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Acute Myeloid Leukemia Treatment Option Overview

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

Different types of treatment are available for patients with acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some 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. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

The treatment of AML usually has two phases.

The two treatment phases of AML are:

  • Remission induction therapy: This 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: This 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.
  • Antibiotics and antifungals for treatment of infections.

The following types of treatment are used:

Chemotherapy

Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Intrathecal chemotherapy may be used to treat adult AML that has spread to the brain and spinal cord. Combination chemotherapy is treatment using more than one anticancer drug.

The way the chemotherapy is given depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord.

For more information, see Drugs Approved for Acute Myeloid Leukemia.

Radiation therapy

Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External 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.

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.

Targeted therapy

Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy.

Monoclonal antibodies: immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells.

  • Gemtuzumab ozogamicin is a type of antibody-drug conjugate used to treat patients with newly diagnosed or relapsed AML. It contains a monoclonal antibody that binds to CD33, which is found on some leukemia cells, and also contains a toxic substance, which may help kill cancer cells.

Other targeted therapies include:

  • Midostaurin: a protein kinase inhibitor used with certain types of chemotherapy to treat newly diagnosed patients with AML that has a mutation in the FLT3 gene.
  • Gilteritinib: a tyrosine kinase inhibitor that may be used to treat patients with AML that has come back or did not get better with other treatment and has a mutation in the FLT3 gene.

Less-intensive targeted therapies in older or frail patients who cannot receive other treatments include:

  • Glasdegib plus low-dose chemotherapy.
  • Ivosidenib with or without low-dose chemotherapy for patients with AML that has a mutation in the IDH1 gene.
  • Enasidenib for patients with AML that has a mutation in the IDH2 gene.

For more information, see Drugs Approved for Acute Myeloid Leukemia.

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.

For more information, see Drugs Approved for Acute Myeloid Leukemia.

New types of treatment are being tested in clinical trials.

This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website.

Treatment for acute myeloid leukemia may cause side effects.

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

Patients may want to think about taking part in a clinical trial.

For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.

Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.

Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.

Patients can enter clinical trials before, during, or after starting their cancer treatment.

Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.

Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Follow-up tests may be needed.

Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order 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.

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). These tests are sometimes called follow-up tests or check-ups.

This information is not intended to replace the advice of a doctor. Navigating Care disclaims any liability for the decisions you make based on this information. This information was sourced and adapted from Adapted from the National Cancer Institute's Physician Data Query (PDQ®) Cancer Information Summaries on www.cancer.gov.