Long-term event-free and overall survival after risk-adapted melphalan and SCT for systemic light chain amyloidosis

Leukemia. 2017 Jan;31(1):136-142. doi: 10.1038/leu.2016.229. Epub 2016 Aug 18.

Abstract

Stem cell transplantation (SCT), an effective therapy for amyloid light chain (AL) amyloidosis patients, is associated with low treatment-related mortality (TRM) with appropriate patient selection and risk-adapted dosing of melphalan (RA-SCT). Consolidation after SCT increases hematologic complete response (CR) rates and may improve overall survival (OS) for patients with <CR. We retrospectively analyzed outcomes for 143 patients who underwent RA-SCT with or without consolidation. Melphalan was administered at 100 (14%), 140 (52%) and 200 (34%) mg/m2. The TRM rate at 100 days was 5%. RA-SCT resulted in CR in 24% (3 months) and 48% (12 months) of patients. The CR rate was particularly high (62%) in patients offered bortezomib consolidation. With a median follow-up among survivors of 7.7 years, median event-free survival (EFS) with RA-SCT was 4.04 years (95% confidence interval (CI): 3.41-5.01 years); median OS was 10.4 years (95% CI: 7.3-not achieved). Patients with CR at 12 months after SCT had significantly longer EFS (P=0.01) and OS (P=0.04). In a multivariate analysis, melphalan dose had no impact on EFS (P=0.26) or OS (P=0.11). For selected patients, RA-SCT was safe and was associated with extended long-term survival. With the availability of novel agents for consolidation, RA-SCT remains a very effective and important backbone treatment for AL amyloidosis.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, N.I.H., Extramural

MeSH terms

  • Amyloidosis / drug therapy*
  • Amyloidosis / mortality*
  • Amyloidosis / therapy
  • Bortezomib / therapeutic use
  • Consolidation Chemotherapy / methods
  • Disease-Free Survival
  • Follow-Up Studies
  • Humans
  • Immunoglobulin Light Chains
  • Melphalan / administration & dosage*
  • Middle Aged
  • Remission Induction
  • Retrospective Studies
  • Risk Adjustment
  • Stem Cell Transplantation
  • Survival Rate

Substances

  • Immunoglobulin Light Chains
  • Bortezomib
  • Melphalan