Chemotherapy and Drug Therapy

Chemotherapy drugs kill fast-growing cells throughout the body, including both cancer cells and normal, healthy cells. The damage to normal, healthy cells can cause side effects. Chemotherapy is typically given in cycles. Each cycle is made up of a certain number of days of treatment, followed by a certain number of days of rest. 

Some patients may also receive targeted therapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific types of cancer cells with less harm to normal cells. Not all cancers have the same targets. Targeted therapy may be used alone or in combination with chemotherapy.

Phases of Treatment 

AML treatment is generally done in two phases

Treatment for patients with acute promyelocytic leukemia (APL), the M3 subtype of AML, differs from other AML treatments. Click here to read about treatment for APL.

AML patients whose leukemia cells have certain genetic mutations are assigned a specific risk status. Talk to your doctor about treatments available to target specific genetic mutations. See the LLS fact sheet, Cancer Molecular Profiling.

Induction Therapy

The first phase of your treatment is induction therapy. The goal of induction is to destroy as many cancer cells as possible in order to achieve (induce) a complete remission. A complete remission is achieved when:

  • No more than 5 percent of cells in the bone marrow are blast cells
  • Blood cell counts are back to normal
  • All signs and symptoms of AML are gone

If the initial treatment does not induce a remission, induction therapy can be repeated, either with the same drugs or with a new chemotherapy regimen.

Induction therapy typically requires a 4-6 week hospital stay. Most patients develop dangerously low blood cell counts and may become very ill, needing supportive (palliative) care with IV antibiotics and frequent blood transfusions during this time. 

What type of treatment is used for AML induction therapy?

The most common induction regimen for AML includes cytarabine and an anthracycline drug, such as daunorubicin or idarubicin. This is called the “7 + 3” regimen, because cytarabine is most often given by continuous intravenous (IV) infusion over 7 days, while the anthracycline drug is given by an IV infusion in a single dose for 3 days during the first week of treatment.

Other drugs may be added or substituted for these “7+3” drugs for higher-risk patients and targeted therapies, such as:

  • Midostaurin (Rydapt®) for FLT3-mutated AML
  • Gemtuzumab ozogamicin (Mylotarg™) for CD33-positive AML
  • Daunorubicin and cytarabine (Vyxeos®) for the treatment of newly-diagnosed therapy-related AML or AML with myelodysplasia-related changes in adults

For information about the drugs listed on this page, visit Drug Listings.

Minimal/Measurable Residual Disease 

Even when a complete remission is achieved, some leukemia cells that cannot be seen with a microscope may still remain in the bone marrow. This is referred to as minimal residual disease (MRD), also called measurable residual disease. Patients who achieve remission after initial treatment but have MRD are at increased risk of disease relapse. Testing for MRD can help doctors identify patients who may benefit from further treatment with intensified therapies, such as allogeneic stem cell transplantation. It is important to get tested for MRD after achieving remission. The tests used most commonly to detect MRD are flow cytometry, polymerase chain reaction (PCR) and next-generation sequencing. 

See the free LLS booklet Minimal Residual Disease (MRD) for more information. 

Consolidation (Post-remission) Therapy

"Consolidation therapy," also called "post-remission therapy," is treatment that is given after cancer is in remission following induction therapy. The goal of consolidation therapy is to lower the number of residual leukemia cells in the body, or eliminate them entirely to help prevent the leukemia from returning. Without additional therapy, the leukemia is likely to relapse within weeks or months.

What type of treatment is used for AML consolidation therapy?

There are three basic treatment choices for consolidation therapy:

Patients with favorable risk factors are often given intensive chemotherapy with high-dose cytarabine and other drugs for their consolidation therapy.

Patients with high-risk AML, based on their  prognostic factors, receive more aggressive therapy, such as allogeneic stem cell transplantation.

For patients 60 years of age or older who achieve a complete remission after induction therapy but who are not able to complete intensive chemotherapy or proceed to allogeneic stem cell transplantation, the doctor may prescribe an oral formulation of azacitidine (Onureg®) as maintenance therapy.

Central Nervous System (CNS) Involvement

AML cells can spread to the cerebrospinal fluid, the fluid around the brain and spinal cord. This is uncommon, occurring in less than 3 percent of AML patients. Because CNS involvement is rare in cases of AML, doctors often do not test for it at the time of diagnosis unless the patient is experiencing neurologic symptoms, such as headache or confusion. If neurologic symptoms are present, the doctor may order imaging tests and/or a lumbar puncture to determine if there are leukemia cell in the spinal fluid. 

What type of treatment is used for CNS involvement?

If leukemia cells are found in the spinal fluid, “intrathecal chemotherapy” is administered, a treatment in which chemotherapy drugs are injected directly into the spinal fluid. 

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