Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002
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.x}-z:3, 百拇医药
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,(Andrew R. Gennery Khulood Khawaja Paul Veys Robbert G. M. Bredius Luigi D. Notarangelo Evelina Mazzo)
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,(Andrew R. Gennery Khulood Khawaja Paul Veys Robbert G. M. Bredius Luigi D. Notarangelo Evelina Mazzo)