Chemotherapy for acute myelogenous leukemia

Chemotherapy uses anticancer, or cytotoxic, drugs to destroy cancer cells. Chemotherapy is the main treatment for acute myelogenous leukemia (AML). Your healthcare team will consider your personal needs to plan the drugs, doses and schedules of chemotherapy. You may also receive other treatments.

Chemotherapy is given for different reasons. You may have chemotherapy to:

  • destroy the leukemia cells, bring about a remission and return blood cell production to normal
  • destroy leukemia cells in the brain and spinal cord (called the central nervous system, or CNS)
  • prepare for a stem cell transplant

Chemotherapy is usually a systemic therapy. This means that the drugs travel through the bloodstream to reach and destroy cancer cells all over the body, including those that may have spread to other parts of the body through the blood. Chemotherapy may also be a regional therapy, which means that it is given to a specific area of the body. Intrathecal chemotherapy is given directly into the spaces containing cerebrospinal fluid (CSF) through a lumbar puncture (spinal tap) or an Ommaya reservoir.

Preparing for chemotherapy

Preparation before chemotherapy may include the following surgical procedures.

The surgeon may place a central venous catheter in a vein in the upper chest so you won’t need as many injections.

If you are receiving intrathecal chemotherapy, the surgeon may place an Ommaya reservoir beneath the scalp. An Ommaya reservoir is a small, dome-shaped device with a short tube, or catheter, attached to it. The chemotherapy drug is injected using a small needle inserted through the scalp into the Ommaya reservoir. The drug then goes directly into the spaces containing cerebrospinal fluid.

Chemotherapy drugs used for AML

Chemotherapy for AML usually involves a combination of 2 or 3 drugs.

The most common chemotherapy combination used is cytarabine (Cytosar, Ara-C) with an anti-tumour antibiotic. The usual approach is called the 7-and-3 protocol. For this protocol, cytarabine is given continuously for 7 days, and then the anti-tumour antibiotic is given daily for 3 days. The anti-tumour antibiotics commonly used in the 7-and-3 protocol are:

  • daunorubicin (Cerubidine, daunomycin)
  • doxorubicin (Adriamycin)
  • idarubicin (Idamycin)
  • mitoxantrone (Novantrone)

Other drugs that may be used to treat AML include:

  • thioguanine (Lanvis, 6-TG)
  • fludarabine (Fludara)
  • cladribine (Leustatin)
  • etoposide (Vepesid, VP-16)
  • cyclophosphamide (Cytoxan, Procytox)
  • topotecan (Hycamtin)
  • methotrexate (Methotrexate)
  • azacitidine (Vidaza)

If AML does not respond to drugs used in earlier treatments, or if it recurs, the following drugs may be used:

  • a repeat course of the 7-and-3 protocol with cytarabine and an anti-tumour antibiotic
  • high-dose cytarabine (HDAC) alone or in combination with an anti-tumour antibiotic
  • MEC – etoposide, cytarabine and mitoxantrone
  • high-dose etoposide and cyclophosphamide
  • GLAG-M – cladrabine, cytarabine, mitoxantrone and granulocyte colony-stimulating factor (G-CSF)
  • fludarabine, cytarabine and G-CSF

Chemotherapy drugs used for acute promyelocytic leukemia (APL)

Tretinoin (all-trans retinoic acid, ATRA, Vesanoid) is given in combination with other drugs to treat APL. Other drugs include:

  • daunorubicin
  • idarubicin
  • cytarabine
  • mercaptopurine (Purinethol, 6-MP)
  • methotrexate
  • arsenic trioxide

Central nervous system treatment

If the leukemia has spread to the brain and spinal cord (called the central nervous system, or CNS), chemotherapy may be given directly into the spinal fluid (called intrathecal chemotherapy). The drug used for intrathecal chemotherapy is methotrexate or cytarabine. It is given during a lumbar puncture or through an Ommaya reservoir.

Side effects

Side effects can happen with any type of treatment for AML but everyone’s experience is different. Some people have many side effects. Other people have few or none at all.

Chemotherapy may cause side effects because it can damage healthy cells as it kills cancer cells. Side effects can develop any time during, immediately after or a few days or weeks after chemotherapy. Sometimes late side effects develop months or years after chemotherapy. Most side effects go away on their own or can be treated, but some side effects may last a long time or become permanent.

Side effects of chemotherapy will depend mainly on the type of drug or drug combination, the dose, how it’s given and your overall health. Some common side effects of chemotherapy drugs used for AML are:

Tell your healthcare team if you have these side effects or others you think might be from chemotherapy. The sooner you tell them of any problems, the sooner they can suggest ways to help you deal with them.

Information about specific cancer drugs

Details on specific drugs change quite regularly. Find out more about sources of drug information and where to get details on specific drugs.

Questions to ask about chemotherapy

Find out more about chemotherapy and side effects of chemotherapy. To make the decisions that are right for you, ask your healthcare team questions about chemotherapy.

Expert review and references

  • American Society of Clinical Oncology (ASCO). Leukemia - Acute Myeloid - AML: Treatment Options. Alexandria, VA.: American Society of Clinical Oncology (ASCO); 2013:
  • Kebriaei P, Champlin R, de Lima M, et al . Management of acute leukemias. DeVita VT Jr, Lawrence TS, & Rosenberg SA. Cancer: Principles & Practice of Oncology. 9th ed. Philadelphia: Wolters Kluwer Health/Lippincott Williams & Wilkins; 2014: 131: pp. 1928-1954.
  • Kurtin SE . Leukemia and myelodysplastic syndromes. Yarbro, CH, Wujcki D, & Holmes Gobel B. (eds.). Cancer Nursing: Principles and Practice. 7th ed. Sudbury, MA: Jones and Bartlett; 2011: 57: pp. 1369-1398.
  • National Cancer Institute. Adult Acute Myeloid Leukemia Treatment (PDQ®) Health Professional Version. Bethesda, MD: National Cancer Institute; 2014:
  • Seiter K . Acute myelogenous leukemia treatment & management. WebMD LLC; 2014.
  • Wiernic PH, Gallagher RE, Tallman MS . Acute promyelocytic leukemia. Wiernik PH, Goldman JM, Dutcher JP & Kyle RA (eds.). Neoplastic Diseases of the Blood. 5th ed. Springer; 2013: 23: pp. 403-453.
  • Wiernik PH . Diagnosis and treatment of adult acute myeloid leukemia other than acute promyelocytic leukemia. Wiernik PH, Goldman JM, Dutcher JP & Kyle RA (eds.). Neoplastic Diseases of the Blood. 5th ed. Springer; 2013: 22: pp. 375-401.