal (or mostly normal), and you have no signs or symptoms of AML. Getting to a complete remission can take three to six weeks. Partial remission (PR) is when cancerous cells are reduced but still detectable. There are several chemotherapy drugs that may be used to treat AML. Two or more of these drugs are usually combined at a time. These drugs are given through an IV (into a vein) at the hospital. Younger patients can sometimes receive higher doses of these drugs than older patients can.

Low-dose Treatment

Some people may not be able to have the standard therapy due to other serious health conditions and increased risk from intensive therapy. Others choose not to receive intensive therapy because of the required time in the hospital or the serious side effects and risks. For these people, less intensive chemotherapy drugs may be used. Low-dose treatment may also be used when the AML has a p53 mutation.

Consolidation or Post-remission

Nearly all patients in complete remission after induction would relapse if they did not have further treatment. For this reason, it is usually followed by one to four more cycles of chemotherapy, called consolidation (post-remission) therapy. This uses a variety of drugs, usually given in the hospital. However, most of your recovery time can be spent at home. Sometimes a stem cell transplant is used as post-remission therapy.

Side Effects of Chemotherapy

Even when you are getting better, cancer treatment for AML can make you feel worse. Coping with the side effects of treatment can be one of the hardest parts of cancer. It helps to talk with your health care team about your side effects and how you are feeling. There are many remedies available for side effects. With high-dose chemotherapy (induction), patients often experience severe side effects. You may have physical symptoms, such as hair loss, mouth sores, and digestive problems. You may have low blood cell counts. This could be low red blood cells (anemia) or low white blood cell count (leukopenia). Both can be treated. Most patients receiving induction chemotherapy require frequent transfusions for a few weeks in the hospital. Fevers and infections can also occur during the hospital stay that require IV antibiotics. Chemotherapy for AML can also have long- term side effects that last for months or years. It may also cause “late effects” that may not show up until years after treatment ends. To manage any of these effects you may have, it is critical to see your doctor for follow-up care. Low-dose chemotherapies and consolidation therapy have less significant side effects.

Targeted Therapy

Targeted therapy may be an option for some patients at different points of their treatment. Some targeted therapies are used only based on the findings of biomarker tests, including genetic testing of your cancer cells. Others are helpful for most cases of AML, and do not require specific testing. Targeted drugs aim to block cancer growth “driven” by changes in the cancer’s genetics.

AML targets that have therapies currently are: FLT3 mutations, IDH1 or IDH2 mutations, Hedgehog signaling pathway, and proteins called BCL-2 and CD33. For some of these drugs you need to be tested for the target. Ask your health care team if you have been tested for all the targets that have current therapies, including FLT3, IDH1 or IDH2.

Side Effects of Targeted Therapy

Each targeted therapy can cause different side effects. Some of them can be very serious. The most common ones are: fatigue, nausea, vomiting, fever, diarrhea, respiratory infections, low blood counts, and mouth sores. Your health care team should monitor you closely for side effects. There are drugs that may prevent or reduce these side effects.

Stem Cell Transplant

Your health care team may recommend a stem cell transplant (SCT). This treatment allows you to have high dose chemotherapy to kill any remaining cancerous cells. Because high dose chemo also kills your normal blood-forming cells (stem cells), those are replaced with stem cells collected from the blood of a donor. Bone marrow transplant (BMT) is similar, but the donor’s stem cells are collected from their bone marrow. This process works best if only small amounts of leukemia remain. That’s why it often isn’t used as a first treatment. It is most often used after you have achieved remission, when your doctor thinks there’s a good chance the AML will come back. For patients healthy enough, a stem cell transplant can offer a chance for a much longer or permanent remission than chemotherapy alone.

If your doctor thinks a stem cell transplant may work for you after induction therapy, it is important to start a search as soon as possible to identify a matching donor. You will work with a new team—a transplant team—to find a donor and coordinate the transplant.

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