Treatment

The treatment of AML depends on the subtype, morphology, and cytogenetics of AML (see Subtypes) and the patient’s overall health. In many cases, a team of doctors will work with the patient to determine the best treatment plan.

The most successful treatment for AML depends on the results of the first treatment, so it is important to undergo the initial treatment at a center with expertise in AML.

This section outlines treatments that are the standard of care (the best treatments available) for this specific type of cancer. Patients are also encouraged to consider clinical trials as a treatment option when making treatment plan decisions. A clinical trial is a research study to test a new treatment to prove it is safe, effective, and possibly better than standard treatment. Your doctor can help you review all treatment options. For more information, visit the Clinical Trials section.

Descriptions of the most common treatment options for AML are listed below.

Chemotherapy

Chemotherapy is the use of drugs to kill cancer cells. The drugs travel through the bloodstream to cancer cells throughout the body. Chemotherapy is given by a medical oncologist, a doctor who specializes in treating cancer with medication, or a hematologist a doctor who specializes in treating blood diseases. Some people may receive chemotherapy in their doctor's office; others may go to the hospital. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a specific time.

Chemotherapy is the primary treatment for AML. Chemotherapy may be given by mouth, injected into a vein, or injected into the CSF. Several drugs are used to treat AML.

Chemotherapy for AML can be divided into three phases: remission induction, post-remission consolidation, and maintenance. Maintenance refers to the prolonged use of chemotherapy for patients in remission and is not commonly used in AML.

Induction. This stage of therapy refers to the initial period of treatment after the diagnosis is made. The goal of induction therapy is a complete remission (CR), which means that the blood counts have returned to normal, the leukemia is not visible when a marrow sample is examined under the microscope, and the signs and symptoms related to the AML are gone.

The combination of cytarabine (Cytosar-U, Tarabine PFS) given over seven days and an anthracycline drug (daunorubicin [Cerubidine, Rubidomycin] or idarubicin [Idamycin]) given for three days is used most often. In addition to killing leukemia cells, these drugs also destroy healthy cells, increasing the risk of infection and bleeding (see below). Most patients require hospitalization for three to five weeks during this period of induction therapy. Sometimes, two courses of therapy are needed to achieve a CR. Approximately 75% of younger adults with AML and about 50% of patients older than 50 achieve a CR after treatment.

Consolidation or intensification. This stage of chemotherapy refers to the use of a variety of different drugs that are designed to kill remaining AML cells after successful induction. It is known that the AML will usually recur if no further therapy is given after a CR. Younger adults in remission are commonly given two to four courses of high-dose cytarabine at monthly intervals, while a number of different regimens are used for older patients. Although the chemotherapy is usually given in the hospital, most of the recovery time is spent as an outpatient.

For some patients, stem cell transplantation (see below) is recommended as a substitute for consolidation therapy.

Learn more about chemotherapy and preparing for treatment. The medications used to treat cancer are continually being evaluated. Talking with your doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications. Learn more about your prescriptions by using searchable drug databases.

Acute promyelocytic leukemia (APL) treatment

The treatment of the APL subtype of AML is somewhat different. This subtype is remarkably sensitive to the effects of all-trans retinoic acid (ATRA), a drug that is similar to vitamin A and is given by mouth. The combination of ATRA and chemotherapy with idarubicin or daunorubicin produces a very high rate of CR.

Bleeding is a common complication of APL, and patients with this subtype of AML often require large numbers of platelet and blood transfusions during the initial stages of their treatment. In contrast to other subtypes of AML where maintenance therapy is not used, patients with APL benefit from the long-term (one to two years) use of ATRA. Arsenic trioxide (Trisenox) is effective when given intravenously (through a vein) for the treatment of APL that has come back after treatment.

Side effects of chemotherapy

The chemotherapy given to treat AML attacks rapidly dividing cells, including those in normal tissues, such as the hair, lining of the mouth, intestines, and bone marrow. People with AML receiving chemotherapy may lose their hair, develop mouth sores, or have nausea and vomiting. Hair will regrow after treatment is completed, and effective drugs help prevent nausea and vomiting.

