Four decades of stem cell transplantation for Fanconi anaemia in the Netherlands

Summary This article presents the haematopoietic stem cell transplantation ( SCT ) results of the complete Dutch Fanconi anaemia ( FA ) patient cohort. Sixty‐eight Dutch FA patients have been transplanted since 1972. In total, 63 (93%) patients engrafted, 54 after first SCT and 9 after second SCT . Fludarabine (FLU)‐based conditioning was associated with decreased graft failure (odds ratio 0·21, P = 0·01), decreased early mortality (hazard ratio 0·25, P = 0·01) and improved 5‐year overall survival (FLU 87·8% [standard error ( SE ) 5·1%] versus non‐FLU 59·3% [ SE 9·5%], P = 0·01). Late mortalit... Mehr ...