Leukemia - Acute Myeloid - AML: Types of Treatment

Approved by the Cancer.Net Editorial Board, 04/2022

ON THIS PAGE: You will learn about the different types of treatments doctors use for adults with acute myeloid leukemia (AML). Use the menu to see other pages.

This section explains the types of treatments, also known as therapies, that are the standard of care for AML. “Standard of care” means the best treatments known. When making treatment plan decisions, you are encouraged to discuss with your doctor whether clinical trials are an option. A clinical trial is a research study that tests a new approach to treatment. Doctors learn through clinical trials whether a new treatment is safe, effective, and possibly better than the standard treatment. Clinical trials can test a new drug, a new combination of standard treatments, or new doses of standard drugs or other treatments. Clinical trials are an option for all stages of leukemia. Your doctor can help you consider all your treatment options. Learn more about clinical trials in the About Clinical Trials and Latest Research sections of this guide.

How AML is treated

In cancer care, different types of doctors often work together to create a patient’s overall treatment plan that combines different types of treatments. This is called a multidisciplinary team. Cancer care teams include a variety of other health care professionals, such as physician assistants, nurse practitioners, oncology nurses, social workers, pharmacists, counselors, dietitians, and others.

Treatment options and recommendations depend on several factors, including the subtype, morphology, and cytogenetics of AML (see Subtypes), possible side effects, and the patient’s preferences and overall health.

Take time to learn about all of your treatment options and be sure to ask questions about things that are unclear. Talk with your doctor about the goals of each treatment and what you can expect while receiving the treatment. The goals of your care will drive your doctor's recommendations and where you seek treatment. If possible, it is helpful to have your treatment at a center experienced with treating AML. These types of talks are called “shared decision-making.” Shared decision-making is when you and your doctors work together to choose treatments that fit the goals of your care. Shared decision-making is particularly important for AML because there are different treatment options. Learn more about making treatment decisions.

The common types of treatments used for AML are described below. Your care plan may also include treatment for symptoms and side effects, an important part of cancer care.

Therapies using medication

Treatments using medication are used to destroy cancer cells. Medication may be given through the bloodstream to reach cancer cells throughout the body. When a drug is given this way, it is called systemic therapy. Medication may also be given locally, which is when the medication is applied directly to the cancer or kept in a single part of the body.

This treatment is generally prescribed by a medical oncologist or a hematologist. A medical oncologist is a doctor who specializes in treating cancer with medication. A hematologist is a doctor who specializes in treating blood disorders. Sometimes, a doctor can be trained as a medical oncologist and a hematologist. Both medical oncologists and hematologists may treat AML.

Medications to treat AML are given in different ways:

  • An intravenous (IV) tube placed into a vein using a needle. When chemotherapy is given by IV, it may be given into a larger vein or a smaller vein, such as in the arm. When it is given into a larger vein, a central venous catheter or port may need to be placed in the body.

  • An injection into the cerebral spinal fluid. This is called intrathecal therapy.

  • In a pill or capsule that is swallowed (orally)

  • An injection under the skin, called a subcutaneous injection

If you are given oral medications, be sure to ask your health care team about how to safely store and handle them.

The types of medications used for AML include:

  • Chemotherapy

  • Targeted therapy

Each of these types of therapies are discussed below in more detail. A person may receive 1 type of medication at a time or a combination of medications given at the same time. They can also be given as part of a treatment plan that includes other treatments.

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.

It is also important to let your doctor know if you are taking any other prescription or over-the-counter medications or supplements. Herbs, supplements, and other drugs can interact with cancer medications, causing unwanted side effects or reduced effectiveness. Learn more about your prescriptions by using searchable drug databases.

Intensive chemotherapy

Chemotherapy is the use of drugs to destroy cancer cells, usually by keeping the cancer cells from growing, dividing, and making more cells. Chemotherapy is the primary treatment for AML.

A chemotherapy regimen, or schedule, usually consists of a specific number of cycles given over a set period of time. A patient may receive 1 drug at a time or a combination of different drugs given at the same time. Several drugs are used to treat AML, which are discussed below.

