Peripheral-blood stem cells versus bone marrow from unrelated donors.
BACKGROUND
Randomized trials have shown that the transplantation of filgrastim-mobilized peripheral-blood stem cells from HLA-identical siblings accelerates engraftment but increases the risks of acute and chronic graft-versus-host disease (GVHD), as compared with the transplantation of bone marrow. Some studies have also shown that peripheral-blood stem cells are associated with a decreased rate of relapse and improved survival among recipients with high-risk leukemia.


METHODS
We conducted a phase 3, multicenter, randomized trial of transplantation of peripheral-blood stem cells versus bone marrow from unrelated donors to compare 2-year survival probabilities with the use of an intention-to-treat analysis. Between March 2004 and September 2009, we enrolled 551 patients at 48 centers. Patients were randomly assigned in a 1:1 ratio to peripheral-blood stem-cell or bone marrow transplantation, stratified according to transplantation center and disease risk. The median follow-up of surviving patients was 36 months (interquartile range, 30 to 37).


RESULTS
The overall survival rate at 2 years in the peripheral-blood group was 51% (95% confidence interval [CI], 45 to 57), as compared with 46% (95% CI, 40 to 52) in the bone marrow group (P=0.29), with an absolute difference of 5 percentage points (95% CI, -3 to 14). The overall incidence of graft failure in the peripheral-blood group was 3% (95% CI, 1 to 5), versus 9% (95% CI, 6 to 13) in the bone marrow group (P=0.002). The incidence of chronic GVHD at 2 years in the peripheral-blood group was 53% (95% CI, 45 to 61), as compared with 41% (95% CI, 34 to 48) in the bone marrow group (P=0.01). There were no significant between-group differences in the incidence of acute GVHD or relapse.


CONCLUSIONS
We did not detect significant survival differences between peripheral-blood stem-cell and bone marrow transplantation from unrelated donors. Exploratory analyses of secondary end points indicated that peripheral-blood stem cells may reduce the risk of graft failure, whereas bone marrow may reduce the risk of chronic GVHD. (Funded by the National Heart, Lung, and Blood Institute-National Cancer Institute and others; ClinicalTrials.gov number, NCT00075816.).
H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL 33612, USA. claudio.anasetti@moffitt.org.
Claudio
C
Anasetti
Brent R
BR
Logan
Stephanie J
SJ
Lee
Edmund K
EK
Waller
Daniel J
DJ
Weisdorf
John R
JR
Wingard
Corey S
CS
Cutler
Peter
P
Westervelt
Ann
A
Woolfrey
Stephen
S
Couban
Gerhard
G
Ehninger
Laura
L
Johnston
Richard T
RT
Maziarz
Michael A
MA
Pulsipher
David L
DL
Porter
Shin
S
Mineishi
John M
JM
McCarty
Shakila P
SP
Khan
Paolo
P
Anderlini
William I
WI
Bensinger
Susan F
SF
Leitman
Scott D
SD
Rowley
Christopher
C
Bredeson
Shelly L
SL
Carter
Mary M
MM
Horowitz
Dennis L
DL
Confer
D000328:Adult; D001855:Bone Marrow Diseases / Q000401:mortality / Q000628:therapy*; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000401:mortality*; D002423:Cause of Death; D005260:Female; D006084:Graft Rejection / Q000453:epidemiology; D006086:Graft vs Host Disease / Q000453:epidemiology; D006650:Histocompatibility Testing; D006801:Humans; D057194:Intention to Treat Analysis; D053208:Kaplan-Meier Estimate; D007938:Leukemia / Q000401:mortality / Q000628:therapy*; D008297:Male; D036102:Peripheral Blood Stem Cell Transplantation / Q000009:adverse effects / Q000401:mortality*; D016016:Proportional Hazards Models; D012008:Recurrence; D015996:Survival Rate; D061349:Unrelated Donors