Because of the effect on the bone marrow, the chemotherapy used in AML will temporarily lower the body’s resistance to infection, leading to increased bruising and bleeding, and fatigue. People with AML will receive a variety of antibiotics to prevent and treat infections and will require transfusions of red blood cells and platelets throughout their chemotherapy treatments. Chemotherapy may also affect the patient’s future fertility (ability to conceive a child or maintain a pregnancy).

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to kill cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. The most common type of radiation therapy is called external-beam radiation therapy, which is radiation given from a machine outside the body. Because AML is found throughout the body in the blood, radiation therapy is generally used only when the cancer has spread to the brain or to shrink a chloroma (a localized mass of tissue).

Side effects from radiation therapy may include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished. Learn more about radiation therapy.

Stem cell transplantation/bone marrow transplantation

A stem cell transplant is a medical procedure in which diseased bone marrow is replaced by highly specialized cells, called hematopoietic stem cells. Hematopoietic stem cells are found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because blood stem cells are typically what is being transplanted, not the actual bone marrow tissue.

There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO).

In an ALLO transplant, stem cells are obtained from a donor whose tissue matches the patient’s on a genetic level; this testing is called HLA-typing. Most often, a patient’s brother or sister serves as the donor, although unrelated donors can serve as the donor too. Millions of people worldwide have volunteered to donate stem cells for patients who do not have matched family members; your health care team will search a computer registry to look for a match. In addition, stem cells derived from umbilical cord blood are sometimes considered if family donors are not available.

In an AUTO transplant, the patient’s own stem cells are used. The stem cells are obtained from the patient when he or she is in remission from previous treatment. The stem cells are then frozen until they are needed, usually after the high-dose treatment (explained below) is completed.

In both types, the goal of transplantation is to destroy cancer cells in the marrow, blood, and other parts of the body and have replacement blood stem cells create healthy bone marrow. In most stem cell transplants, the patient is treated with high doses of chemotherapy and/or radiation therapy to destroy as many cancer cells as possible. This also destroys the patient’s bone marrow tissue and suppresses the patient’s immune system so that, in an ALLO transplant, the donor cells are not rejected by the body. After the high-dose treatment is given, blood stem cells are infused into the patient’s vein to replace the bone marrow and restore normal blood counts from donor cells. Sometimes, ALLO transplants can also be performed after giving lower doses of chemotherapy and/or radiation therapy that are still sufficient to suppress the immune system and allow growth of the donor cells. (These transplants, sometimes termed “mini-transplants” or “reduced intensity transplants” have less immediate side effects, allowing the procedure to be used for older patients.)

Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of cancer, results of any previous treatment, and patient’s age and general health.

For both ALLO and AUTO transplant types, the replacement cells engraft (begin to make new blood cells) and turn into healthy, blood-producing tissue in 10 days to three weeks. Destroying the patient’s own marrow reduces the body’s natural defenses, temporarily leaving the patient at an increased risk of infection. Until the patient’s immune system is back to normal, patients may need antibiotics and blood transfusions.

In an ALLO transplant, another major risk is that the donor’s cells will recognize the patient’s body as foreign, causing graft-versus-host disease (GVHD). GVHD may be a serious complication of allogeneic transplants and can be fatal. Other side effects may include liver problems, diarrhea, infections, and rashes. However, GVHD can also be a benefit, in that the donor cells can recognize the cancer cells as foreign and destroy these cells, a mechanism that is one of the major reasons why ALLO transplantation generally works so well over the long term. The risk of GVHD can be reduced with exact HLA-type matching and the use of preventive drugs.

In an AUTO transplant, there is little risk of GVHD because the replacement stem cells are the patient’s own cells. However, there is a risk in an autologous transplant that some of the cells that are put back into the patient could still be cancerous.

Learn more about bone marrow and stem cell transplantation.

Recurrent/refractory AML

If leukemia is still present after initial treatment, the disease is referred to as refractory AML. Patients with refractory disease may be offered new drugs being tested in clinical trials. Patients should also be considered for ALLO transplant as part of their treatment program.

The treatment for recurrent AML often depends on the length of the initial remission. If AML comes back after a long remission, it may respond again to the original treatment. If the remission was short, then other drugs are used, often in the form of new drugs being tested in clinical trials. An ALLO stem cell transplant is often offered to patients whose leukemia has come back after initial treatment. However, the best treatment after a recurrence is not known, and many drugs and other approaches are being evaluated in clinical trials.

Find out more about common terms used during cancer treatment.