busulfan

Overview

Busulfan is a bifunctional alkylating agent that cross-links DNA strands, inducing apoptosis. It has a unique tropism for myeloid precursors, making it a cornerstone of myeloablative conditioning regimens for allogeneic hematopoietic stem-cell transplantation (allo-SCT) in AML and MDS, often combined with cyclophosphamide (Bu/Cy) or fludarabine (Bu/Flu).

Evidence in the corpus

  • Referenced as a component of high-dose myeloablative conditioning for allogeneic SCT — the treatment modality associated with the largest survival benefit in the WashU decitabine AML/MDS trial (NCT01687400, N=116); allogeneic SCT vs no SCT was the dominant Cox survival predictor (P<0.001), and benefit was not adversely affected by TP53 status PMID:27959731
  • Component of the myeloablative high-dose chemotherapy conditioning in a phase II trial (NCT00588523) for newly diagnosed 1p/19q-codeleted anaplastic oligodendroglioma (ODG3): busulfan 3.2 mg/kg/day IV × 3 days (days −5 to −3) preceded PBSC reinfusion; 21 patients received HDC-ASCT with no toxic deaths; median neutrophil engraftment 9 days (range 8–12). PMID:28472509

Resistance mechanisms

  • Not specifically reported in the corpus.

Cancer types (linked)

  • AML — myeloablative conditioning for allo-SCT.
  • MDS — conditioning for allo-SCT as consolidation after HMA response.
  • ODG3 — HDC-ASCT conditioning (busulfan + thiotepa) in 1p/19q-codeleted anaplastic oligodendroglioma; well tolerated with no toxic deaths.

Sources

  • PMID:27959731 — Welch et al. 2016, NEJM. WashU AML/MDS decitabine trial; busulfan referenced as SCT conditioning context; SCT was the dominant survival predictor.
  • PMID:28472509 — Thomas et al. 2017, J Clin Oncol. Phase II NCT00588523; busulfan 3.2 mg/kg/day × 3 as myeloablative conditioning in HDC-ASCT for 1p/19q-codeleted anaplastic oligodendroglioma; no toxic deaths.

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