Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation in Sweden, long-term results from all patients treated in 1994-2009

Bone Marrow Transplant. 2016 Dec;51(12):1569-1572. doi: 10.1038/bmt.2016.249. Epub 2016 Oct 3.

Abstract

High-dose melphalan and autologous stem cell transplantation (HDM/ASCT) is widely used in immunoglobulin light chain (AL) amyloidosis, but the benefit is debated mainly because of the high treatment-related mortality (24% in a randomised study comparing HDM/ASCT with oral melphalan/dexamethasone). We report here on the long-term outcome of all patients treated with HDM/ASCT for AL amyloidosis in Sweden between 1994 and 2009. Seventy-two patients were treated at eight Swedish centres. Median follow-up was 67.5 months. At least partial response (organ or haematological) was seen in 64% of the patients. Median overall survival was 98 months or 8.2 years, with 5-year survival 63.9% and 10-year survival 43.4%. In patients with cardiac involvement or multiple organ involvement, survival was significantly shorter, median overall survival 49 and 56 months, respectively. All mortality within 100 days from ASCT was 12.5% for all patients and 17.2% in the patients with cardiac involvement. For patients treated in the earlier time period (1994-2001), 100-day mortality was 23.8% compared with 7.8% in the later period (2002-2009). In conclusion, long survival times can be achieved in patients with AL amyloidosis treated with HDM/ASCT, also in smaller centres. Early mortality is high, but with a decreasing trend over time.

Publication types

  • Multicenter Study

MeSH terms

  • Adult
  • Disease-Free Survival
  • Female
  • Follow-Up Studies
  • Heart Diseases
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Immunoglobulin Light-chain Amyloidosis / mortality
  • Immunoglobulin Light-chain Amyloidosis / pathology
  • Immunoglobulin Light-chain Amyloidosis / therapy*
  • Male
  • Melphalan / administration & dosage*
  • Middle Aged
  • Multiple Organ Failure
  • Survival Rate
  • Sweden
  • Time-to-Treatment
  • Transplantation, Autologous
  • Treatment Outcome

Substances

  • Melphalan