A second course of high-dose melphalan and auto-SCT for the treatment of relapsed AL amyloidosis

Bone Marrow Transplant. 2011 Jul;46(7):976-80. doi: 10.1038/bmt.2010.239. Epub 2010 Oct 18.

Abstract

High-dose melphalan and auto-SCT (HDM/SCT) induces hematological complete responses (HCRs) in 40% of patients with immunoglobulin light chain (AL) amyloidosis. However, relapses occur in 8% of patients who initially achieve HCR. We conducted a study to explore the feasibility and efficacy of a second HDM/SCT in this setting. Eleven patients were enrolled. Five patients underwent repeat stem cell mobilization with G-CSF; the others had stem cells cryopreserved from the first mobilization. Six patients received 200 mg/m(2) HDM; five patients received modified HDM at 140 mg/m(2). Engraftment occurred at a median of 10 days for neutrophils and 12 days for platelets. There was no treatment-related mortality or death within the first year, but significant grade III/IV non-hematological toxicities occurred. In all, 4 of 11 patients (36%) achieved HCR at 1 year. Two of these patients are in continuous remission at 3 and 6 years; the other two relapsed at 2 and 3 years. Of the four patients who achieved partial hematological response at 1 year, three have relapsed at a median of 3 years. Three patients died of progressive disease at 1-2 years. In conclusion, one-third of patients with AL amyloidosis who relapse after HDM/SCT can achieve HCR with a second SCT.

Publication types

  • Clinical Trial

MeSH terms

  • Adult
  • Amyloidosis / drug therapy
  • Amyloidosis / surgery
  • Amyloidosis / therapy*
  • Cohort Studies
  • Disease-Free Survival
  • Female
  • Hematopoietic Stem Cell Mobilization / methods
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Immunoglobulin Light-chain Amyloidosis
  • Kaplan-Meier Estimate
  • Male
  • Melphalan / administration & dosage*
  • Middle Aged
  • Recurrence
  • Survival Analysis

Substances

  • Melphalan