Abstract:
: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.
journal_name
Bloodjournal_title
Bloodauthors
Peffault de Latour R,Porcher R,Dalle JH,Aljurf M,Korthof ET,Svahn J,Willemze R,Barrenetxea C,Mialou V,Soulier J,Ayas M,Oneto R,Bacigalupo A,Marsh JC,Peters C,Socie G,Dufour C,FA Committee of the Severe Aplastic Anemia Wdoi
10.1182/blood-2013-01-479733subject
Has Abstractpub_date
2013-12-19 00:00:00pages
4279-86issue
26eissn
0006-4971issn
1528-0020pii
blood-2013-01-479733journal_volume
122pub_type
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