title: High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis.
abstract: BACKGROUND
  High-dose chemotherapy followed by autologous hematopoietic stem-cell transplantation has been reported to provide higher response rates and better overall survival than standard chemotherapy in immunoglobulin-light-chain (AL) amyloidosis, but these two strategies have not been compared in a randomized study.
  
  
  METHODS
  We conducted a randomized trial comparing high-dose intravenous melphalan followed by autologous hematopoietic stem-cell rescue with standard-dose melphalan plus high-dose dexamethasone in patients with AL amyloidosis. Patients (age range, 18 to 70 years) with newly diagnosed AL amyloidosis were randomly assigned to receive intravenous high-dose melphalan plus autologous stem cells or oral melphalan plus oral high-dose dexamethasone.
  
  
  RESULTS
  Fifty patients were enrolled in each group. The results were analyzed on an intention-to-treat basis, with overall survival as the primary end point. After a median follow-up of 3 years, the estimated median overall survival was 22.2 months in the group assigned to receive high-dose melphalan and 56.9 months in the group assigned to receive melphalan plus high-dose dexamethasone (P=0.04). Among patients with high-risk disease, overall survival was similar in the two groups. Among patients with low-risk disease, there was a nonsignificant difference between the two groups in overall survival at 3 years (58% in the group assigned to receive high-dose melphalan vs. 80% in the group assigned to receive melphalan plus high-dose dexamethasone; P=0.13).
  
  
  CONCLUSIONS
  The outcome of treatment of AL amyloidosis with high-dose melphalan plus autologous stem-cell rescue was not superior to the outcome with standard-dose melphalan plus dexamethasone. (ClinicalTrials.gov number, NCT00344526 [ClinicalTrials.gov].).
authors.affiliation: Department of Hematology, Centre Hospitalier Universitaire, Université et Centre National de la Recherche Scientifique, UMR 6101, Limoges, France. arnaud.jaccard@chu-limoges.fr
authors.forename: Arnaud
authors.initials: A
authors.lastname: Jaccard
authors.forename: Philippe
authors.initials: P
authors.lastname: Moreau
authors.forename: Veronique
authors.initials: V
authors.lastname: Leblond
authors.forename: Xavier
authors.initials: X
authors.lastname: Leleu
authors.forename: Lotfi
authors.initials: L
authors.lastname: Benboubker
authors.forename: Olivier
authors.initials: O
authors.lastname: Hermine
authors.forename: Christian
authors.initials: C
authors.lastname: Recher
authors.forename: Bouchra
authors.initials: B
authors.lastname: Asli
authors.forename: Bruno
authors.initials: B
authors.lastname: Lioure
authors.forename: Bruno
authors.initials: B
authors.lastname: Royer
authors.forename: Fabrice
authors.initials: F
authors.lastname: Jardin
authors.forename: Frank
authors.initials: F
authors.lastname: Bridoux
authors.forename: Bernard
authors.initials: B
authors.lastname: Grosbois
authors.forename: Jérome
authors.initials: J
authors.lastname: Jaubert
authors.forename: Jean-Charles
authors.initials: JC
authors.lastname: Piette
authors.forename: Pierre
authors.initials: P
authors.lastname: Ronco
authors.forename: Fabrice
authors.initials: F
authors.lastname: Quet
authors.forename: Michel
authors.initials: M
authors.lastname: Cogne
authors.forename: Jean-Paul
authors.initials: JP
authors.lastname: Fermand
mesh_terms: D000368:Aged; D000686:Amyloidosis / Q000188:drug therapy / Q000401:mortality / Q000628:therapy*; D003131:Combined Modality Therapy; D003907:Dexamethasone / Q000008:administration & dosage*; D018450:Disease Progression; D004359:Drug Therapy, Combination; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007147:Immunoglobulin Light Chains; D053208:Kaplan-Meier Estimate; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000008:administration & dosage*
chemical_list: D007147:Immunoglobulin Light Chains; D019653:Myeloablative Agonists; D003907:Dexamethasone; D008558:Melphalan