TY - JOUR
T1 - Allogeneic hematopoietic stem cell transplantation in Fanconi anemia
T2 - the European Group for Blood and Marrow Transplantation experience
AU - de Latour, Regis Peffault
AU - Porcher, Raphael
AU - Dalle, Jean-Hugues
AU - Aljurf, Mahmoud
AU - Korthof, Elisabeth T.
AU - Svahn, Johanna
AU - Willemze, Roelof
AU - Barrenetxea, Cristina
AU - Mialou, Valerie
AU - Soulier, Jean
AU - Ayas, Mouhab
AU - Oneto, Rosi
AU - Bacigalupo, Andrea
AU - Marsh, Judith C. W.
AU - Peters, Christina
AU - Socie, Gerard
AU - Dufour, Carlo
AU - FA Comm Severe Aplastic Anemia
AU - European Grp Blood Marrow
PY - 2013/12/19
Y1 - 2013/12/19
N2 - Although allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for patients with Fanconi anemia (FA), published series mostly refer to single-center experience with limited numbers of patients. We analyzed results in 795 patients with FA who underwent first HSCT between May 1972 and January 2010. With a 6-year median follow-up, overall survival was 49% at 20 years (95% confidence interval, 38-65 years). Better outcome was observed for patients transplanted before the age of 10 years, before clonal evolution (ie, myelodysplastic syndrome or acute myeloid leukemia), from a matched family donor, after a conditioning regimen without irradiation, the latter including fludarabine. Chronic graft-versus-host disease and secondary malignancy were deleterious when considered as time-dependent covariates. Age more than 10 years at time of HSCT, clonal evolution as an indication for transplantation, peripheral blood as source of stem cells, and chronic graft-versus-host disease were found to be independently associated with the risk for secondary malignancy. Changes in transplant protocols have significantly improved the outcome of patients with FA, who should be transplanted at a young age, with bone marrow as the source of stem cells.
AB - Although allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for patients with Fanconi anemia (FA), published series mostly refer to single-center experience with limited numbers of patients. We analyzed results in 795 patients with FA who underwent first HSCT between May 1972 and January 2010. With a 6-year median follow-up, overall survival was 49% at 20 years (95% confidence interval, 38-65 years). Better outcome was observed for patients transplanted before the age of 10 years, before clonal evolution (ie, myelodysplastic syndrome or acute myeloid leukemia), from a matched family donor, after a conditioning regimen without irradiation, the latter including fludarabine. Chronic graft-versus-host disease and secondary malignancy were deleterious when considered as time-dependent covariates. Age more than 10 years at time of HSCT, clonal evolution as an indication for transplantation, peripheral blood as source of stem cells, and chronic graft-versus-host disease were found to be independently associated with the risk for secondary malignancy. Changes in transplant protocols have significantly improved the outcome of patients with FA, who should be transplanted at a young age, with bone marrow as the source of stem cells.
KW - ACQUIRED APLASTIC-ANEMIA
KW - VERSUS-HOST-DISEASE
KW - MATCHED UNRELATED DONORS
KW - SOLID MALIGNANT-TUMORS
KW - BONE-MARROW
KW - CONDITIONING REGIMEN
KW - CUMULATIVE INCIDENCE
KW - ALTERNATIVE DONORS
KW - COMPETING RISK
KW - SIBLING DONORS
U2 - 10.1182/blood-2013-01-479733
DO - 10.1182/blood-2013-01-479733
M3 - Article
SN - 0006-4971
VL - 122
SP - 4279
EP - 4286
JO - Blood
JF - Blood
IS - 26
ER -