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At a glance

Busulphan is an anti-cancer medication used in the treatment of chronic myeloid leukemia (CML) and as a conditioning regimen prior to hematopoietic progenitor cell transplantation (HPCT). It works by damaging the genetic material (DNA and RNA) of cancer cells, stopping their growth and multiplication.

Indications
  • Chronic myeloid leukemia (CML) - palliative treatment
  • Conditioning regimen prior to hematopoietic progenitor cell transplantation (HPCT)
  • Polycythemia vera
  • Essential thrombocythemia
Pharmacology

Busulphan reacts with the N-7 position of guanosine and creates intra- and inter-strand crosslinks between DNA strands. This disrupts DNA replication and RNA transcription, leading to cell cycle arrest and cancer cell death.

Dosage

Chronic Myeloid Leukemia (CML) - Palliative: 60 mcg/kg/day. Maintenance: 0.5-2 mg/day. Max: 4 mg/day.

Polycythemia Vera: 4-6 mg/day for 4-6 weeks (with blood count monitoring).

Essential Thrombocythemia: 2-4 mg/day.

Bone Marrow Transplantation Conditioning: 3.5-4 mg/kg/day in divided doses for 4 days, total 14-16 mg/kg. Usually used with cyclophosphamide.

Hepatic impairment: Limited information; caution required.

Renal impairment: Probably safe; dose adjustment may not be needed.

Side effects

Common side effects: Decreased blood cells (red cells, white cells, platelets), abdominal pain, anxiety, nausea, vomiting, loss of appetite, decreased magnesium, decreased potassium, diarrhea, fever, headache, increased glucose, insomnia, stomatitis (mouth inflammation).

Serious: Bone marrow suppression, pulmonary fibrosis (with long-term use), adrenal insufficiency, hyperpigmentation, veno-occlusive disease (VOD), seizures.

Use in pregnancy

Unsafe during pregnancy (Category D). Definite evidence of risk to the developing baby. May be prescribed rarely in life-threatening situations if benefits outweigh risks.

Pregnancy Category: D — always consult a doctor before taking during pregnancy.

Precautions & warnings
  • Busulphan can lower blood clotting ability and infection resistance; seek immediate medical attention if unusual bruising/bleeding or signs of infection (fever, chills, body aches) occur.
  • Inform your doctor if you have thalassemia, gout, or history of radiotherapy.
  • Do not receive live vaccines; avoid contact with individuals who have recently received live vaccines.
  • Do not use during pregnancy and lactation (Category D).
  • Use effective contraception during and after treatment.
  • Regular monitoring of CBC, kidney, liver, and cardiac function is required.
  • Inform your doctor if you develop dry cough or shortness of breath (pulmonary toxicity).
Overdose effects

Overdose symptoms: Bone marrow suppression (severe pancytopenia), pulmonary fibrosis, seizures, veno-occlusive disease. Consult your doctor immediately if overdose is suspected.

Storage conditions

Store below 30°C, away from light and moisture. Keep out of reach of children.

Busulphan — see the full informationPharmacology, precautions, pregnancy, overdose and the rest on the generic page
⚠️ This information is for educational purposes only — not a substitute for medical advice. Consult a registered physician before taking any medicine.