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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

2007; Elsevier BV; Volume: 111; Issue: 1 Linguagem: Inglês

10.1182/blood-2007-03-076679

1528-0020

Hülya Özşahin, Marina Cavazzana, Luigi D. Notarangelo, Ansgar Schulz, Adrian J. Thrasher, Evelina Mazzolari, Mary Slatter, Françoise Le Deist, Stéphane Blanche, Paul Veys, Anders Fasth, Robbert G. M. Bredius, Petr Sedláček, Nico Wulffraat, Juan Ortega, Carsten Heilmann, Anne O’Meara, Jacek Wachowiak, Krzysztof Kałwak, Susanne Matthes‐Martin, Tayfun Güngör, Aydan İkincioğulları, Paul Landais, Andrew J. Cant, Wilhelm Friedrich, Alain Fischer,

Blood disorders and treatments

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.