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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 (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 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. Social Bookmarking                   What's this?  Previous Article  |  Next Article  Submitted March 6, 2007 Accepted September 16, 2007 Long-term outcome following hematopoietic stem cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and the European Group for Blood and Marrow Transplantation Hulya Ozsahin*, Marina Cavazzana-Calvo, Luigi D Notarangelo, Ansgar Schulz, Adrian J Thrasher, Evelina Mazzolari, Mary A Slatter, Francoise Le Deist, Stephane Blanche, Paul Veys, Anders Fasth, Robbert Bredius, Petr Sedlacek, Nico Wulffraat, Juan Ortega, Carsten Heilmann, Anne O'Meara, Jacek Wachowiak, Krzysztof Kalwak, Susanne Matthes-Martin, Tayfun Gungor, Aydan Ikinciogullari, Paul Landais, Andrew J Cant, Wilhelm Friedrich, and Alain Fischer Department of Pediatrics, Geneva University Hospital, Geneva, Switzerland AP-HP Unite d'Immunologie et d'Hematologie pediatriques, Dept of Biotherapy, Dept of Biostatistics, Hopital Necker-Enfants Malades, Universite Rene Descartes, Paris, France Department of Pediatrics, University of Brescia, Brescia, Italy Department of Pediatrics, University of Ulm, Ulm, Germany Institute of Child Health, University College London, London, United Kingdom Department of Paediatric Immunology and Infectious Diseases, Newcastle General Hospital, Newcastle upon Tyne, United Kingdom Department of Pediatrics, Great Ormond Street Hospital for Children, London, United Kingdom Paediatric Immunology & Infectious Diseases Unit, Department of Pediatrics, Goteborg University, Goteborg, Sweden Department of Pediatrics, Leiden University Medical Center, Leiden, Netherlands Department of Pediatric Hematology and Oncology, University Hospital Motol, Charles University, Prague, Czech Republic Department of Pediatrics, Section of Immunology, University Medical Center, Utrecht, Netherlands Department of Hematology/Oncology, Hospital Universitario Materno-Infantil Val d'Hebron, Barcelona, Spain Pediatric Clinic, Rigshospitalet, Copenhagen, Denmark Department of Hematology/Oncology & HSCT, Our Lady's Children's Hospital, Dublin, Republic of Ireland Department of Pediatric Hematolog & Oncology, & Hematopoietic Stem Cell Transplantation, University of Medical Sciences, Poznan, Poland Department of Pediatric Hematology and Oncology, Medical University of Wroclaw, Wroclaw, Poland The Children's Cancer Research Institute, Saint Anna Children's Hospital, Vienna, Austria Division of Immunology, Hematology, and HSCT, University Children's Hospital, Zurich, Switzerland Department of Pediatric Immunology and Allergy, Ankara University Medical School, Ankara, Turkey * Corresponding author; email: hulya.ozsahin{at}gmail.com. 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 transplanted between 1979-2001 who survived at least two years following hematopoietic stem cell transplantation (HSCT). Events included chronic graft-versus-host disease (cGVHD), autoimmunity, infections and sequelae of pre/post HSCT complications. Three patients (3%) died 2.1 to 21 years post-HSCT. Overall 7-year event-free survival rate was 75% (median 7 years). 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, and strongly associated with a mixed/split chimerism status (P < .0001) suggesting that residual host lymphocytes can mediate autoimmune disease despite 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 transplanted WAS patients in order 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: M. Bosticardo, F. Marangoni, A. Aiuti, A. Villa, and M. Grazia Roncarolo Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome Blood, June 18, 2009; 113(25): 6288 - 6295. [Abstract] [Full Text] [PDF] L. S. Westerberg, M. A. de la Fuente, F. Wermeling, H. D. Ochs, M. C. I. Karlsson, S. B. Snapper, and L. D. Notarangelo WASP confers selective advantage for specific hematopoietic cell populations and serves a unique role in marginal zone B-cell homeostasis and function Blood, November 15, 2008; 112(10): 4139 - 4147. [Abstract] [Full Text] [PDF] 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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