Acute myeloid leukemia (AML) treatment options

Treatment for acute myeloid leukemia (AML) usually begins soon after diagnosis because the disease can progress quickly. Treatment plans are personalized based on factors such as a person’s age, overall health, AML subtype and how the leukemia responds to initial therapy.

Your care team will help explain available treatment options and recommend the best approach for your individual situation.

How AML is treated

AML treatment is designed to help remove leukemia cells and allow the body to begin making healthy blood cells again. Depending on the type of AML and how the disease responds to treatment, patients may receive one treatment or a combination of therapies.

Treatment plans may also change over time based on how the body responds to therapy and whether additional treatment is needed to reduce the risk of relapse.

Common AML treatment options include:

Chemotherapy
Blood or marrow transplant (BMT)
Clinical trials

Treating AML with chemotherapy

Chemotherapy uses powerful medicines to destroy leukemia cells and help the body begin producing healthy blood cells again. Because AML often progresses quickly, chemotherapy treatment may begin soon after diagnosis. For many people with AML, chemotherapy is used to help achieve remission, meaning there are no visible signs of leukemia in the body.

Treatment plans may include one or multiple phases of chemotherapy depending on the subtype of AML, a person’s overall health and how the leukemia responds to treatment. Some chemotherapy medicines are given through an intravenous (IV) line, while others may be taken by mouth.

Chemotherapy may be used:

As the first treatment after diagnosis
Before a blood or marrow transplant (BMT)
After transplant to help reduce the risk of relapse
Alongside clinical trials or cell therapies

Induction therapy

The first phase of AML chemotherapy is often called induction therapy. The goal of induction therapy is to destroy as many leukemia cells as possible and help achieve remission.

Because induction treatment can be intensive, some patients may stay in the hospital for several weeks while their care team monitors blood counts, manages side effects and helps prevent infections.

Consolidation therapy

If remission is achieved, additional chemotherapy called consolidation therapy may be recommended. The goal of consolidation therapy is to destroy any remaining leukemia cells that may still be present in the body and reduce the risk of AML returning.

Many patients move forward with a blood or marrow transplant as part of consolidation treatment, especially if their care team believes there is a higher risk of relapse.

Side effects of chemotherapy

Chemotherapy can affect both leukemia cells and healthy fast-growing cells in the body, which may lead to side effects. Side effects vary depending on the medicines used and how a person’s body responds to treatment.

Common side effects may include:

Fatigue: Chemotherapy can lower healthy red blood cell counts, making it harder for the body to carry oxygen efficiently.
Nausea or vomiting: Some chemotherapy medicines can irritate the digestive system and affect the parts of the brain that control nausea.
Hair loss: Chemotherapy may damage hair follicle cells, leading to temporary hair loss.
Increased risk of infection: Treatment can lower white blood cell counts, reducing the body’s ability to fight infections.

Easy bruising or bleeding: Low platelet counts caused by chemotherapy can make it harder for blood to clot properly.
Mouth sores: Chemotherapy can irritate the lining of the mouth and digestive tract, leading to tenderness or sores.
Loss of appetite: Treatment side effects such as nausea, taste changes or fatigue may make eating more difficult.
Brain fog or trouble concentrating: Some patients experience memory or concentration difficulties during treatment, sometimes called “chemo brain.”

Your health care team can help manage side effects and provide supportive care throughout chemotherapy treatment.

Blood or marrow transplant for AML

A blood or marrow transplant (BMT), also known as a bone marrow transplant, blood stem cell transplant or stem cell transplant, replaces unhealthy blood-forming cells with healthy donor cells.

For many people with AML, BMT is an important part of treatment planning and may offer the possibility of long-term disease control or cure. Doctors may recommend transplant based on factors such as AML subtype, risk of relapse and response to chemotherapy.

Because finding a suitable donor can take time, transplant planning and donor searches often begin early in the AML treatment process. A patient’s medical team works with NMDP^SM to access a network of donor registries worldwide to find the right donor for them.

NMDP also provides educational resources, support services and guidance to help patients, families and caregivers navigate AML treatment and transplant. Research initiatives such as Donor for All are also helping expand access to blood or marrow transplant for more patients with AML.

Learn more about blood or marrow transplant for AML

Treating AML with clinical trials and cell therapies

Clinical trials help researchers study new treatments and improve existing cell therapies available for AML. Some clinical trials explore new medicines, while others focus on improving treatment outcomes or reducing side effects.

Participation in a clinical trial is always voluntary, and patient safety is closely monitored throughout the process. Depending on a person’s diagnosis and treatment response, clinical trials may be an option at different stages of care.

NMDP’s Patient Support Center can help patients better understand available clinical trial options, eligibility requirements and next steps. Patients, families and caregivers should always speak with their care team to better understand whether a clinical trial may be appropriate for their situation.

Explore clinical trial support

How to choose the right AML treatment option

The best AML treatment plan will vary from person to person. Doctors consider several factors when recommending treatment options, including:

Age and overall health
AML subtype
Genetic or molecular testing results
Risk of relapse
Response to initial treatment
Whether a donor is available for transplant

Your health care team can help explain the risks, benefits and goals of each treatment option so you can make informed decisions about your care.

Support for you and your loved ones facing an AML diagnosis

A diagnosis of AML can affect every part of life for patients, caregivers and families. NMDP offers educational resources, transplant support information and guidance to help people better understand AML, navigate treatment decisions and access additional support throughout their journey.

NMDP can help by:

Answering questions about AML and treatment options
Offering counseling support resources to help you cope with uncertainty
Helping you explore financial assistance resources
Providing access to free educational resources including fact sheets, booklets and videos
Answering questions about the transplant process
Helping you find and join clinical trials
Connecting you with others who have been through transplant for AML

Having as much information as possible on your treatment options for AML will help you and your doctor make the best treatment decisions for your individual case. We’ve outlined some of key resources below.

Learn more about the services and resources available to patients