title: 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).
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
authors.forename: C R
authors.initials: CR
authors.lastname: Shin
authors.forename: M-O
authors.initials: MO
authors.lastname: Kim
authors.forename: D
authors.initials: D
authors.lastname: Li
authors.forename: J J
authors.initials: JJ
authors.lastname: Bleesing
authors.forename: R
authors.initials: R
authors.lastname: Harris
authors.forename: P
authors.initials: P
authors.lastname: Mehta
authors.forename: S
authors.initials: S
authors.lastname: Jodele
authors.forename: M B
authors.initials: MB
authors.lastname: Jordan
authors.forename: R A
authors.initials: RA
authors.lastname: Marsh
authors.forename: S M
authors.initials: SM
authors.lastname: Davies
authors.forename: A H
authors.initials: AH
authors.lastname: Filipovich
mesh_terms: D000293:Adolescent; D000328:Adult; D015331:Cohort Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D014923:Wiskott-Aldrich Syndrome / Q000276:immunology / Q000601:surgery*; D055815:Young Adult