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Title

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

Authors
Cavazzana-Calvo, Marina
Notarangelo, Luigi D.
Schulz, Ansgar
Thrasher, Adrian J.
Mazzolari, Evelina
Slatter, Mary A.
Le Deist, Francoise
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Published in Blood. 2008, vol. 111, no. 1, p. 439-445
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.
Keywords AdolescentAutoimmune Diseases/etiologyChildChild, PreschoolCooperative BehaviorDisease-Free SurvivalEuropeGraft vs Host Disease/etiology*Hematopoietic Stem Cell TransplantationHumansImmune System/*immunologyInfantRecovery of Function/*immunologyRetrospective StudiesSplenectomySurvival RateTransplantation ChimeraTreatment OutcomeWiskott-Aldrich Syndrome/*immunology/surgery/*therapy
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PMID: 17901250
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Research group Groupe Ozsahin Ayse Hulya (onco-hémato pédiatrie) (889)
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OZSAHIN, Ayse Hulya et al. 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. In: Blood, 2008, vol. 111, n° 1, p. 439-445. https://archive-ouverte.unige.ch/unige:19064

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Deposited on : 2012-03-27

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