Learn more about the basics of chemotherapy.

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Chemotherapy by phase

Chemotherapy for AML can be divided into 3 phases: induction, post-remission consolidation, and maintenance.

Induction therapy. This is the first period of treatment after a person’s diagnosis. The goal of induction therapy is a complete remission (CR). A person has a CR when:

  • Blood counts have returned to normal

  • Leukemia cannot be found in a bone marrow sample when examined under the microscope

  • There are no longer any signs and symptoms of AML

The combination of cytarabine (Cytosar-U) given over 4 to 7 days and an anthracycline drug, such as daunorubicin (Cerubidine) or idarubicin (Idamycin), given for 3 days is used most often. Targeted therapy (see below) may also be a recommended part of this treatment.

Patients may also be given hydoxyurea (Droxia, Hydrea) to help lower white blood cell counts before starting induction therapy. In addition to killing leukemia cells, these drugs also damage healthy cells, increasing the risk of infection and bleeding (see below). Most patients will need to stay in the hospital for 3 to 5 weeks during induction therapy before their blood counts return to normal. Sometimes, 2 rounds of therapy are needed to achieve a CR. Approximately 75% of younger adults with AML and about 50% of patients older than 60 achieve a CR after treatment.

Some older adults may not be able to have induction therapy with the standard drugs. The drugs of azacitidine (Vidaza), low-dose cytarabine, decitabine (Dacogen), glasdegib (Daurismo), and venetoclax (Venclexta, Venclyxto) may be used instead. A clinical trial is also an option.

Post-remission Consolidation. After induction therapy, a variety of different drugs are used to destroy AML cells that remain in small amounts that cannot be detected by medical tests. AML will almost certainly recur if no further treatment is given after a CR. For some patients, bone marrow/stem cell transplantation (see below) is recommended as part of post-remission therapy.

Chemotherapy or stem cell transplantation may be used for consolidation therapy.

  • Younger adults in remission are commonly given 2 to 4 rounds of high- or intermediate-dose cytarabine or other intensive chemotherapy at monthly intervals. Several different regimens are used for older patients. Although chemotherapy is usually given in the hospital, most of the recovery time can be spent at home.

  • A bone marrow/stem cell transplantation is often recommended as post-remission consolidation therapy for patients in whom cytogenetic or molecular studies predict a poorer prognosis with only chemotherapy or targeted therapy.

Maintenance. Even after completion of post-remission consolidation, there is a risk that AML can still return. Lower strength medications can be given on an ongoing basis for several years to reduce the chance of the disease returning, called recurrence. Medications such as azacitidine (Vidaza), decitabine (Dacogen), or midostaurin (Rydapt) are examples.

Bone marrow transplantation/stem cell transplantation. A bone marrow transplant is a medical procedure in which bone marrow that contains leukemia is destroyed and then replaced by highly specialized cells. These cells, called hematopoietic stem cells, develop into healthy bone marrow. Hematopoietic stem cells are blood-forming cells found both in the bloodstream and in the bone marrow. This procedure is also called a stem cell transplant or hematopoietic stem cell transplant.

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

There are 2 types of hematopoietic stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). ALLO uses donated stem cells, while AUTO uses the patient’s own stem cells. ALLO transplants are generally used for AML.

The goal is to destroy all of the cancer cells in the marrow, blood, and other parts of the body using high doses of chemotherapy and/or radiation therapy and then allow replacement blood stem cells to create healthy bone marrow.

Side effects depend on the type of transplant, your general health, and other factors. Learn more about the basics of stem cell and bone marrow transplantation.

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Side effects of chemotherapy

Chemotherapy for AML attacks rapidly dividing cells, including those in healthy 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 finished, and there are effective drugs to help prevent and control nausea and vomiting. The side effects of chemotherapy may be different depending on the drugs used. Patients are encouraged to talk with their doctors about short-term and long-term side effects before treatment begins.

