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Permanent URL to this publication: http://dx.doi.org/10.5167/uzh-5684

Ozsahin, H; Cavazzana-Calvo, M; Notarangelo, L D; Schulz, A; Thrasher, A J; Mazzolari, E; Slatter, M A; 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-Martin, S; Gungor, T; Ikinciogullari, A; Landais, P; Cant, A J; Friedrich, W; Fischer, A (2008). 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. Blood, 111(1):439-445.

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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.


94 citations in Web of Science®
107 citations in Scopus®
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Additional indexing

Item Type:Journal Article, refereed, original work
Communities & Collections:04 Faculty of Medicine > University Children's Hospital Zurich > Medical Clinic
Dewey Decimal Classification:610 Medicine & health
Deposited On:06 Feb 2009 08:25
Last Modified:05 Apr 2016 12:34
Publisher:American Society of Hematology
Additional Information:This research was originally published in Blood 2008, 111(1), 439-445. Copyright by the American Society of Hematology.
Publisher DOI:10.1182/blood-2007-03-076679
Official URL:http://bloodjournal.hematologylibrary.org/cgi/reprint/111/1/439
PubMed ID:17901250

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