Outcomes following hematopoietic cell transplantation for Wiskott–Aldrich syndrome

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).