Outcome after a cord blood transplantation using busulfan pharmacokinetic targeted myeloablative conditioning for Hurler syndrome

We report cord blood transplantation (CBT) outcomes of 97 children with Hurler syndrome (HS) after a busulfan pharmacokinetic targeted myeloablative conditioning regimens from 2004 to 2016. The median age at transplant was 10.8 months (range 0.23 – 63.2 months). The median follow-up for surviving patients was 4.2 years (range, 1.0 to 12.8 years). 5-year overall survival (OS) and engrafted survival (ES) were 88% and 79%. OS was 95% after Busulfan-Fludarabine-ATG, 90% after Busulfan-Cyclophosphamide-ATG and 74% after Busulfan-Cyclophosphamid e-Alemtuzumab (p=0.02).
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research