Because of the effect on healthy blood cells in the bone marrow, chemotherapy used for AML will lower the body’s ability to fight infection for a short time, and increased bruising, bleeding, and fatigue may be common. People with AML often receive antibiotics to prevent and treat infections and will need transfusions of red blood cells and platelets throughout chemotherapy. Chemotherapy may also affect the patient’s fertility, or ability to have a child in the future. Patients concerned about this are encouraged to talk with a fertility specialist before treatment begins.

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Targeted therapy

Targeted therapy is a treatment that targets the leukemia’s specific genes, proteins, or the tissue environment that contributes to the growth and survival of the leukemia. This type of treatment blocks the growth and spread of leukemia cells and limits damage to healthy cells.

Not all cancers have the same targets. To find the most effective treatment, your doctor may run tests to identify the genes, proteins, and other factors in your tumor. This helps doctors better match each patient with the most effective treatment whenever possible. In addition, research studies continue to find out more about specific molecular targets and new treatments directed at them. Learn more about the basics of targeted treatments.

The following targeted treatments may be used for AML, depending on the gene mutations found in the leukemia cells:

Drugs targeting FLT3 gene mutations. There are 2 drugs approved to AML with an FLT3 gene mutation. About 25% to 30% of people with AML have AML with an FLT3 mutation.

  • Midostaurin (Rydapt) is approved in combination with chemotherapy to treat people who have been newly diagnosed with AML with an FLT3 mutation.

  • Gilteritinib (Xospata) is approved to treat relapsed or refractory AML with an FLT3 mutation.

Drugs targeting IDH1 or IDH2 gene mutations. There are 2 drugs and 1 drug combination approved to treat relapsed or refractory AML with IDH1 and IDH2 mutations. About 10% of people with AML have AML with an IDH1 or IDH2 mutation.

  • Enasidenib (IDHIFA) is approved to treat relapsed or refractory AML with an IDH2 mutation.

  • Ivosidenib (Tibsovo) is approved to treat relapsed or refractory AML with an IDH1 mutation.

  • Ivosidenib (Tibsovo) is also approved in combination with azacitidine (Vidaza), a type of chemotherapy, for newly diagnosed AML, with an IDH1 mutation, in patients aged 75 years and older or patients under 75 years of age with co-existing conditions preventing use of intensive induction chemotherapy.

Talk with your doctor about possible side effects for a specific medication and how they can be managed

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Acute promyelocytic leukemia (APL) treatment

The treatment of the APL subtype of AML is very different. This subtype is very sensitive to the effects of all-trans retinoic acid (ATRA). ATRA is a drug that is similar to vitamin A and is given by mouth. People with the APL subtype who receive a combination of ATRA and arsenic trioxide (Trisenox) are very likely to have a CR and be cured. Less commonly, chemotherapy containing regimens (see above) with idarubicin, daunorubicin, or cytarabine may also be used. Arsenic trioxide may be used during induction therapy alone or in combination with ATRA during post-remission therapy or if APL comes back after treatment.

Mild to severe bleeding is a common symptom of APL. Patients with this subtype often need many platelet and blood transfusions during initial treatment. Some patients with APL may benefit from use of ATRA plus low dose oral chemotherapy for 1 to 2 years after the initial treatment.

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Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to destroy 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. A radiation therapy regimen, or schedule, usually consists of a specific number of treatments given over a set period of time. Because AML is found throughout the blood, radiation therapy is generally used only when leukemia cells have spread to the brain or to shrink a myeloid sarcoma.

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 the basics of radiation therapy.

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Physical, emotional, and social effects of leukemia

Leukemia and its treatment cause physical symptoms and side effects, as well as emotional, social, and financial effects. Managing all of these effects is called palliative care or supportive care. It is an important part of your care that is included along with treatments intended to slow, stop, or eliminate the leukemia.

Palliative care focuses on improving how you feel during treatment by managing symptoms and supporting patients and their families with other, non-medical needs. Any person, regardless of age or type and stage of cancer, may receive this type of care. And it often works best when it is started right after diagnosis. People who receive palliative care along with treatment for the cancer often have less severe symptoms, better quality of life, and report that they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional and spiritual support, and other therapies. You may also receive palliative treatments similar to those meant to get rid of the leukemia, such as chemotherapy or radiation therapy.

