What are the medications and treatments for acute myeloid leukemia?

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AML treatment depends on a person’s age, overall health, and cancer stage.

Additionally, some people have genetic mutations present in their cancer cells. People with these mutations may be candidates for certain targeted therapies to kill the cancer.

An oncologist may recommend several tests to determine which treatment is appropriate. In general, for young or otherwise healthy people, the treatment regimen is:

1. Induction chemotherapy: This is the first stage of chemotherapy and aims to reduce the number of cancer cells. Most people use the 7+3 regimen, which includes 7 days of receiving the chemotherapy medication cytarabine, with the last 3 days including the addition of an anthracycline.
2. Stem cell transplant: The next stage of treatment is a hematopoietic stem cell transplant. The stem cells help a person’s body recover better, enabling doctors to give them a stronger dose of chemotherapy.
3. Consolidation therapy: If a person is healthy enough, the next step is high-dose chemotherapy.

Chemotherapy is the treatment doctors most commonly use to treat AML. They may also use other treatments, such as targeted drugs and corticosteroids.

The Food and Drug Administration (FDA) has approved numerous drugs specifically for the treatment of AML. These include:

Chemotherapy

Chemotherapy drugs kill cancer cells. However, they can also damage healthy cells.

The most common chemotherapy treatment for AML includes a combination of cytarabine and a group of drugs called anthracyclines. Examples of anthracyclines include daunorubicin, idarubicin, and daunomycin.

Some examples of other chemotherapy drugs used to treat AML include:

• cladribine
• fludarabine
• hydroxyurea
• decitabine
• methotrexate

Targeted drugs

Certain genes and proteins can enable cancer cell growth. Targeted drugs aim to attack these genes and proteins. A doctor or oncologist will prescribe different targeted drugs depending on a person’s specific gene mutation.

Targeted drugs for AML include:

FLT3 inhibitors

Some leukemia cells have a mutation on the FLT3 gene. Drugs that target this gene can slow the cancer’s growth.

If a person’s cancer cells have a mutation on this particular gene, doctors may use FLT3 inhibitors to treat it. The drugs midostaurin and gilteritinib are FLT3 inhibitors, and more are currently in clinical development.

IDH inhibitors

Cancer cells can also have mutations in the IDH1 or IDH2 genes. Having one of these mutations can stop a person’s blood cells from developing properly, which can make them sicker.

IDH inhibitors prevent this. Examples include ivosidenib and enasidenib.

BCL-2 inhibitor

BCL-2 is a protein in cancer cells that prolongs their life.

Doctors can prescribe a BCL-2 inhibitor called venetoclax to prevent this. Doctors usually give it to people over 75 who cannot safely receive chemotherapy.

Corticosteroids

Corticosteroids are a type of anti-inflammatory drug that can reduce the body’s immune response.

They can help treat some unpleasant cancer and chemotherapy side effects, such as anemia and allergic reactions. Some corticosteroids used for AML include prednisone and dexamethasone.

Other medications

Other medications may help a person manage the side effects of cancer treatment. These include:

• anti-diarrheal medications such as loperamide
• anti-nausea medications such as ondansetron
• antibiotics
• medications to manage depression and anxiety
• pain relief medication
• laxatives for chemotherapy constipation
• antifungal medication for fungal infections

Killing cancer cells can also damage healthy cells, causing side effects. The specific medication side effects a person may experience depend on the treatment their doctor prescribes.

Chemotherapy

Most chemotherapy drugs target cells that grow quickly, including healthy rapid-growing cells in the hair, digestive tract, and bone marrow.

Some common side effects include:

• neurotoxicity, which is brain damage that may affect mood, thinking, memory, and personality
• hair loss
• vomiting, nausea, and diarrhea
• skin reactions at the chemotherapy injection site
• damage to the skin in the mouth, including sores and bleeding gums
• exhaustion
• increased vulnerability to infections

Corticosteroid drugs

Corticosteroid drugs can cause a wide range of side effects, including:

• higher risk of infection
• weight gain, especially in the face
• osteoporosis, or low bone density
• high blood pressure
• fluid retention that may damage the organs, including the heart
• cataracts
• ulcers
• insomnia
• anxiety and other psychological changes, including aggression

Targeted therapy

Targeted drugs can cause side effects such as:

• nausea
• vomiting
• shortness of breath
• bleeding
• pain
• infections
• organ damage, such as heart or lung problems

Complementary therapies are remedies designed to add to, not replace, the standard of care for AML. Some options include:

• Psychotherapy: This may help a person manage the challenges of living with AML.
• Natural and herbal remedies: A number of natural and plant remedies have shown early promise in lab tests, though not in the human body. These remedies should not replace chemotherapy.
• Mind-body therapies: Mindfulness, meditation, and gentle movement may help ease pain and support people to better manage the emotional challenges of AML.
• Complementary medicine: Complementary medical practices such as acupuncture and aromatherapy could offer some relief, though the research on these interventions has not proven they work.

AML is an aggressive form of cancer, and a person’s outlook will depend on their age, disease progression, and other factors.

About 40% of people under the age of 60 reach remission, but that number drops to 15% for people over 60. Additionally, the long-term survival odds depend on the type of AML a person has. For example, the group of mutations that includes t(8;21), t(15;17), or inv(16) has a 3-year survival rate of 66% for people under 60, and 33% for those over 60.

Early diagnosis and prompt treatment are important factors in improving a person’s AML outlook.

There is no treatment regimen or medication that works for everyone. While chemotherapy is the primary form of treatment for AML, targeted therapies and corticosteroids can also improve a person’s prognosis.

It is important for a person to consider treatment goals, the odds of success, and the side effects of treatment when weighing options.

Discussing options with a doctor or oncologist can help a person and their loved ones make the right decisions for their quality of life.