TY - JOUR
T1 - Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia
AU - Ayas, Mouhab
AU - Saber, Wael
AU - Davies, Stella M.
AU - Harris, Richard E.
AU - Hale, Gregory A.
AU - Socie, Gerard
AU - Lerademacher, Jennifer
AU - Thakar, Monica
AU - Deeg, H. Joachim J.
AU - Al-Seraihy, Amal
AU - Battiwalla, Minoo
AU - Camitta, Bruce M.
AU - Olsson, Richard
AU - Bajwa, Rajinder S.
AU - Bonfim, Carmem M.
AU - Pasquini, Ricardo
AU - MacMillan, Margaret L.
AU - George, Biju
AU - Copelan, Edward A.
AU - Wirk, Baldeep
AU - Jefri, Abdullah Al
AU - Fasth, Anders L.
AU - Guinan, Eva C.
AU - Horn, Biljana N.
AU - Lewis, Victor A.
AU - Slavin, Shimon
AU - Stepensky, Polina
AU - Bierings, Marc
AU - Gale, Robert Peter
N1 - Publisher Copyright:
Copyright © 2017 American Society of Clinical Oncology. All rights reserved.
PY - 2013/5/1
Y1 - 2013/5/1
N2 - Purpose Allogeneic hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi anemia (FA). Data on outcomes in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome (MDS), or acute leukemia have not been separately analyzed. Patients and Methods We analyzed data on 113 patients with FA with cytogenetic abnormalities (n 54), MDS (n 45), or acute leukemia (n 14) who were reported to the Center for International Blood and Marrow Transplant Research from 1985 to 2007. Results Neutrophil recovery occurred in 78% and 85% of patients at days 28 and 100, respectively. Day 100 cumulative incidences of acute graft-versus-host disease grades B to D and C to D were 26% (95% CI, 19% to 35%) and 12% (95% CI, 7% to 19%), respectively. Survival probabilities at 1, 3, and 5 years were 64% (95% CI, 55% to 73%), 58% (95% CI, 48% to 67%), and 55% (95% CI, 45% to 64%), respectively. In univariate analysis, younger age was associated with superior 5-year survival ( v 14 years: 69% [95% CI, 57% to 80%] v 39% [95% CI, 26% to 53%], respectively; P .001). In transplantations from HLA-matched related donors (n 82), younger patients ( v 14 years: 78% [95% CI, 64% to 90%] v 34% [95% CI, 20% to 50%], respectively; P .001) and patients with cytogenetic abnormalities only versus MDS/acute leukemia (67% [95% CI, 52% to 81%] v 43% [95% CI, 27% to 59%], respectively; P .03) had superior 5-year survival. Conclusion Our analysis indicates that long-term survival for patients with FA with cytogenetic abnormalities, MDS, or acute leukemia is achievable. Younger patients and recipients of HLA-matched related donor transplantations who have cytogenetic abnormalities only have the best survival.
AB - Purpose Allogeneic hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi anemia (FA). Data on outcomes in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome (MDS), or acute leukemia have not been separately analyzed. Patients and Methods We analyzed data on 113 patients with FA with cytogenetic abnormalities (n 54), MDS (n 45), or acute leukemia (n 14) who were reported to the Center for International Blood and Marrow Transplant Research from 1985 to 2007. Results Neutrophil recovery occurred in 78% and 85% of patients at days 28 and 100, respectively. Day 100 cumulative incidences of acute graft-versus-host disease grades B to D and C to D were 26% (95% CI, 19% to 35%) and 12% (95% CI, 7% to 19%), respectively. Survival probabilities at 1, 3, and 5 years were 64% (95% CI, 55% to 73%), 58% (95% CI, 48% to 67%), and 55% (95% CI, 45% to 64%), respectively. In univariate analysis, younger age was associated with superior 5-year survival ( v 14 years: 69% [95% CI, 57% to 80%] v 39% [95% CI, 26% to 53%], respectively; P .001). In transplantations from HLA-matched related donors (n 82), younger patients ( v 14 years: 78% [95% CI, 64% to 90%] v 34% [95% CI, 20% to 50%], respectively; P .001) and patients with cytogenetic abnormalities only versus MDS/acute leukemia (67% [95% CI, 52% to 81%] v 43% [95% CI, 27% to 59%], respectively; P .03) had superior 5-year survival. Conclusion Our analysis indicates that long-term survival for patients with FA with cytogenetic abnormalities, MDS, or acute leukemia is achievable. Younger patients and recipients of HLA-matched related donor transplantations who have cytogenetic abnormalities only have the best survival.
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U2 - 10.1200/JCO.2012.45.9719
DO - 10.1200/JCO.2012.45.9719
M3 - Article
C2 - 23547077
AN - SCOPUS:84884540764
SN - 0732-183X
VL - 31
SP - 1669
EP - 1676
JO - Journal of Clinical Oncology
JF - Journal of Clinical Oncology
IS - 13
ER -