المؤلفون: Barzaghi F; San Raffaele Telethon Institute for Gene Therapy, Pediatric Immunohematology and Bone Marrow Transplantation Unit, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy., Amaya Hernandez LC; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, Calif., Neven B; Paediatric Immunology, Haematology and Rheumatology Department, Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris Descartes-Université Sorbonne Paris Cité, Institut Imagine, Paris, France., Ricci S; Pediatric Immunology, 'Anna Meyer' Children's Hospital, Florence, Italy., Kucuk ZY; Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio., Bleesing JJ; Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio., Nademi Z; Institute of Cellular Medicine, Newcastle University and Children's Bone Marrow Transplant Unit, Great North Children's Hospital, Newcastle Upon Tyne, United Kingdom., Slatter MA; Institute of Cellular Medicine, Newcastle University and Children's Bone Marrow Transplant Unit, Great North Children's Hospital, Newcastle Upon Tyne, United Kingdom., Ulloa ER; Pediatrics, Boston Children's Hospital, Boston, Mass., Shcherbina A; Department of Immunology, Research and Clinical Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russia., Roppelt A; Department of Immunology, Research and Clinical Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russia., Worth A; Department of Immunology and Gene Therapy, Great Ormond Street Hospital, London, United Kingdom., Silva J; Department of Stem Cell Transplantation, Great Ormond Street Hospital, London, United Kingdom., Aiuti A; San Raffaele Telethon Institute for Gene Therapy, Pediatric Immunohematology and Bone Marrow Transplantation Unit, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy., Murguia-Favela L; Division of Clinical Immunology and Allergy, Department of Pediatrics, Hospital for Sick Children, Toronto, Ontario, Canada., Speckmann C; Department of Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany; Center for Chronic Immunodeficiency, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany., Carneiro-Sampaio M; Department of Pediatrics, Faculdade de Medicina da Universidade de São Paulo and Hospital Albert Einstein, São Paulo, Brazil., Fernandes JF; Stem Cell Transplantation Unit, Instituto da Criança, Faculdade de Medicina da Universidade de São Paulo and Hospital Albert Einstein, São Paulo, Brazil., Baris S; Pediatric Allergy and Immunology, Marmara University, Istanbul, Turkey., Ozen A; Pediatric Allergy and Immunology, Marmara University, Istanbul, Turkey., Karakoc-Aydiner E; Pediatric Allergy and Immunology, Marmara University, Istanbul, Turkey., Kiykim A; Pediatric Allergy and Immunology, Marmara University, Istanbul, Turkey., Schulz A; Department of Pediatrics, University Medical Center, Ulm, Germany., Steinmann S; Department of Pediatrics, University Medical Center, Ulm, Germany., Notarangelo LD; Pediatric Onco-Hematology and Bone Marrow Transplant (BMT) Unit, Children's Hospital, Spedali Civili, Brescia, Italy., Gambineri E; Department of Hematology-Oncology: Bone Marrow Transplant (BMT) Unit, 'Anna Meyer' Children's Hospital, Florence, Italy., Lionetti P; Gastroenterology Unit, University of Florence, Department of 'NEUROFARBA': Section of Child's Health, 'Anna Meyer' Children's Hospital, Florence, Italy., Shearer WT; Department of Pediatrics, Section of Immunology Allergy Rheumatology, Baylor College of Medicine Texas Children's Hospital, Houston, Tex., Forbes LR; Department of Pediatrics, Section of Immunology Allergy Rheumatology, Baylor College of Medicine Texas Children's Hospital, Houston, Tex., Martinez C; Department of Pediatric Hematology and Oncology, Baylor College of Medicine Texas Children's Hospital, Houston, Tex., Moshous D; Paediatric Immunology, Haematology and Rheumatology Department, Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris Descartes-Université Sorbonne Paris Cité, Institut Imagine, Paris, France., Blanche S; Paediatric Immunology, Haematology and Rheumatology Department, Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris Descartes-Université Sorbonne Paris Cité, Institut Imagine, Paris, France., Fisher A; Paediatric Immunology, Haematology and Rheumatology Department, Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris Descartes-Université Sorbonne Paris Cité, Institut Imagine, Paris, France., Ruemmele FM; Pediatric Gastroenterology unit, Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris Descartes-Université Sorbonne Paris Cité, Paris, France., Tissandier C; Pediatric Gastroenterology unit, Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris Descartes-Université Sorbonne Paris Cité, Paris, France., Ouachee-Chardin M; Hematology Unit, Robert Debré Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France., Rieux-Laucat