Outcome After Cord Blood Transplantation Using Busulfan Pharmacokinetics-Targeted Myeloablative Conditioning for Hurler Syndrome

Successful hematopoietic cell transplantation (HCT) provides life-long disease-modifying therapy for children with Hurler syndrome (HS), most severe phenotype of α-L-iduronidase (IDUA) deficiency, as donor-derived leukocyte engraftment serves as a stable source of the deficient enzyme for in situ cross-correction of neighboring tissues and neurons [1-5]. Although recent outcomes suggest that overall survival (OS) and event-free survival (EFS) may approach 9 5% and 90%, respectively, in experienced centers [6], we have recently reported a striking evolution in the pattern of graft failure from delayed autologous reconstitution observed in recipients of transplantation before implementation of busulfan (Bu) pharmacokinetics (PK)-guided myeloablative cond itioning (MAC) regimens to aplastic forms after implementation of Bu-PK MAC, and this primary/secondary aplastic-type graft failure is more commonly observed following umbilical cord blood transplantation (CBT) [7].
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research