title: Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients.
abstract: Previous studies have suggested that, in patients with AL amyloidosis treated with high-dose melphalan and autologous stem-cell transplantation (HDM/SCT), the greatest benefit is seen in those patients achieving a hematologic complete response (CR). We analyzed a series of 421 consecutive patients treated with HDM/SCT at a single referral center and compared outcomes for patients with and without CR. Treatment-related mortality was 11.4% overall (5.6% in the last 5 years). By intention-to-treat analysis, the CR rate was 34% and the median event-free survival (EFS) and overall survival (OS) were 2.6 and 6.3 years, respectively. Eighty-one patients died within the first year after HDM/SCT and were not evaluable for hematologic and organ response. Of 340 evaluable patients, 43% achieved CR and 78% of them experienced an organ response. For CR patients, median EFS and OS were 8.3 and 13.2 years, respectively. Among the 195 patients who did not obtain CR, 52% achieved an organ response, and their median EFS and OS were 2 and 5.9 years, respectively. Thus, treatment of selected AL patients with HDM/SCT resulted in a high organ response rate and long OS, even for those patients who did not achieve CR.
authors.affiliation: Amyloid Treatment and Research Program, Department of Medicine, Boston University School of Medicine, Boston, MA 02118, USA.
authors.forename: Maria Teresa
authors.initials: MT
authors.lastname: Cibeira
authors.forename: Vaishali
authors.initials: V
authors.lastname: Sanchorawala
authors.forename: David C
authors.initials: DC
authors.lastname: Seldin
authors.forename: Karen
authors.initials: K
authors.lastname: Quillen
authors.forename: John L
authors.initials: JL
authors.lastname: Berk
authors.forename: Laura M
authors.initials: LM
authors.lastname: Dember
authors.forename: Adam
authors.initials: A
authors.lastname: Segal
authors.forename: Frederick
authors.initials: F
authors.lastname: Ruberg
authors.forename: Hans
authors.initials: H
authors.lastname: Meier-Ewert
authors.forename: Nancy T
authors.initials: NT
authors.lastname: Andrea
authors.forename: J Mark
authors.initials: JM
authors.lastname: Sloan
authors.forename: Kathleen T
authors.initials: KT
authors.lastname: Finn
authors.forename: Gheorghe
authors.initials: G
authors.lastname: Doros
authors.forename: Joan
authors.initials: J
authors.lastname: Blade
authors.forename: Martha
authors.initials: M
authors.lastname: Skinner
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000686:Amyloidosis / Q000188:drug therapy / Q000378:metabolism / Q000628:therapy*; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage / Q000627:therapeutic use*; D018572:Disease-Free Survival; D005260:Female; D006403:Hematologic Tests; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000378:metabolism*; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000627:therapeutic use*; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000008:administration & dosage / Q000627:therapeutic use*; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D018906:Antineoplastic Agents, Alkylating; D007147:Immunoglobulin Light Chains; D019653:Myeloablative Agonists; D008558:Melphalan