busulfan at a glance
Medicine class
Alkylating cytotoxic chemotherapy
Main role
Conditioning before a blood stem-cell transplant
Available forms
Tablets and a concentrate diluted for intravenous infusion
Important monitoring
Blood counts and liver function require close monitoring
What is busulfan used for?
Licensed uses represented by the selected UK products include:
Conditioning before haematopoietic progenitor-cell transplantation in adults and children, alongside other specified conditioning medicines.
Palliative treatment of chronic-phase chronic myeloid leukaemia using tablets.
Producing remission in polycythaemia vera, particularly when thrombocytosis is marked.
Selected cases of essential thrombocythaemia or myelofibrosis using tablets.
How does busulfan work?
Busulfan forms reactive molecules that attach to DNA. This disrupts cell replication and strongly reduces bone-marrow activity, helping remove diseased or existing marrow cells before transplanted stem cells are given.
busulfan preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 6 mg/mL
Tablet
Route: Oral
Strengths: 2 mg
How to take busulfan
Busulfan must be used under specialist supervision.
Swallow tablets whole with water; do not break, crush or chew them.
The infusion concentrate is diluted and administered by healthcare professionals through a central venous catheter. It must not be given as a rapid injection or through a peripheral vein.
Anti-sickness treatment and seizure prevention are commonly provided with transplant-conditioning busulfan.
Follow the treatment label or hospital schedule exactly and check uncertainties with the specialist team.
busulfan side effects
Common or expected effects
- Reduced white cells, red cells and platelets
- Infections, fever, chills and weakness
- Nausea, vomiting, diarrhoea and mouth soreness
- Loss of appetite and changes in blood electrolytes
- Headache, dizziness or sleep disturbance
- Rash, itching or hair loss
- Raised liver tests or jaundice
- Menstrual, fertility or sperm-production problems
Get urgent medical advice
- Severe infection or profound bone-marrow suppression
- Hepatic sinusoidal obstruction syndrome, including jaundice, abdominal swelling or pain and rapid weight gain
- Seizures
- Serious lung toxicity causing cough, breathlessness or low oxygen
- Severe bleeding or aplastic anaemia
- Thrombotic microangiopathy after transplantation
- Severe allergic reaction
- A later secondary cancer
busulfan in pregnancy
Intravenous busulfan used for transplant conditioning is contraindicated during pregnancy, while tablet use should be avoided whenever possible. Busulfan may harm a developing baby, so pregnancy, pregnancy planning and contraception must be discussed with the specialist team before treatment. It may also permanently impair fertility in any sex.
busulfan while breastfeeding
Breastfeeding should stop during busulfan treatment. It is unknown whether busulfan enters human milk, and serious toxicity is possible; specialist advice is needed about any future return to breastfeeding.
busulfan interactions
The transplant or haematology team should review prescribed, pharmacy-bought and complementary products before busulfan is given.
Itraconazole or metronidazole
These may reduce busulfan clearance and increase exposure and toxicity; close monitoring is required.
Deferasirox
Busulfan exposure may increase, so plasma concentrations may require additional monitoring.
Common questions about busulfan
Answers are fully visible for fast scanning and source review.
Is busulfan chemotherapy?
Yes. It is an alkylating cytotoxic chemotherapy that damages DNA and suppresses bone-marrow activity.
Why is busulfan given before a stem-cell transplant?
It is used as conditioning to remove existing marrow cells and suppress immunity so transplanted stem cells can establish themselves.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.