title: Sequential heart and autologous stem cell transplantation for systemic AL amyloidosis.
abstract: Extensive cardiac amyloid deposition in systemic AL amyloidosis is associated with a grave prognosis. Heart transplantation is rarely performed because of the systemic and progressive nature of the disease. Patients with severe cardiac amyloid infiltration are ineligible for the preferred treatment of melphalan chemotherapy with stem cell transplantation (SCT) rescue because of the high risk for treatment-related mortality. Heart transplantation followed by SCT was performed in 5 patients with AL amyloidosis and predominant cardiomyopathy. Patients were followed up for a median of 95 months (range, 37-118 months) from diagnosis. At censor, 3 of 5 patients were well without evidence of intracardiac or extracardiac amyloid accumulation, and median overall survival by Kaplan-Meier estimate was not reached. Two patients died of progressive amyloidosis 33 and 90 months after heart transplantation after relapse of their underlying plasma cell dyscrasia. Heart transplantation followed by SCT is feasible in selected patients with cardiac AL amyloidosis and may confer substantial survival benefit.
authors.affiliation: National Amyloidosis Centre, Department of Medicine, Royal Free and University College Medical School, London, United Kingdom. j.gillmore@medsch.ucl.ac.uk
authors.forename: Julian D
authors.initials: JD
authors.lastname: Gillmore
authors.forename: Hugh J
authors.initials: HJ
authors.lastname: Goodman
authors.forename: Helen J
authors.initials: HJ
authors.lastname: Lachmann
authors.forename: Mark
authors.initials: M
authors.lastname: Offer
authors.forename: Ashutosh D
authors.initials: AD
authors.lastname: Wechalekar
authors.forename: Jayshree
authors.initials: J
authors.lastname: Joshi
authors.forename: Mark B
authors.initials: MB
authors.lastname: Pepys
authors.forename: Philip N
authors.initials: PN
authors.lastname: Hawkins
mesh_terms: D000328:Adult; D000682:Amyloid / Q000378:metabolism; D000686:Amyloidosis / Q000150:complications / Q000378:metabolism / Q000401:mortality* / Q000628:therapy; D009202:Cardiomyopathies / Q000150:complications / Q000378:metabolism / Q000401:mortality* / Q000628:therapy; D018572:Disease-Free Survival; D005260:Female; D016027:Heart Transplantation / Q000401:mortality; D006801:Humans; D008297:Male; D008875:Middle Aged; D033581:Stem Cell Transplantation; D014182:Transplantation, Autologous / Q000401:mortality
chemical_list: D000682:Amyloid