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Brand names:

Busulfex®, Myleran®

Other names:

Bussulfam, Busulfanum, Busulphan

Often used for:

Leukemia, Conditioning for hematopoietic cell transplant

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About Busulfan

Busulfan is given to prepare patients for a hematopoietic cell transplant (also called a bone marrow transplant or a stem cell transplant). It works by slowing or stopping the growth of cells.

Patients will have regular blood draws to check blood counts and monitor liver function. Blood levels of busulfan may be monitored to see whether the dose should be changed.

Busulfan can cause lung damage. Chest X-rays and breathing tests may be used to check for lung problems.

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May be taken as a tablet by mouth

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May be given as a liquid into a vein by IV

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May be taken as a liquid by mouth

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Possible Side Effects

  • Low blood counts (may cause increased risk of infection, bleeding, anemia, and/or fatigue)
  • Seizures (while receiving the medicine and up to 3 days after taking the medicine)
  • Change in skin color (darkening of skin)
  • Liver problems (called veno-occlusive disease)
  • Nausea and vomiting
  • Loss of appetite and weight loss
  • Diarrhea
  • Constipation
  • Mouth sores
  • Dry mouth
  • Fatigue or weakness

Not all patients who take busulfan will experience these side effects. Common side effects are in bold, but there may be others. Please report all suspected side effects to your doctor or pharmacist.

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Possible Late Effects

Some patients may experience long-term or late effects of treatment that may continue or develop months or years after treatment ends. Possible late effects due to busulfan include:

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Tips for Families

Be sure to discuss these and other recommendations with your doctor or pharmacist.

  • A doctor may prescribe medicines to prevent seizures and medicines to reduce nausea and vomiting.
  • Sexually active patients should take steps to prevent pregnancy during treatment and for 6 months after completion of therapy.
  • Patients should tell the doctor if they are pregnant or breastfeeding.
  • Caregivers should follow instructions for safe handling and disposal of the medicine and avoid contact with patient body fluids, which can contain the drug for 48 hours after it is given.

Busulfan at home:

  • Take busulfan at about the same time every day.
  • Give a missed dose as soon as possible. If it is near the time for the next dose, skip the dose. Do not give 2 doses at the same time. 
  • Store tablets at room temperature.
  • Store liquid busulfan in the refrigerator.
  • When giving liquid busulfan, shake the bottle for at least 30 seconds before filling the oral syringe. Wash the syringe thoroughly after each use.
  • Do not use the medicine past the expiration date.
  • Follow instructions for safe handling and disposal.