Busulfan

Summary

Busulfan is an alkylating agent that works by cross-linking DNA strands. It is cell cycle nonspecific, meaning it can target and damage cells in all phases of the cell cycle. Its main clinical use is for bone marrow ablation before a donor bone marrow transplant.

Busulfan causes severe myelosuppression in nearly all patients. While this is the intended effect for marrow ablation, it can also lead to dangerously low blood counts. Other potential side effects include pulmonary fibrosis and skin hyperpigmentation.

Key Points

• Busulfan
  • Mechanisms
    • Alkylating agent
      • Cross-links DNA
    • Cell-cycle nonspecific
  • Clinical Use
    • Used to ablate patient’s bone marrow before bone marrow transplantation
    • CML
      • Historically was the standard treatment for CML before imatinib was discovered; now is less preferred vs. more targeted therapies
  • Side Effects
    • Severe myelosuppression (occurs in almost all cases)
      • This also happens to be the goal of bone marrow ablation
    • Pulmonary fibrosis
    • Hyperpigmentation