Busulfan, melphalan, and bortezomib compared to melphalan as a high dose regimen for autologous hematopoietic stem cell transplantation in multiple myeloma: long term follow up of a novel high dose regimen

Leuk Lymphoma. 2020 Dec;61(14):3484-3492. doi: 10.1080/10428194.2020.1811275. Epub 2020 Aug 31.

Abstract

Melphalan at a dose of 200 mg/m2 (MEL200) remains the standard high dose therapy before autologous stem cell transplantation (ASCT) for multiple myeloma (MM). Intensifying the high dose regimen has shown promising results. We report here 7-year follow up of our novel high dose regimen of busulfan and melphalan followed by bortezomib (BuMelVel). Forty-three MM patients received BuMelVel high dose therapy with pharmacokinetic adjusted busulfan. Outcomes were compared to a matched control cohort from the CIBMTR database (n = 162) receiving MEL200. The primary endpoint was progression free survival. Five year PFS was 47% v 30% (95% CI; 32-62) in favor or the BuMelVel group (95% CI; 23-37) (p = 0.05). In multivariate analysis for PFS, BuMelVel (HR 0.65; 95% CI 0.44-0.97)(p = 0.036) was predictive. Similar to recent reports of double alkylator therapy, although depth of response was similar between the BuMelVel group and MEL200, the BUMELVEL group experienced an improved PFS.

Keywords: Multiple myeloma; autologous stem cell transplantation; bortezomib; busulfan; melphalan.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Bortezomib / therapeutic use
  • Busulfan / therapeutic use
  • Follow-Up Studies
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Melphalan / therapeutic use
  • Multiple Myeloma* / drug therapy
  • Transplantation Conditioning
  • Transplantation, Autologous

Substances

  • Bortezomib
  • Busulfan
  • Melphalan