Acute Myeloid Leukemia (AML): Treatment Options 

There are many treatment choices for acute myeloid leukemia (AML). Which one may work best for you? It depends on a number of factors, such as:

  • Your age

  • Your overall health

  • Your subtype of AML

  • Results of your lab tests

  • If the leukemia cells have gene or chromosome changes

  • If the leukemia is affecting your brain and spinal cord

  • Your personal needs and preferences

  • How well the leukemia responds to the first treatment (induction)

Learning about your treatment options

You may have questions and concerns about your treatment options. You may also want to know how you’ll feel and function after treatment, and if you’ll have to change your normal activities.

Your healthcare provider is the best person to answer your questions. Your provider can tell you what your treatment choices are, how well they’re expected to work, and what the risks and side effects are. Your healthcare provider may suggest a certain treatment. Or you might be offered more than one and asked to decide which one you’d like to use. It can be hard to make this decision. While it's important to take the time you need to make the best decision for you, AML treatment tends to be started very soon after diagnosis.

Types of treatment for AML

  • Chemotherapy. This is the main way to treat AML. The treatment uses strong medicines to kill cancer cells. The chemo medicines are put into your blood. The goal is to kill the AML cells quickly and put the cancer into remission. Remission means there are no signs of cancer in your body.

  • Stem cell transplant with high-dose chemotherapy. If the normal doses of chemo don't work, or if the leukemia is expected to relapse, you may need very high doses of chemo. These high doses can damage the stem cells in your bone marrow. Blood stem cells are the "starter" cells for new blood cells. Sometimes, high-dose chemo is used to kill all your stem cells. Then you're given stem cells from a volunteer donor. Over time, they go into your bone marrow and restore your body's ability to make blood cells. This is called a stem cell transplant.

  • All-trans retinoic acid (ATRA) and arsenic trioxide. These medicines are used to treat a subtype of AML called acute promyelocytic leukemia (APL). They're not used for other types of AML. ATRA is a lot like vitamin A. It can be given along with other chemo. Arsenic trioxide can be used either alone or along with ATRA. 

  • Radiation therapy. This type of therapy uses high-energy X-rays to kill cancer cells. It's not part of the main treatment for AML. But it may be used to kill or prevent the spread of cancer in your central nervous system (brain and spine). It may be used as part of a stem cell transplant. In rare cases, it may be used to shrink a tumor that's affecting a certain part of the body.

  • Targeted therapy. These medicines target abnormal proteins, such as those caused by gene changes in the AML cells. The medicines include enasidenib, ivosidenib, midostaurin, gilteritinib, sorafenib, and gemtuzumab. They may be helpful in treating AML subtypes that have certain gene changes. Most of them are taken as pills.

    Targeted therapy and lower doses of chemotherapy may be a choice for people age 75 or older who aren't healthy enough to get stronger chemo. The targeted medicines used are venetoclax or glasdegib. In addition, other medicines called hypomethylating agents include azacitidine and decitabine.

Clinical trials for new treatments

Researchers are always finding new and better ways to treat AML. These new methods are tested in clinical trials. Talk with your healthcare provider to find out if there are any clinical trials you should consider.

Talking with your healthcare providers

At first, thinking about treatment options may seem overwhelming. Talk with your healthcare providers, nurses, and loved ones. Make a list of questions. Consider the benefits and possible side effects of each option. Discuss your concerns with your healthcare providers before making a decision.

Online Medical Reviewer: Amy Finke RN BSN
Online Medical Reviewer: Jessica Gotwals RN BSN MPH
Online Medical Reviewer: Todd Gersten MD
Date Last Reviewed: 4/1/2023
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