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Blood, 1 February 2004, Vol. 103, No. 3, pp. 1152-1157. Prepublished online as a Blood First Edition Paper on October 2, 2003; DOI 10.1182/blood-2003-06-2014. This Article Full Text Full Text (PDF) All Versions of this Article: 2003-06-2014v1 103/3/1152    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 Gennery, A. R. Articles by Davies, E. G. Search for Related Content PubMed PubMed Citation Articles by Gennery, A. R. Articles by Davies, E. G. Related Collections Immunobiology Transplantation Clinical Trials and Observations Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  TRANSPLANTATION Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002 Andrew R. Gennery, Khulood Khawaja, Paul Veys, Robbert G. M. Bredius, Luigi D. Notarangelo, Evelina Mazzolari, Alain Fischer, Paul Landais, Marina Cavazzana-Calvo, Wilhelm Friedrich, Anders Fasth, Nico M. Wulffraat, Susanne Matthes-Martin, Danielle Bensoussan, Pierre Bordigoni, Andrzej Lange, Antonio Pagliuca, Marino Andolina, Andrew J. Cant, and E. Graham Davies, the European Group for Blood and Bone Marrow Transplantation and the European Society for Immunodeficiency From Newcastle General Hospital, Newcastle upon Tyne, United Kingdom; Great Ormond Street Hospital, London, United Kingdom; Leiden University Medical Centre, Leiden, the Netherlands; University of Brescia, Brescia, Italy; Necker Hospital, Paris, France; University Childrens Hospital, Ulm, Germany; Göteborg University, Göteborg, Sweden; The Wilhelmina Childrens Hospital, Utrecht, the Netherlands; St Anna Children's Hospital, Vienna, Austria; The Childrens Hospital, Vandoeuvre-Les-Nancy, France; the Lower Silesian Center for Cellular Transplantation, Wroclaw, Poland; King's College Hospital, London, United Kingdom; and the Pediatric Clinic, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Burlo Garofolo, Trieste, Italy. CD40 ligand (CD40L) deficiency causes recurrent sinopulmonary infection, Pneumocystis carinii pneumonia, and Cryptosporidium parvum infection. Approximately 40% to 50% of patients survive to the third decade: long-term survival is unclear. Hematopoietic stem cell transplantation (HSCT) is curative. We present a retrospective analysis of 38 European patients undergoing HSCT for CD40L deficiency in 8 European countries between 1993 and 2002. Donor stem cell source included 14 HLA-identical siblings, 22 unrelated donors, and 2 phenotypically matched parental stem cells (12 T-cell depleted). Of the patients, 34 engrafted and 26 (68%) survived; 3 had autologous reconstitution, 22 (58%) were cured, and 1 engrafted but has poor T-cell immune reconstitution. There were 18 evaluated patients who responded to vaccination. Of the patients, 12 (32%) died from infection-related complications, with severe cryptosporidiosis in 6. Grades 2 to 4 graft-versus-host disease (GvHD) associated with infection occurred in 6 of 12 fatal cases. HSCT cured 58% of patients, 72% of those without hepatic disease. Early T-cell function following whole marrow HSCT may limit cryptosporidial disease, but survival was similar after T-cell-depleted HSCT. Preexisting lung damage was the most important adverse risk factor. Further studies will determine optimal timing and type of HSCT. (Blood. 2004;103:1152-1157) CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: P F K Yong, F A Post, K C Gilmour, D Grosse-Kreul, A King, P Easterbrook, and M A A Ibrahim Cerebral toxoplasmosis in a middle-aged man as first presentation of primary immunodeficiency due to a hypomorphic mutation in the CD40 ligand gene J. Clin. Pathol., November 1, 2008; 61(11): 1220 - 1222. [Abstract] [Full Text] [PDF] Y. Tsuji, K. Imai, Y. Morinishi, K. Kogawa, M. Morino, and S. Nonoyama Successful unrelated cord blood transplantation for a patient with CD40 ligand deficiency Haematologica, December 1, 2007; 92(12): 1727 - 1728. [Abstract] [Full Text] [PDF] A. R. Gennery, M. A. Slatter, R. G. Bredius, M. M. Hagleitner, C. Weemaes, A. J. Cant, and A. C. Lankester Hematopoietic Stem Cell Transplantation Corrects the Immunologic Abnormalities Associated With Immunodeficiency Centromeric Instability Facial Dysmorphism Syndrome Pediatrics, November 1, 2007; 120(5): e1341 - e1344. [Abstract] [Full Text] [PDF]

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