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Long-term outcome f...
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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Ozsahin, Hulya (author)
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Cavazzana-Calvo, Marina (author)
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Notarangelo, Luigi D (author)
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Schulz, Ansgar (author)
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Thrasher, Adrian J (author)
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Mazzolari, Evelina (author)
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Slatter, Mary A (author)
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Le Deist, Francoise (author)
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Blanche, Stephane (author)
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Veys, Paul (author)
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- Fasth, Anders, 1945 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper,Institute of Clinical Sciences
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Bredius, Robbert (author)
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Sedlacek, Petr (author)
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Wulffraat, Nico (author)
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Ortega, Juan (author)
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Heilmann, Carsten (author)
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O'Meara, Anne (author)
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Wachowiak, Jacek (author)
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Kalwak, Krzysztof (author)
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Matthes-Martin, Susanne (author)
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Gungor, Tayfun (author)
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Ikinciogullari, Aydan (author)
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Landais, Paul (author)
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Cant, Andrew J (author)
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Friedrich, Wilhelm (author)
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Fischer, Alain (author)
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(creator_code:org_t)
- American Society of Hematology, 2008
- 2008
- English.
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In: Blood. - : American Society of Hematology. - 0006-4971 .- 1528-0020. ; 111:1, s. 439-45
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https://www.zora.uzh...
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https://gup.ub.gu.se...
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https://doi.org/10.1...
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Abstract
Subject headings
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- 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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Pediatrik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Pediatrics (hsv//eng)
Keyword
- Adolescent
- Autoimmune Diseases
- etiology
- Child
- Child
- Preschool
- Cooperative Behavior
- Disease-Free Survival
- Europe
- Graft vs Host Disease
- etiology
- Hematopoietic Stem Cell Transplantation
- Humans
- Immune System
- immunology
- Infant
- Recovery of Function
- immunology
- Retrospective Studies
- Splenectomy
- Survival Rate
- Transplantation Chimera
- Treatment Outcome
- Wiskott-Aldrich Syndrome
- immunology
- surgery
- therapy
Publication and Content Type
- ref (subject category)
- art (subject category)
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Blood
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- By the author/editor
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Ozsahin, Hulya
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Cavazzana-Calvo, ...
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Notarangelo, Lui ...
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Schulz, Ansgar
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Thrasher, Adrian ...
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Mazzolari, Eveli ...
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show more...
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Slatter, Mary A
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Le Deist, Franco ...
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Blanche, Stephan ...
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Veys, Paul
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Fasth, Anders, 1 ...
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Bredius, Robbert
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Sedlacek, Petr
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Wulffraat, Nico
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Ortega, Juan
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Heilmann, Carste ...
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O'Meara, Anne
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Wachowiak, Jacek
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Kalwak, Krzyszto ...
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Matthes-Martin, ...
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Gungor, Tayfun
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Ikinciogullari, ...
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Landais, Paul
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Cant, Andrew J
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Friedrich, Wilhe ...
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Fischer, Alain
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- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Pediatrics
- Articles in the publication
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Blood
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University of Gothenburg