Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation: a case-control study.
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.
Dispenzieri
Angela
A
Division of Hematology and Internal Medicine, Mayo Clinic, Rochester, MN 55905, USA. dispenzieri.angela@mayo.edu
Kyle
Robert A
RA
Lacy
Martha Q
MQ
Therneau
Terry M
TM
Larson
Dirk R
DR
Plevak
Matthew F
MF
Rajkumar
S Vincent
SV
Fonseca
Rafael
R
Greipp
Philip R
PR
Witzig
Thomas E
TE
Lust
John A
JA
Zeldenrust
Steven R
SR
Snow
Denise S
DS
Hayman
Susan R
SR
Litzow
Mark R
MR
Gastineau
Dennis A
DA
Tefferi
Ayalew
A
Inwards
David J
DJ
Micallef
Ivana N
IN
Ansell
Stephen M
SM
Porrata
Luis F
LF
Elliott
Michelle A
MA
Gertz
Morie A
MA
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