title: Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation: a case-control study.
abstract: Primary systemic amyloidosis (AL) is a plasma cell dyscrasia resulting in multisystem failure and death. High-dose chemotherapy with peripheral blood stem cell transplantation (PBSCT) has been associated with higher response rates and seemingly higher overall survival than standard chemotherapy. Selection bias, however, confounds interpretation of these results. We performed a case-match-control study comparing overall survival of 63 AL patients undergoing transplantation with 63 patients not undergoing transplantation. Matching criteria included age, sex, time to presentation, left ventricular ejection fraction, serum creatinine, septal thickness, nerve involvement, 24-hour urine protein, and serum alkaline phosphatase. According to design, there was no difference between the groups with respect to sex (57% males), age (median, 53 years), left ventricular ejection fraction (65%), number of patients with peripheral nerve involvement (17%), cardiac interventricular septal wall thickness (12 mm), serum creatinine (1.1 mg/dL [97.24 micromol/L]), and bone marrow plasmacytosis (8%). Sixty-six patients have died (16 cases and 50 controls). For PBSCT and control groups, respectively, the 1-, 2-, and 4-year overall survival rates are 89% and 71%; 81% and 55%; and 71% and 41%. Outside a randomized clinical trial, these results present the strongest data supporting the role of PBSCT in selected patients with AL.
authors.affiliation: Division of Hematology and Internal Medicine, Mayo Clinic, Rochester, MN 55905, USA. dispenzieri.angela@mayo.edu
authors.forename: Angela
authors.initials: A
authors.lastname: Dispenzieri
authors.forename: Robert A
authors.initials: RA
authors.lastname: Kyle
authors.forename: Martha Q
authors.initials: MQ
authors.lastname: Lacy
authors.forename: Terry M
authors.initials: TM
authors.lastname: Therneau
authors.forename: Dirk R
authors.initials: DR
authors.lastname: Larson
authors.forename: Matthew F
authors.initials: MF
authors.lastname: Plevak
authors.forename: S Vincent
authors.initials: SV
authors.lastname: Rajkumar
authors.forename: Rafael
authors.initials: R
authors.lastname: Fonseca
authors.forename: Philip R
authors.initials: PR
authors.lastname: Greipp
authors.forename: Thomas E
authors.initials: TE
authors.lastname: Witzig
authors.forename: John A
authors.initials: JA
authors.lastname: Lust
authors.forename: Steven R
authors.initials: SR
authors.lastname: Zeldenrust
authors.forename: Denise S
authors.initials: DS
authors.lastname: Snow
authors.forename: Susan R
authors.initials: SR
authors.lastname: Hayman
authors.forename: Mark R
authors.initials: MR
authors.lastname: Litzow
authors.forename: Dennis A
authors.initials: DA
authors.lastname: Gastineau
authors.forename: Ayalew
authors.initials: A
authors.lastname: Tefferi
authors.forename: David J
authors.initials: DJ
authors.lastname: Inwards
authors.forename: Ivana N
authors.initials: IN
authors.lastname: Micallef
authors.forename: Stephen M
authors.initials: SM
authors.lastname: Ansell
authors.forename: Luis F
authors.initials: LF
authors.lastname: Porrata
authors.forename: Michelle A
authors.initials: MA
authors.lastname: Elliott
authors.forename: Morie A
authors.initials: MA
authors.lastname: Gertz
mesh_terms: D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000401:mortality / Q000628:therapy*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D016022:Case-Control Studies; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010265:Paraproteinemias / Q000401:mortality / Q000628:therapy; D036102:Peripheral Blood Stem Cell Transplantation / Q000401:mortality*; D016016:Proportional Hazards Models; D012189:Retrospective Studies; D016019:Survival Analysis; D015996:Survival Rate