Before treatment begins, talk with your doctor about the goals of each treatment in the recommended treatment plan. You should also talk about the possible side effects of the specific treatment plan and palliative care options. Many patients also benefit from talking with a social worker and participating in support groups. Ask your doctor about these resources, too.

During treatment, your health care team may ask you to answer questions about your symptoms and side effects and to describe each problem. Be sure to tell the health care team if you are experiencing a problem. This helps the health care team treat any symptoms and side effects as quickly as possible. It can also help prevent more serious problems in the future.

Learn more about the importance of tracking side effects in another part of this guide. Learn more about palliative care in a separate section of this website.

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Refractory AML

If leukemia is still present after initial treatment, the disease is called refractory AML. If this happens, it is a good idea to talk with doctors who have experience in treating it. Doctors can have different opinions about the best standard treatment plan. Clinical trials might also be an option. Learn more about getting a second opinion before starting treatment, so you are comfortable with your chosen treatment plan.

Your treatment plan may include new drugs being tested in clinical trials. An ALLO bone marrow/stem cell transplant (see above) should also be considered. Palliative care will also be important to help relieve symptoms and side effects.

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Remission and the chance of recurrence

A remission is when the leukemia cannot be detected in the body, there are no symptoms, and a patient’s blood counts are normal. This may also be called having “no evidence of disease” or NED.

A remission may be temporary or permanent. This uncertainty causes many people to worry that the cancer will come back. While many remissions are permanent, it is important to talk with your doctor about the possibility of the leukemia returning. Understanding your risk of recurrence and the treatment options may help you feel more prepared if the disease does return. Learn more about coping with the fear of recurrence.

If the leukemia returns after the original treatment, it is called recurrent or relapsed leukemia. If the leukemia returns, a new cycle of testing will begin again to learn as much as possible about the recurrence. After this testing is done, you and your doctor will talk about the treatment options. Often the treatment plan will include the treatments described above, such as chemotherapy, stem cell transplantation, targeted therapy, and radiation therapy, but they may be used in a different combination or given at a different pace. Your doctor may suggest clinical trials that are studying new ways to treat this type of recurrent leukemia. Whichever treatment plan you choose, palliative care will be important for relieving symptoms and side effects.

The treatment for recurrent AML often depends on the length of the initial remission. If the AML comes back after a long remission, the original treatment may work again. If the remission was short, then other drugs are used, often through a clinical trial. An ALLO stem cell transplant may be the best option for patients whose leukemia has come back after initial treatment. However, many drugs and other approaches are being researched in clinical trials and these may provide other treatment options.

People with recurrent leukemia sometimes experience emotions such as disbelief or fear. You are encouraged to talk with your health care team about these feelings and ask about support services to help you cope. Learn more about dealing with cancer recurrence.

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If treatment does not work

Recovery from leukemia is not always possible. If the leukemia cannot be cured or controlled, the disease may be called advanced or terminal.

This diagnosis is stressful, and for some people, advanced leukemia may be difficult to discuss because it is incurable. However, it is important to have open and honest conversations with your health care team to express your feelings, preferences, and concerns. The health care team has special skills, experience, and knowledge to support patients and their families and is there to help. Making sure a person is physically comfortable, free from pain, and emotionally supported is extremely important.

People with advanced leukemia who have no more effective treatment options available may want to consider hospice care. Hospice care is designed to provide the best possible quality of life for people who are near the end of life. You and your family are encouraged to talk with the health care team about hospice care options, which include hospice care at home, a special hospice center, or other health care locations. Nursing care and special equipment can make staying at home a workable option for many families. Learn more about advanced cancer care planning.

After the death of a loved one, many people need support to help them cope with the loss. Learn more about grief and loss.

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The next section in this guide is About Clinical Trials. It offers more information about research studies that are focused on finding better ways to care for people with cancer. Use the menu to choose a different section to read in this guide.