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Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation.

Abstract：

:Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency with microthrombocytopenia, eczema, recurrent infections, autoimmune disorders, and malignancies that are life-threatening in the majority of patients. In this long-term, retrospective, multicenter study, we analyzed events that occurred in 96 WAS patients who received transplants between 1979 and 2001 who survived at least 2 years following hematopoietic stem-cell transplantation (HSCT). Events included chronic graft-versus-host disease (cGVHD), autoimmunity, infections, and sequelae of before or after HSCT complications. Three patients (3%) died 2.1 to 21 years following HSCT. Overall 7-year event-free survival rate was 75%. It was lower in recipients of mismatched related donors, also in relation with an older age at HSCT and disease severity. The most striking finding was the observation of cGVHD-independent autoimmunity in 20% of patients strongly associated with a mixed/split chimerism status (P < .001), suggesting that residual-host lymphocytes can mediate autoimmune disease despite the coexistence of donor lymphocytes. Infectious complications (6%) related to splenectomy were also significant and may warrant a more restrictive approach to performing splenectomy in WAS patients. Overall, this study provides the basis for a prospective, standardized, and more in-depth detailed analysis of chimerism and events in long-term follow-up of WAS patients who receive transplants to design better-adapted therapeutic strategies.

journal_name

Blood

journal_title

Blood

authors

Ozsahin H,Cavazzana-Calvo M,Notarangelo LD,Schulz A,Thrasher AJ,Mazzolari E,Slatter MA,Le Deist F,Blanche S,Veys P,Fasth A,Bredius R,Sedlacek P,Wulffraat N,Ortega J,Heilmann C,O'Meara A,Wachowiak J,Kalwak K,Matthes-

doi

10.1182/blood-2007-03-076679

subject

Has Abstract

pub_date

2008-01-01 00:00:00

pages

439-45

issue

eissn

0006-4971

issn

1528-0020

pii

blood-2007-03-076679

journal_volume

111

pub_type

杂志文章,多中心研究

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Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation.