High-dose melphalan and autologous hematopoietic stem cell transplantation in primary amyloidosis: single-center results

Transplant Proc. 2014 Oct;46(8):2877-81. doi: 10.1016/j.transproceed.2014.09.053.

Abstract

Background: Systemic immunoglobulin light-chain amyloidosis (AL) is a plasma cell dyscrasia resulting in multisystem organ failure and death. Autologous hematopoietic stem-cell transplantation (ASCT) has been widely used to treat patients with AL. However, treatment-related mortality remains high and reported series are subject to selection bias.

Methods: To define the role of patient selection in stem cell transplantation, we evaluated 24 consecutive AL patients transplanted at our center.

Results: Complete hematologic response was achieved in all 20 patients surviving >100 days posttransplantation. The 1-year overall survival (OS) rate after ASCT was 78.5%. The 5- and 10-year progression-free and OS rates were 57% and 47%, respectively. Treatment-related deaths owing to cardiovascular problems occurred in 16% of cases.

Conclusion: ASCT for AL amyloidosis can be safely performed in experienced transplantation centers, and increased risk is associated mainly with cardiovascular system involvement.

MeSH terms

  • Adult
  • Aged
  • Amyloidosis / drug therapy*
  • Amyloidosis / surgery*
  • Female
  • Hematopoietic Stem Cell Transplantation* / mortality
  • Humans
  • Immunoglobulin Light-chain Amyloidosis
  • Male
  • Melphalan / administration & dosage*
  • Melphalan / therapeutic use
  • Middle Aged
  • Myeloablative Agonists / administration & dosage*
  • Patient Selection
  • Retrospective Studies
  • Survival Analysis
  • Transplantation, Autologous

Substances

  • Myeloablative Agonists
  • Melphalan