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Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study
Authors:Francesca Ferrua  Stefania Galimberti  Virginie Courteille  Mary Anne Slatter  Claire Booth  Despina Moshous  Benedicte Neven  Stephane Blanche  Marina Cavazzana  Alexandra Laberko  Anna Shcherbina  Dmitry Balashov  Elena Soncini  Fulvio Porta  Hamoud Al-Mousa  Bandar Al-Saud  Hasan Al-Dhekri  Rand Arnaout  Andrew Richard Gennery
Institution:1. Department of Pediatric Immunology and HSCT, Great North Children''s Hospital, Newcastle upon Tyne, United Kingdom;2. San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy;3. Vita-Salute San Raffaele University, Milan, Italy;4. Center of Biostatistics for Clinical Epidemiology, School of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy;5. Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France;6. Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom;7. Department of Pediatric Immunology, Great Ormond Street Hospital, London, United Kingdom;8. Pediatric Hematology-Immunology and Rheumatology Unit, Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France;9. Biotherapy Department, Necker Children''s Hospital, AP-HP, Paris, France;10. Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM, Paris, France;11. INSERM UMR 1163, Laboratory of Human Lymphohematopoiesis, Paris, France;12. Dmitry Rogachev Federal Research Centre of Pediatric Hematology, Oncology and Immunology, Moscow, Russia;13. Pediatric Oncology-Hematology and BMT Unit, Spedali Civili di Brescia, Brescia, Italy;14. Department of Pediatrics, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia;15. Department of Pediatric Hematology and Oncology, University Hospital Motol Prague, Prague, Czech Republic;p. Institut d''Hematologie et d''Oncologie Pediatrique, Hospices Civils de Lyon, Lyon, France;q. L. Hirszfeld Institute of Immunology and Experimental Therapy, Polish Academy of Sciences, Wroc?aw, Poland;r. Lower Silesian Center for Cellular Transplantation & National Bone Marrow Donor Registry, Wroc?aw, Poland;s. Department of Allergy and Immunology, Royal Children''s Hospital, Melbourne, Australia;t. Immunology Department, Children''s Memorial Health Institute, Warsaw, Poland;u. Department of Pediatrics, University of Texas Southwestern Medical Center Dallas, Dallas, Tex;v. Division of Pediatric Allergy, Immunology & Bone Marrow Transplantation, University of California, San Francisco, Calif;w. Department of Pediatrics, Sahlgrenska Academy at University of Gothenburg and Queen Silvia Children''s Hospital, Gothenburg, Sweden;x. Pediatric Oncology and Hematology Unit, Children Hospital, University Hospital Nancy, Vandoeuvre-les-Nancy, France;y. Pediatric Clinic, Rigshospitalet, Copenhagen, Denmark;z. Department of Pediatrics, University Medical Center Ulm, Ulm, Germany;11. Department of Pediatrics, Division of Allergology, Clinical Immunology, Respiratory Diseases and Rheumatology, University Hospital Center Zagreb, Zagreb, Croatia;22. Department of Pediatrics/Willem-Alexander Children''s hospital, Leiden University Medical Center, Leiden, The Netherlands;33. Department of Pediatrics, University Medical Centre Utrecht, Utrecht University, Utrecht, The Netherlands;44. Princess Maxima Center for Pediatric Oncology, Utrecht, The Netherlands;55. Hématologie Adulte, Hôpital Necker, AP-HP, Paris, France;66. Division of Allergy Immunology, Department of Pediatrics, Children''s Hospital of Philadelphia, Philadelphia, Pa;77. Pediatric Hematology/Oncology, Dr. von Hauner University Children''s Hospital, Munich, Germany;88. Department of Pediatric Hematology and Oncology, Wroclaw Medical University, Wroc?aw, Poland;99. Service d''hématologie pédiatrique, Hôpital de la Timone Enfants, Marseille, France;1010. Pediatric Stem Cell Transplantation, Columbia University College of Physicians and Surgeons, New York, NY;1111. Pediatric Hematology–Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium;1212. University Children''s Hospital of Cracow, Cracow, Poland;1313. Pediatric Hematology and Oncology Department, Hospital Universitario MaternoInfantil Vall d’Hebron, Barcelona, Spain;1414. Department of Pediatric Immunology and Allergy, Ankara University School of Medicine, Ankara, Turkey;1515. Department of Clinical Immunology and Transplantology, Jagiellonian University, Medical Collage, Transplantation Center, University Children''s Hospital, Cracow, Poland;pp. Department of Pediatric Immunology-Allergy and BMT Unit, Ankara University Medical School, Ankara, Turkey;qq. Department of Pediatric Hematology and Stem Cell Transplantation United St. István and St László Hospital, Budapest, Hungary;rr. Department of Hematology, Karolinska University Hospital, Stockholm, Sweden;ss. Department of Pediatrics, University Hospitals Leuven, Division of Pediatric Immunology, Department of Immunology and Microbiology, Catholic University Leuven, Leuven, Belgium;tt. Nationwide Children''s Hospital, Columbus, Ohio;uu. Pediatric Blood and Marrow Transplant, University of Minnesota, Minneapolis, Minn;vv. Center for Chronic Immunodeficiency, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany;ww. Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany;xx. Division of Pediatrics, CLINTEC, Karolinska Institutet, Stockholm, Sweden;yy. Pediatric Blood Disorders, Immunodeficiency and SCT, Astrid Lindgren Children''s Hospital, Karolinska University Hospital, Stockholm, Sweden;zz. Cancer Centre for Children, Children''s Hospital at Westmead, Sydney, Australia;111. University of Sydney Medical Program, Sydney, Australia;222. Department of Pediatrics, Memorial Sloan Kettering Cancer Center, BMT and Cell Therapies Program, New York, NY;333. Laboratory for Translational Immunology, Tumor-immunology, University Medical Center Utrecht, Utrecht, The Netherlands;444. Department of BMT, Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom;555. French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker Enfants Malades University Hospital, AP-HP, Paris, France;666. INSERM UMR 1163, Laboratory of Human Genetics of Infectious Diseases, Necker Branch, Paris, France;777. Center for Pediatrics and Center for Chronic Immunodeficiency, Medical Center, University of Freiburg, Freiburg, Germany;888. College de France, Paris, France
Abstract:
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  • Keywords:on behalf of  CD40 ligand  hematopoietic stem cell transplantation  X-linked hyper-IgM syndrome  primary immunodeficiency  BM  Bone marrow  CD40L  CD40 ligand  DFS  Disease-free survival  DLI  Donor lymphocyte infusion  EBMT  European Society for Blood and Marrow Transplantation  EFS  Event-free survival  ESID  European Society for Immunodeficiencies  FU  Follow-up  GVHD  Graft-versus-host disease  HSCT  Hematopoietic stem cell transplantation  IEWP  Inborn Errors Working Party  MAC  Myeloablative conditioning  MAC low tox  Myeloablative conditioning with low toxicity  MMFD  Mismatched family donor  MMUD  Mismatched unrelated donor  MSD  Matched sibling donor  MUD  Matched unrelated donor  NMA  Nonmyeloablative  OS  Overall survival  PBSC  Peripheral blood stem cell  PID  Primary immunodeficiency  PIDTC  Primary Immune Deficiency Treatment Consortium  RIC  Reduced-intensity conditioning  SCETIDE  Stem Cell Transplant for Primary Immune Deficiencies in Europe  UCB  Umbilical cord blood
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