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Ozsahin, Hulya; Cavazzana-Calvo, Marina; Notarangelo, Luigi D.; Schulz, Ansgar; Thrasher, Adrian J.; Mazzolari, Evelina; Slatter, Mary A.; Le Deist, Francoise; Blanche, Stephane; Veys, Paul; Fasth, Anders; Bredius, Robbert; Sedlacek, Petr; Wulffraat, Nico; Ortega, Juan; Heilmann, Carsten; O'Meara, Anne; Wachowiak, Jacek; Kalwak, Krzysztof; Matthes-Martin, Susanne; Gungor, Tayfun; Ikinciogullari, Aydan; Landais, Paul; Cant, Andrew J.; [...]

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

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  • Media type: E-Article
  • 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
  • Contributor: Ozsahin, Hulya; Cavazzana-Calvo, Marina; Notarangelo, Luigi D.; Schulz, Ansgar; Thrasher, Adrian J.; Mazzolari, Evelina; Slatter, Mary A.; Le Deist, Francoise; Blanche, Stephane; Veys, Paul; Fasth, Anders; Bredius, Robbert; Sedlacek, Petr; Wulffraat, Nico; Ortega, Juan; Heilmann, Carsten; O'Meara, Anne; Wachowiak, Jacek; Kalwak, Krzysztof; Matthes-Martin, Susanne; Gungor, Tayfun; Ikinciogullari, Aydan; Landais, Paul; Cant, Andrew J.; [...]
  • imprint: American Society of Hematology, 2008
  • Published in: Blood
  • Language: English
  • DOI: 10.1182/blood-2007-03-076679
  • ISSN: 1528-0020; 0006-4971
  • Keywords: Cell Biology ; Hematology ; Immunology ; Biochemistry
  • Origination:
  • Footnote:
  • Description: <jats:p>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 &lt; .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.</jats:p>
  • Access State: Open Access