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Generic NameBrand Name
busulfanMyleran, Busulfex

Busulfan is available as a pill you can swallow. It is also available in an IV form, used for stem cell transplants.

How It Works

Busulfan is classified as an alkylating agent. It kills cells by interfering with the way they multiply.

Why It Is Used

Busulfan is used to lower the number of blood cells in people who have diseases such as leukemia and non-Hodgkin's lymphoma.

How Well It Works

Busulfan may help to gradually lower the blood counts for older people who have chronic myelogenous leukemia (CML) but who cannot take other medicines or who are unable to maintain close and frequent medical supervision.1

Side Effects

Side effects are common with busulfan and may include:

  • Thinning or brittleness of the hair.
  • Darkening and drying of the skin.
  • Loss of appetite or weight loss.
  • Diarrhea.
  • Weakness and fatigue.
  • Mouth sores (stomatitis).
  • Low blood counts, which may make you tired and more likely to get an infection. Your blood counts may stay low for a long time.

See Drug Reference for a full list of side effects. (Drug Reference is not available in all systems.)

What To Think About

Busulfan should be used only under the supervision of a medical oncologist or hematologist.

You may not be able to become pregnant or father a child after taking busulfan. Talk to your doctor about this before starting treatment.

Busulfan can cause birth defects. Do not use this drug if you are pregnant or wish to become pregnant or father a child while you are taking it.

Women who take this drug may experience symptoms of menopause, including hot flashes and vaginal dryness.

Complete the new medication information form (PDF) (What is a PDF document?) to help you understand this medication.



  1. Liesveld JL, Lichtman MA (2010). Chronic myelogenous leukemia and related disorders. In K Kaushanksy et al., eds., Williams Hematology, 8th ed., pp. 1331–1379. New York: McGraw-Hill.


By Healthwise Staff
Primary Medical Reviewer Kathleen Romito, MD - Family Medicine
Primary Medical Reviewer Anne C. Poinier, MD - Internal Medicine
Specialist Medical Reviewer Brian Leber, MDCM, FRCPC - Hematology
Last Revised February 9, 2011

This information does not replace the advice of a doctor. Healthwise, Incorporated disclaims any warranty or liability for your use of this information.