Outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome.
Bone Marrow Transplant
; 47(11): 1428-35, 2012 Nov.
Article
en En
| MEDLINE
| ID: mdl-22426750
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).
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Síndrome de Wiskott-Aldrich
/
Trasplante de Células Madre Hematopoyéticas
Tipo de estudio:
Etiology_studies
/
Incidence_studies
/
Observational_studies
/
Risk_factors_studies
Límite:
Adolescent
/
Adult
/
Humans
/
Male
Idioma:
En
Revista:
Bone Marrow Transplant
Asunto de la revista:
TRANSPLANTE
Año:
2012
Tipo del documento:
Article
País de afiliación:
Estados Unidos