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Chemotherapy uses drugs to destroy cancer cells. The drugs enter the bloodstream and travel through the body to the leukemia cells. Chemotherapy may be given either alone or along with radiation therapy. Higher dosing is used when chemotherapy is given alone. When combined with radiation therapy, chemotherapy is delivered at a lower dose. Using lower doses of chemotherapy may make the cancer more sensitive to the radiation.

A variety of chemotherapy drugs may be used to treat leukemia. The type and combinations of drugs that are used to treat leukemia will vary depending on the type of leukemia you have, your age, and overall health. Chemotherapy for leukemia is usually given through an IV, but some forms can be given by mouth. It is delivered in cycles over a set period of time. A medical oncologist will determine how many cycles of chemotherapy are needed and what combination of drugs will work best.

Some leukemias, mainly acute leukemias, have a higher risk of spreading to the cerebrospinal fluid (CSF) found around the brain and spinal cord. If leukemia is found in the CSF, or is at a high risk of spreading to this area, chemotherapy may be delivered directly into the CSF.

Three phases of chemotherapy are used to treat acute leukemia:

  • Induction—To bring about a complete remission.
  • Consolidation—To kill any leukemia cells still in the body after complete remission has been achieved and reduce the chances of cancer coming back.
  • Maintenance—To prevent cancer from coming back once complete remission has been achieved.

Chemotherapy may not be given for chronic leukemias until it has progressed. Progression may include rapidly increasing cell counts, increase in immature cells in the blood, or other significant symptoms.

Side Effects and Management

Though the drugs are targeted to cancer cells, they can affect healthy cells as well. The death of cancer cells and impact on healthy cells can cause a range of side effects. A medical oncologist will work to find the best drug combination and dosage to have the most impact on the cancer cells and minimal side effects on healthy tissue. Side effects or complications from chemotherapy may include:

  • Nausea and vomiting
  • Fatigue
  • Hair loss
  • Irritation to the tube that connects the mouth to the stomach—esophagitis
  • Infertility and/or premature menopause—If you plan on having children, talk to your doctor about options to preserve fertility before starting treatment. This also applies to adolescents who have gone through puberty.

A variety of treatments are available to help manage side effects including medication, lifestyle changes, and alternative treatments. In some cases, the chemotherapy regimen may be adjusted to reduce severe side effects. The earlier the side effects are addressed, the more likely they will be controlled with a minimum of discomfort.

Long-term side effects can include damage to the heart and peripheral nerves, some cognitive dysfunction, and, very rarely, development of other cancers, including other leukemias.


Acute leukemia overview. Merck Manual Professional Version website. Available at: Updated October 2014. Accessed February 4, 2016.

Acute lymphoblastic leukemia/lymphoma (ALL) management. EBSCO DynaMed website. Available at: Updated February 20, 2015. Accessed February 4, 2016.

Initial management of acute myeloid leukemia (AML). EBSCO DynaMed website. Available at: Updated January 22, 2016. Accessed February 4, 2016.

Initial management of chronic lymphocytic leukemia (CLL). EBSCO DynaMed website. Available at: Updated January 11, 2016. Accessed February 4, 2016.

Management of chronic myeloid leukemia in accelerated or blast phase. EBSCO DynaMed website. Available at: Updated April 17, 2015. Accessed February 4, 2016.

Management of chronic myeloid leukemia in chronic phase. EBSCO DynaMed website. Available at: Updated March 12, 2015. Accessed February 4, 2016.

Management of relapsed or refractory chronic lymphocytic leukemia (CLL). EBSCO DynaMed website. Available at: Updated November 5, 2015. Accessed February 4, 2016.

Last reviewed December 2015 by Mohei Abouzied, MD

Please be aware that this information is provided to supplement the care provided by your physician. It is neither intended nor implied to be a substitute for professional medical advice. CALL YOUR HEALTHCARE PROVIDER IMMEDIATELY IF YOU THINK YOU MAY HAVE A MEDICAL EMERGENCY. Always seek the advice of your physician or other qualified health provider prior to starting any new treatment or with any questions you may have regarding a medical condition.

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