Allogeneic hematopoietic stem cell transplantation in XXXX : a single center experience.

@entity1 @entity229 @entity786 @entity205 @entity2132 @entity74 @entity1863 @entity3980 @entity1499
@entity3980 is a rare @entity74 . The severe phenotype is fatal unless hematopoietic stem cell transplantation (HSCT) is performed. A retrospective analysis was performed in 11 @entity1 with @entity3980 who underwent HSCT and monitoring over a period of 19 years at our institution. The median age at HSCT was 8.8 months. Stem cell sources were unmanipulated bone marrow from an HLA-matched related donor in 7 @entity1 , unrelated  umbilical cord blood in 3 @entity1 , and a mismatched related donor in 1 @entity1 . Three @entity1 underwent a second HSCT. Conditioning was provided with a @entity1863 - and @entity786 -based regimen, with anti-thymocyte immunoglobulin added for the cord blood transplant recipients. @entity2132 prophylaxis consisted of @entity205 and @entity1499 for related donor recipients (8 @entity1 ) and @entity205 and @entity229 for cord blood transplant recipients (3 @entity1 ). The overall event-free survival rate was 91% with a median follow-up of 94 months (range, 15-223 months). Ten @entity1 had immune reconstitution and demonstrated sustained engraftment that ranged from 11% to 100% for lymphoid lines and from 0% to 100% for myeloid lines. HSCT from a matched related donor or unrelated cord blood provided excellent outcome, and mixed chimerism appeared satisfactory to prevent recurrent infections.
