Acute Myeloid Leukemia Treatment Option Overview

There are different types of treatment for people with acute myeloid leukemia (AML).

Different types of treatments are available for people with AML. You and your cancer care team will work together to decide your treatment plan, which may include more than one type of treatment. Many factors will be considered, such as the phase of the cancer, your overall health, and your preferences. Your plan will include information about your cancer, the goals of treatment, your treatment options and the possible side effects, and the expected length of treatment. 

Talking with your cancer care team before treatment begins about what to expect will be helpful. You’ll want to learn what you need to do before treatment begins, how you’ll feel while going through it, and what kind of help you will need. To learn more, see Questions to Ask Your Doctor about Treatment. 

The treatment of AML usually has two phases.

There are two treatment phases of AML:

• Remission induction therapy is the first phase of treatment. The goal is to kill the leukemia cells in the blood and bone marrow. This puts the leukemia into remission.
• Postremission therapy is the second phase of treatment. It begins after the leukemia is in remission. The goal of postremission therapy is to kill any remaining leukemia cells that may not be active but could begin to regrow and cause a relapse. This phase is also called remission continuation therapy.

Patients receive supportive care for side effects of treatment.

Patients must be closely monitored during treatment of AML. Myelosuppression, a condition which results in fewer red blood cells, white blood cells, and platelets, is a side effect of both AML and treatment with chemotherapy. Supportive care during remission induction therapy may include:

• red blood cell and platelet transfusions to replace blood cells destroyed by the cancer or its treatment
• antibiotics and antifungals to prevent or treat infections

The following types of treatment are used:

Chemotherapy

Chemotherapy (also called chemo) uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. The way the chemotherapy is given depends on the subtype of AML being treated and whether the leukemia cells have spread to the central nervous system (CNS; brain and spinal cord).

Systemic chemotherapy is when chemotherapy drugs are taken by mouth or injected into a vein or muscle. When given this way, the drugs enter the bloodstream and can reach cancer cells throughout the body.

Systemic chemotherapy drugs used to treat AML include:

Combinations of these drugs may be used. Other chemotherapy drugs not listed here may also be used.

Intrathecal chemotherapy may be used to treat AML that has spread to the CNS (brain and spinal cord). Intrathecal chemotherapy is a method of placing chemotherapy directly into the cerebrospinal fluid, which is the fluid that surrounds the brain and spinal cord. This approach is used because the blood-brain barrier, a protective layer around the brain, can prevent chemotherapy drugs given by mouth or into a vein from reaching the CNS.

Cytarabine and are two chemotherapy drugs given as intrathecal chemotherapy to treat AML. These drugs can also be given systemically.

To learn more about how chemotherapy works, how it is given, common side effects, and more, see Chemotherapy to Treat Cancer and Chemotherapy and You: Support for People With Cancer.

Radiation therapy

Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. AML is sometimes treated with external radiation therapy. This type of radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Total-body irradiation sends radiation toward the whole body. It is a type of external radiation that may be used to prepare the body for a stem cell transplant when the leukemia has recurred.

Learn more about External Beam Radiation Therapy for Cancer and Radiation Therapy Side Effects.

Chemotherapy with stem cell transplant

High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and/or total-body irradiation, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.

Learn more about Stem Cell Transplants in Cancer Treatment.

Targeted therapy

Targeted therapy uses drugs or other substances to identify and attack specific cancer cells. Your doctor may suggest biomarker tests to help predict your response to certain targeted therapy drugs. Learn more about Biomarker Testing for Cancer.

Targeted therapies used to treat AML include:

Less-intensive targeted therapies in people who are unable or unwilling to receive other treatments include:

Learn more about Targeted Therapy to Treat Cancer. 

Other drug therapy

Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia.

New types of treatment are being tested in clinical trials.

A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option.

Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers.

Treatment for acute myeloid leukemia may cause side effects.

For information about side effects caused by treatment for cancer, visit our Side Effects page.

Follow-up care may be needed.

As you go through treatment, you will have follow-up tests or checkups. Some tests that were done to diagnose or stage the cancer may be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. These tests are sometimes called follow-up tests or check-ups.

Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back).