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Alkylating agent (specialist)

Busulfan

Brand names: Myleran, Busilvex

Busulfan is an alkylating cytotoxic agent used predominantly as part of conditioning regimens before haematopoietic stem cell transplantation and in some myeloproliferative disorders.

Dosing — being independently re-sourced

ClinCalc Pro is rebuilding its dose data from primary open sources — the manufacturer SmPC (eMC), the WHO Model Formulary and other official references — under clinician review. This drug's structured dose is not yet published here. Confirm all doses against the product SmPC and your local formulary before prescribing.

Clinical monograph

How it works

It alkylates and cross-links DNA, interfering with replication and transcription and producing profound suppression of bone marrow stem cells.

Prescribing in practice

  • It causes profound, prolonged myelosuppression and can cause hepatic veno-occlusive disease (sinusoidal obstruction syndrome), so it is used only under specialist transplant supervision.
  • Seizure prophylaxis is required with high-dose conditioning regimens because busulfan lowers the seizure threshold.
  • Pulmonary fibrosis, infertility and a risk of secondary malignancy are recognised long-term toxicities.

Monitoring

Monitor the full blood count closely along with liver function, and observe for hepatic veno-occlusive disease and seizures during conditioning.

Counselling the patient

  • Report fever, bleeding, breathlessness or right upper abdominal pain and jaundice promptly.
  • Treatment can cause long-term infertility; discuss fertility preservation beforehand where relevant.
  • Effective contraception is required during and for a period after treatment.

Evidence & guidelines

Its role in transplant conditioning is long-established in haematology practice and supported by extensive clinical experience and the SPC.

Reference: BSH HSCT; ESMO; SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. Monograph status: clinician-reviewed (2026-07-04).

Related

Curated clinical cross-links plus same-class fallbacks.

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