What Is the Treatment for Acute Myeloid Leukemia?

Medically Reviewed by Sabrina Felson, MD on December 11, 2022
4 min read

Acute myeloid leukemia (AML) pushes your bone marrow to make large numbers of abnormal and underdeveloped blood cells called blast cells. These cells crowd out healthy mature red blood cells, white blood cells, and platelets. The goal of AML treatments is to destroy unhealthy immature blood cells in your bone marrow and blood. The goal is to put you into a complete remission, which means you have no blood markers or symptoms of cancer.

Several different treatments work on AML:

  • Chemotherapy
  • Stem cell transplant
  • Radiation
  • Targeted therapy

Your treatment will have two phases:

Phase 1: Remission induction therapy. You'll get high doses of chemotherapy to destroy as many leukemia blast cells as possible. There are targeted therapy drugs as well.

Four to 6 weeks after treatment, your bone marrow should start to make healthy blood cells. Your doctor will take a bone marrow sample and do tests to see if any leukemia cells are left in your blood. If no signs of leukemia cells are present, doctors call that being “in remission.” You'll still need to go through post-remission therapy to help you stay in remission.

Phase 2: Post-remission therapy (Consolidation). Post-remission therapy uses more treatments to wipe out any cancer cells that might have been left behind after chemotherapy. This is called a complete remission. You have three options:

  • Chemotherapy. You may get several cycles of high-dose chemotherapy once a month.
  • Allogeneic (from a donor) stem cell transplant
  • Autologous (from yourself) stem cell transplant

Chemotherapy uses strong drugs to kill cancer cells all over your body. You get these drugs by mouth, through an IV, or via an injection under your skin.

If the cancer has spread to your brain or spinal cord, you'll get chemotherapy into the fluid around your brain and spinal cord. Doctors call this intrathecal chemotherapy.

Side effects: Chemotherapy works by killing quickly dividing cells in your body. Cancer cells divide quickly, but so do other cells -- like those in your immune system, the lining of your mouth and intestines, and your hair follicles. When chemotherapy damages these healthy cells, you can have side effects like these:

  • Nausea and vomiting
  • Hair loss
  • Mouth sores
  • Fatigue
  • Loss of appetite
  • Diarrhea and constipation
  • Easy bruising and bleeding
  • Increased risk for infections

Most of these side effects should go away once your treatment ends. Your doctor can give you medicines and other treatments to help you manage chemotherapy side effects.

The higher the dose of chemotherapy you get, the more cancer cells it will kill. Yet high-dose chemotherapy can also damage your bone marrow and cause a dangerous drop in your blood cell levels.

Your doctor can do a stem cell transplant after chemotherapy to replace your damaged bone marrow with young stem cells from your own body or from a donor. These stem cells will grow into new, healthy blood cells.

There are two types of stem cell transplant:

An allogeneic stem cell transplant uses stem cells taken from a donor. This is the most common type of stem cell transplant. A close relative like a parent, brother, or sister will be the best match. One risk of an allogenic transplant is graft-versus-host disease. The donor's cells recognize your body as foreign and attack your organs and tissues.  Symptoms include rashes, itching, nausea, diarrhea, mouth sores, and jaundice -- yellowing of the eyes and skin.

An autologous stem cell transplant removes healthy stem cells from your own bone marrow or blood before you have chemotherapy. Those cells are frozen and then put back into your blood after your treatment has killed cells in the bone marrow. Because the stem cells come from your body, there's less risk of rejection. The downside is that it's hard to separate healthy stem cells from leukemia cells. You might get some leukemia cells put back in during the transplant.

After a stem cell transplant, you'll have to stay in the hospital for a while to be watched and treated for side effects. Because this treatment uses very high doses of chemotherapy, it can cause serious complications like these:

  • Higher risk of infection and bleeding from low blood cell counts
  • Damage to the lungs, bones, and thyroid gland
  • Cataracts -- a clouding of the clear outer cover of the eye
  • Loss of fertility
  • Another cancer years later

Acute promyelocytic leukemia (APL) is a subtype of AML that doctors treat a little differently. In APL, the leukemia cells contain special proteins that change the way your blood clots. Chemotherapy damages the leukemia cells and releases this protein, which can cause dangerous blood clots or severe bleeding.

If you have APL, you'll get medicine to make your leukemia cells change into mature, healthy blood cells so they don't burst open and release their protein.

You might also get chemotherapy with this medicine.

Radiation uses high-energy X-rays to destroy cancer cells. You might get radiation to treat AML that has spread to your brain and spinal cord, or to your bone. Radiation is sometimes also used before a stem cell transplant. Usually adult AML is treated with external radiation therapy, which is given from outside of your body.

Side effects from radiation include:

  • Sunburn-like redness of the skin
  • Mouth sores -- if you get radiation to your head or neck
  • Nausea, vomiting, or diarrhea -- if you get radiation to your belly
  • Fatigue
  • Bleeding or bruising
  • Increased risk of infection

If AML treatments don't work for you, or if they stop working and your cancer begins to grow again, you have another option: You can try a clinical trial.

Clinical trials are studies in which researchers test new treatments. They often are a way for you to try a new medicine that isn't available to everyone. Your doctor can tell you if one of these trials might be a good fit for you, how to sign up, and what to consider first.