title: High-dose melphalan and autologous stem-cell transplantation in patients with AL amyloidosis: an 8-year study.
abstract: BACKGROUND
  AL amyloidosis is a fatal disease resulting from tissue deposition of amyloid fibrils derived from monoclonal immunoglobulin light chains. Treatment with oral chemotherapy is minimally effective.
  
  
  OBJECTIVE
  To test survival and organ response in a large sample of patients treated with high-dose intravenous melphalan (100 to 200 mg/m2) and autologous blood stem-cell transplantation.
  
  
  METHODS
  8-year longitudinal analysis of clinical effectiveness.
  
  
  METHODS
  University-affiliated specialty referral clinic.
  
  
  METHODS
  701 consecutive new patients with AL amyloidosis.
  
  
  METHODS
  High-dose chemotherapy and autologous stem-cell transplantation for patients who met eligibility requirements based on organ involvement and clinical status.
  
  
  METHODS
  Survival analysis of all patients evaluated and a detailed analysis of treatment outcome in the subgroup that received high-dose melphalan and stem-cell transplantation.
  
  
  RESULTS
  Among 701 patients with AL amyloidosis, 394 (56%) were eligible for high-dose melphalan and stem-cell transplantation; 82 did not proceed with treatment because of patient choice or disease progression. Median survival of the 312 patients who initiated treatment was 4.6 years. A complete hematologic response, defined as no evidence of an underlying plasma cell dyscrasia 1 year after treatment, was achieved in 40% of patients and was associated with prolonged survival. Statistically significant improvements occurred in end-organ disease and were greater in patients with a complete hematologic response. Mortality rate within 100 days of treatment with high-dose melphalan and stem-cell transplantation was 13%; patients with cardiomyopathy had the highest mortality rates.
  
  
  CONCLUSIONS
  Treatment of selected patients with AL amyloidosis by using high-dose melphalan and stem-cell transplantation resulted in hematologic remission, improved 5-year survival, and reversal of amyloid-related disease in a substantial proportion.
authors.affiliation: Boston University School of Medicine, Boston, Massachusetts 02118, USA. mskinner@bu.edu
authors.forename: Martha
authors.initials: M
authors.lastname: Skinner
authors.forename: Vaishali
authors.initials: V
authors.lastname: Sanchorawala
authors.forename: David C
authors.initials: DC
authors.lastname: Seldin
authors.forename: Laura M
authors.initials: LM
authors.lastname: Dember
authors.forename: Rodney H
authors.initials: RH
authors.lastname: Falk
authors.forename: John L
authors.initials: JL
authors.lastname: Berk
authors.forename: Jennifer J
authors.initials: JJ
authors.lastname: Anderson
authors.forename: Carl
authors.initials: C
authors.lastname: O'Hara
authors.forename: Kathleen T
authors.initials: KT
authors.lastname: Finn
authors.forename: Caryn A
authors.initials: CA
authors.lastname: Libbey
authors.forename: Janice
authors.initials: J
authors.lastname: Wiesman
authors.forename: Karen
authors.initials: K
authors.lastname: Quillen
authors.forename: Niall
authors.initials: N
authors.lastname: Swan
authors.forename: Daniel G
authors.initials: DG
authors.lastname: Wright
mesh_terms: D000368:Aged; D000686:Amyloidosis / Q000097:blood / Q000150:complications / Q000401:mortality / Q000628:therapy*; D005260:Female; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D008875:Middle Aged; D033581:Stem Cell Transplantation; D016019:Survival Analysis; D014182:Transplantation, Autologous
chemical_list: D008558:Melphalan