Revisiting conditioning dose in newly diagnosed light chain amyloidosis undergoing frontline autologous stem cell transplant: impact on response and survival

Bone Marrow Transplant. 2017 Aug;52(8):1126-1132. doi: 10.1038/bmt.2017.68. Epub 2017 Apr 10.

Abstract

Autologous stem cell transplantation (ASCT) is an important treatment modality in light chain (AL) amyloidosis. Use of reduced-dose melphalan conditioning is common, given the associated organ and functional decline. The impact of full-intensity melphalan conditioning (n=314) was compared to reduced-dose conditioning (n=143). Patients in the full-intensity group were younger, with better performance status, fewer involved organs, lower tumor burden and lower Mayo stage. Full-dose conditioning was associated with higher rate of very good partial response or better (79% vs 62%; P<0.001), complete response rate (53% vs 37%; P=0.003) and organ response rate (74% vs 59%; P=0.002) as compared to reduced-dose conditioning. PFS was superior in the full-intensity group compared to the reduced-dose group (4-year PFS 55% vs 31%; P<0.001) as well as a longer overall survival (OS) 4-year OS (86% vs 54%; P<0.001). In addition, the OS and PFS were significantly lower in the reduced-dose group compared to the full-intensity group in Mayo stage III/IV as well as stage I/II. A multivariate analysis confirmed an independent impact for conditioning dose on PFS/OS. This study calls for re-assessment of the use of reduced-dose conditioning in ASCT for AL amyloidosis.

MeSH terms

  • Aged
  • Amyloidosis / mortality
  • Amyloidosis / therapy*
  • Dose-Response Relationship, Drug
  • Female
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cell Transplantation / mortality
  • Humans
  • Immunoglobulin Light Chains
  • Male
  • Melphalan / administration & dosage*
  • Middle Aged
  • Remission Induction
  • Retrospective Studies
  • Survival Analysis
  • Transplantation Conditioning / methods*
  • Transplantation Conditioning / mortality
  • Transplantation, Autologous
  • Treatment Outcome

Substances

  • Immunoglobulin Light Chains
  • Melphalan