Chemotherapy and Drug Therapy

Acute myeloid leukemia (AML) is often treated in two phases, induction therapy and postremission (consolidation) therapy.

What is Chemotherapy?

Chemotherapy is the use of potent drugs or chemicals, often in combinations or intervals, to kill or damage cancer cells.
Chemotherapy can be hard on the body: The drugs’ toxicity can also damage or kill healthy cells and cause side effects.
Everyone experiences side effects differently.

AML treatment is generally done in two phases (cycles)

Induction therapy
Postremission (consolidation) therapy.

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.

Treatment for patients with the FLT3 mutation or other genetic mutations may receive different drugs or drug combinations such as midostaurin (Rydapt®).

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. Its goal is to "induce" (bring on) remission (when no evidence of the disease is left). Specifically, induction therapy for AML attempts to

Kill as many AML cells as possible with chemotherapy
Get healthy blood cell counts back to normal
Get rid of all signs of the disease for an extended time.

What type of treatment is used for AML induction therapy?

Doctors commonly combine two or more chemotherapy drugs to treat AML. Each type of drug works in a different way to kill the cancerous cells. Combining drug types can strengthen their effectiveness.
Most AML patients are treated with a combination of an anthracycline (such as daunorubicin [Cerubidine®], doxorubicin [Adriamycin® PFS, Adriamycin®] or idarubicin [Idamycin®]) and cytarabine (also called cytosine arabinoside or ara-C [Cytosar-U®]).
Gemtuzumab ozogamicin (Mylotarg^TM)
Midostaurin (Rydapt®)
Daunorubicin and cytarabine (Vyxeos®)
Venetoclax (Venclexta®)
Glasdegib (Daurismo^TM)
Ivosidenib (Tibsovo®)
Other drugs may be added or substituted for higher-risk, refractory or relapsed patients.

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

Postremission Therapy

"Postremission therapy," also called "consolidation 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 months.

Minimal Residual Disease. The term “minimal residual disease” (MRD) is used after treatment to refer to the leukemia cells that may still be present in the patient’s body but cannot be detected in the bone marrow with standard tests (such as examining a bone marrow sample under a microscope). However, these residual cancer cells can be detected with more sensitive tests, such as flow cytometry and polymerase chain reaction (PCR). If there are a small number of residual AML cells, they will not interfere with normal blood cell development, but they do have the potential to multiply and cause a relapse. Postremission therapy can help prevent relapse.

What type of treatment is used for AML postremission therapy?

There are two basic treatment choices for postremission therapy:

Intensive chemotherapy
Stem cell transplantation

Patients with favorable risk outcomes are often given intensive chemotherapy with high-dose cytarabine and other drugs for their consolidation therapy. Patients generally receive multiple cycles of chemotherapy.

Patients with high-risk AML, based on their prognostic factors, are rarely cured with chemotherapy alone. The treatment options that may be offered to these patients are allogeneic stem cell transplantation and/or participation in a clinical trial.

Where is the treatment done?

Postremission therapy happens in the hospital and the length of stay depends on the treatment and other factors.

Central Nervous System (CNS) Involvement

What is 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.

Unlike adults with AML, children usually receive intrathecal chemotherapy as central nervous system (CNS) prophylaxis to prevent the spread of leukemia cells to the central nervous system.