High-dose melphalan and autologous stem cell transplantation for AL amyloidosis: recent trends in treatment-related mortality and 1-year survival at a single institution

Amyloid. 2011 Jun;18 Suppl 1(Suppl 1):127-9. doi: 10.3109/13506129.2011.574354047.

Abstract

Treatment with high-dose melphalan chemotherapy supported by hematopoietic rescue with autologous stem cells produces high rates of hematologic responses and improvement in survival and organ function for patients with AL amyloidosis. Ongoing clinical trials explore pre-transplant induction regimens, post-transplant consolidation or maintenance approaches, and compare transplant to non-transplant regimens. To put these studies into context, we reviewed our recent experience with transplant for AL amyloidosis in the Amyloid Treatment and Research Program at Boston Medical Center and Boston University School of Medicine. Over the past 10 years, there was a steady reduction in rates of treatment-related mortality and improvement in 1-year survival, now approximately 5% and 90%, respectively, based upon an intention-to-treat analysis. Median overall survival of patients treated with this approach at our center exceeds 7.5 years.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Amyloidosis / drug therapy
  • Amyloidosis / immunology
  • Amyloidosis / physiopathology
  • Amyloidosis / surgery
  • Amyloidosis / therapy*
  • Combined Modality Therapy
  • Dose-Response Relationship, Drug
  • Humans
  • Immunoglobulin Light Chains*
  • Stem Cell Transplantation*
  • Survival Rate
  • Transplantation, Autologous

Substances

  • Immunoglobulin Light Chains