[x] close Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: the EBMT experience Régis Peffault de Latour1,*, Raphael Porcher2, Jean-Hugues Dalle3, et al. Corresponding author; email: regis.peffaultdelatour{at}sls.aphp.fr Key points •Best survival benefit of HSCT is observed in FA patients transplanted before 10 years with bone marrow following a fludarabine-based regimen. •Long-term outcome of FA patients after transplantation is mainly affected by secondary malignancies and chronic graft versus host disease. 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 FA patients who underwent first HSCT between May 1972 and January 2010. With a six-year median follow-up, overall survival (OS) was 49% at 20 years (95%CI 38-65). Better outcome was observed for patients transplanted after the age of 10 years, before clonal evolution (i.e. myelodysplastic syndrome or acute myeloid leukemia), from a matched family donor, following an irradiation-free containing conditioning regimen, the latter including fludarabine. Chronic graft versus host disease (GvHD) 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 GvHD were found independently associated with the risk of secondary malignancy. Changes in transplant protocols have significantly improved the outcome of FA patients, who should be transplanted at a young age with bone marrow as the source of stem cells. Submitted January 23, 2013.Accepted October 9, 2013. Copyright © 2005 American Society of Hematology
Posted on: Wed, 13 Nov 2013 12:48:48 +0000
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