Busulfan is used to treat CML (chronic myelogenous leukemia), and is used because of its low cost. The gold standard of treatment for CML, now, is imatinib.
This drug is used to ablate patient's bone marrow, and is indicated as a conditioning agent prior to bone marrow transplantation, especially patients with CML.
Busulfan is an alkylating agent that forms DNA-DNA intrastrand crosslinks. This prevents DNA replication, as these crosslinks cannot be repaired by cellular machinery, causing the cancer cell to undergo apoptosis.
This drug leads to severe myelosuppression, hence its use as an ablation agent.
The most notable toxicity of this medication is interstitial pulmonary fibrosis, leading to the term "Busulfan lung."
Patients can develop skin hyperpigmentation with the use of Busulfan. The presentation of this skin manifestation mimics Addison disease, affecting the joints and skin creases as bronzing.
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