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Blood, 1 January 2008, Vol. 111, No. 1, pp. 439-445. Prepublished online as a Blood First Edition Paper on September 27, 2007; DOI 10.1182/blood-2007-03-076679. This Article Full Text Full Text (PDF) Supplemental Appendix All Versions of this Article: blood-2007-03-076679v1 111/1/439    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Ozsahin, H. Articles by Fischer, A. Search for Related Content PubMed PubMed Citation Articles by Ozsahin, H. Articles by Fischer, A. Related Collections Transplantation Clinical Trials and Observations Immunobiology Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  TRANSPLANTATION 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 Hulya Ozsahin1, Marina Cavazzana-Calvo2, Luigi D. Notarangelo3, Ansgar Schulz4, Adrian J. Thrasher5, Evelina Mazzolari3, Mary A. Slatter6, Francoise Le Deist2, Stephane Blanche2, Paul Veys7, Anders Fasth8, Robbert Bredius9, Petr Sedlacek10, Nico Wulffraat11, Juan Ortega12, Carsten Heilmann13, Anne O'Meara14, Jacek Wachowiak15, Krzysztof Kalwak16, Susanne Matthes-Martin17, Tayfun Gungor18, Aydan Ikinciogullari19, Paul Landais2, Andrew J. Cant6, Wilhelm Friedrich4, and Alain Fischer2 1 Department of Pediatrics, Geneva University Hospital, Geneva, Switzerland; 2 Assistance Publique–Hôpitaux de Paris Unité d'Immunologie et d'Hématologie pédiatriques and Department of Biotherapy, Department of Biostatistics, Hôpital Necker-Enfants Malades, Université René Descartes, Paris, France; 3 Department of Pediatrics, University of Brescia, Brescia, Italy; 4 Department of Pediatrics, University of Ulm, Ulm, Germany; 5 Institute of Child Health, University College London, London, United Kingdom; 6 Newcastle General Hospital, Newcastle upon Tyne, United Kingdom; 7 Great Ormond Street Hospital for Children, London, United Kingdom; 8 Paediatric Immunology and Infectious Diseases Unit, Department of Pediatrics, Göteborg University, Göteborg, Sweden; 9 Leiden University Medical Center, Department of Pediatrics, Leiden, the Netherlands; 10 Department of Pediatric Hematology and Oncology, University Hospital Motol, Charles University, Prague, Czech Republic; 11 Department of Pediatrics, Section of Immunology, University Medical Center, Utrecht, the Netherlands; 12 Department of Hematology/Oncology, Hospital Universitario Materno-Infantil Val d'Hebron, Barcelona, Spain; 13 Paediatric Clinic Rigshospitalet, Copenhagen, Denmark; 14 Department of Haematology/Oncology and Hematopoietic Stem Cell Transplantation (HSCT), Our Lady's Children's Hospital Crumlin, Dublin, Ireland; 15 Department of Pediatric Hematology and Oncology and Hematopoietic Stem Cell Transplantation, University of Medical Sciences, Pozna, Poland; 16 Department of Pediatric Hematology and Oncology, Medical University of Wroclaw, Wroclaw, Poland; 17 Children's Cancer Research Institute, St Anna Children's Hospital, Vienna, Austria; 18 University Children's Hospital, Division of Immunology, Hematology, and HSCT, Zürich, Switzerland; and 19 Ankara University, Medical School, Department of Pediatric Immunology and Allergy, Ankara, Turkey 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. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: G. Bouma, S. Burns, and A. J. Thrasher Impaired T-cell priming in vivo resulting from dysfunction of WASp-deficient dendritic cells Blood, December 15, 2007; 110(13): 4278 - 4284. [Abstract] [Full Text] [PDF]

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