title: High-dose melphalan and peripheral blood stem cell transplantation for light-chain amyloidosis with cardiac involvement.
abstract: High-dose melphalan (HDM) plus stem cell transplantation is an effective treatment for light-chain amyloidosis (AL), but is associated with high treatment-related mortality in patients with cardiac involvement. We studied 187 patients with cardiac involvement with AL who underwent HDM between 1996 and 2008. The median age was 57 years and the median time from diagnosis to HDM was 3.6 months. Half of the patients received reduced-dose melphalan (100-160 mg/m(2)). The median overall survival (OS) was 66 months, 54 months from diagnosis and HDM, respectively, and 91 patients (49%) were alive at the last follow-up 52 months (median) from HDM. Thirty patients (16%) died within 100 days of transplantation; only low serum albumin predicted early deaths. Overall, hematologic response (HR) and cardiac responses were seen in 66% and 41% of patients, respectively. The median OS for patients with and without HR was not reached and 22 months, respectively (P < .01); and for those with any decrease and no decrease in N-terminal-pro-brain natriuretic peptide was not reached and 26 months, respectively (P < .01). In multivariate analysis of baseline factors, only reduced-dose melphalan predicted shorter OS. HDM is feasible in patients with cardiac amyloidosis, and achievement of HR and organ response is associated with improved survival.
authors.affiliation: Division of Hematology, Department of Medicine, Mayo Clinic, 200 First Street, Rochester, MN 55905, USA.
authors.forename: Sumit
authors.initials: S
authors.lastname: Madan
authors.forename: Shaji K
authors.initials: SK
authors.lastname: Kumar
authors.forename: Angela
authors.initials: A
authors.lastname: Dispenzieri
authors.forename: Martha Q
authors.initials: MQ
authors.lastname: Lacy
authors.forename: Suzanne R
authors.initials: SR
authors.lastname: Hayman
authors.forename: Francis K
authors.initials: FK
authors.lastname: Buadi
authors.forename: David
authors.initials: D
authors.lastname: Dingli
authors.forename: S Vincent
authors.initials: SV
authors.lastname: Rajkumar
authors.forename: William J
authors.initials: WJ
authors.lastname: Hogan
authors.forename: Nelson
authors.initials: N
authors.lastname: Leung
authors.forename: Martha
authors.initials: M
authors.lastname: Grogan
authors.forename: Morie A
authors.initials: MA
authors.lastname: Gertz
mesh_terms: D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000150:complications / Q000378:metabolism / Q000401:mortality / Q000628:therapy*; D004305:Dose-Response Relationship, Drug; D005240:Feasibility Studies; D005260:Female; D005500:Follow-Up Studies; D006331:Heart Diseases / Q000209:etiology / Q000401:mortality / Q000628:therapy*; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000378:metabolism; D008297:Male; D008558:Melphalan / Q000008:administration & dosage* / Q000009:adverse effects; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000008:administration & dosage / Q000009:adverse effects; D036102:Peripheral Blood Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D012189:Retrospective Studies; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods
chemical_list: D007147:Immunoglobulin Light Chains; D019653:Myeloablative Agonists; D008558:Melphalan