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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.
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
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 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.
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| Copyright © 2008 by American Society of Hematology Online ISSN: 1528-0020 | |||||||||
, 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
