Outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome.

Abstract:

:HLA-identical sibling donor transplantation remains the treatment of choice for Wiskott-Aldrich Syndrome (WAS). Since 1990, utilization of alternative donor sources has increased significantly. We report the hematopoietic cell transplantation (HCT) outcomes of 47 patients with WAS treated at a single center since 1990. Improved outcomes were observed after 2000 despite the increased number of alternative donors. Five-year OS improved from 62.5% (95% CI: 34.9% to 81.1%) to 90.8% (95% CI: 67.7% to 97.6%) for patients transplanted during 1990-2000 and 2001-2009, respectively. In multivariate analysis, transplant era significantly impacted OS (P=0.04), whereas age was only marginally significant (P=0.06, Cox proportional hazard analysis). No TRM occurred within the first 100 days among patients transplanted during 2001-2009 compared with 3/16 during 1990-2000, (P=0.03, Fisher's exact test). The extent of HLA mismatch did not significantly affect the incidence of acute GVHD, chronic GVHD or survival. Post-HCT autoimmune cytopenias were frequently diagnosed after 2001: 17/31 (55%) patients. Their occurrence was not associated with transplant donor type (P=0.53), acute GVHD (P=0.74), chronic GVHD (P=0.12), or post-transplant mixed chimerism (P=0.50).

journal_name

Bone Marrow Transplant

authors

Shin CR,Kim MO,Li D,Bleesing JJ,Harris R,Mehta P,Jodele S,Jordan MB,Marsh RA,Davies SM,Filipovich AH

doi

10.1038/bmt.2012.31

subject

Has Abstract

pub_date

2012-11-01 00:00:00

pages

1428-35

issue

11

eissn

0268-3369

issn

1476-5365

pii

bmt201231

journal_volume

47

pub_type

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