F; Institut national de la santé et de la recherche médicale (INSERM) UMR 1163, Laboratory of Immunogenetics of Pediatric Autoimmune Disease, Paris, France., Cavazzana M; Biotherapy Department, Necker-Enfants Malades University Hospital, Paris Descartes -Université Sorbonne Paris Cité, Institut Imagine, Paris, France., Qasim W; Great Ormond Street Institute of Child Health, University College London, London, United Kingdom., Lucarelli B; Department of Pediatric Hematology-Oncology, University of Pavia, Istituto di Ricovero e Cura a Carattere Scientifico, Bambino Gesù Children's Hospital, Rome, Italy., Albert MH; Pediatric Hematology-Oncology, Dr. von Hauner Children's hospital, Ludwig-Maximilians Universität, Munich, Germany., Kobayashi I; Center for Pediatric Allergy and Rheumatology, KKR Sapporo Medical Center, Sapporo, Japan., Alonso L; Paediatric Haematology and Oncology, Hospital Universitario Vall D'Hebron, Barcelona, Spain., Diaz De Heredia C; Paediatric Haematology and Oncology, Hospital Universitario Vall D'Hebron, Barcelona, Spain., Kanegane H; Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan., Lawitschka A; St. Anna Children's Hospital, Medical University Vienna, Vienna, Austria., Seo JJ; Pediatrics, University of Ulsan College of Medicine and Asan Medical Center, Seoul, Korea., Gonzalez-Vicent M; Hematopoietic Stem Cell Transplantation Unit, Pediatric Department, Children's University Hospital Niño Jesús, Madrid, Spain., Diaz MA; Hematopoietic Stem Cell Transplantation Unit, Pediatric Department, Children's University Hospital Niño Jesús, Madrid, Spain., Goyal RK; Division of Blood and Marrow Transplantation and Cellular Therapies, Department of Pediatrics, Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center, Pittsburgh, Pa., Sauer MG; Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany., Yesilipek A; Pediatric Stem Cell Transplantation Unit, Medicalpark Hospital Goztepe and Antalya Hospitals, Antalya, Turkey., Kim M; Pediatric Allergy, Immunology and Rheumatology, Columbia Medical Center, New York, NY., Yilmaz-Demirdag Y; Pediatric Allergy, Immunology and Rheumatology, Columbia Medical Center, New York, NY., Bhatia M; Pediatric Hematology, Oncology and Stem Cell Transplantation, Columbia Medical Center, New York, NY., Khlevner J; Pediatric Gastroenterology, Hepatology, and Nutrition, Columbia Medical Center, New York, NY., Richmond Padilla EJ; Pediatric Endocrinology, National Children's Hospital, San Jose, Israel., Martino S; Division of Immunology and Rheumatology, Department of Paediatric Infectious Diseases, University of Turin, Regina Margherita Children's Hospital, Turin, Italy., Montin D; Division of Immunology and Rheumatology, Department of Paediatric Infectious Diseases, University of Turin, Regina Margherita Children's Hospital, Turin, Italy., Neth O; Department of Paediatric Infectious Diseases, Rheumatology and Immunodeficiency, Instituto de Biomedicina de Sevilla/CSIC/Universidad de Sevilla, Seville, Spain., Molinos-Quintana A; Department of Pediatric Hematology, Instituto de Biomedicina de Sevilla/CSIC/Universidad de Sevilla, Seville, Spain., Valverde-Fernandez J; Department of Paediatirc Gastroenterology, Hospital Universitario Virgen del Rocío, Instituto de Biomedicina de Sevilla/Unite Mixte de Recherche (UMR) or Mixed Unit of Research Consejo Superior de Investigaciones Científicas (CSIC)/Universidad de Sevilla, Seville, Spain., Broides A; Pediatric Immunology Clinic, Soroka University Medical Center, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer Sheva, Israel., Pinsk V; Pediatric Ambulatory Care Unit, Soroka University Medical Center, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer Sheva, Israel., Ballauf A; Department of Pediatrics, Helios Children's Hospital, Krefeld, Germany., Haerynck F; Department of Pediatrics, Center for Primary Immunodeficiencies, Ghent University Hospital, Ghent, Belgium., Bordon V; Department of Pediatrics, Center for Primary Immunodeficiencies, Ghent University Hospital, Ghent, Belgium., Dhooge C; Department of Pediatrics, Center for Primary Immunodeficiencies, Ghent University Hospital, Ghent, Belgium., Garcia-Lloret ML; Division of Pediatric Allergy, Immunology and Rheumatology, University of California-Los Angeles School of Medicine, Los Angeles, Calif., Bredius RG; Pediatric Immunology, Infections and Stem Cell Transplantation (SCT), Leiden University Medical Center, Leiden, The Netherlands., Kałwak K; Department of Pediatric Hematology, Oncology and Bone Marrow Transplant (BMT) Unit, Wroclaw Medical University, Wroclaw, Poland., Haddad E; Department of Pediatrics, Saint Justine Hospital, University of Montreal, Montreal, Quebec, Canada., Seidel MG; Division of Pediatric Hematology-Oncology, Research Unit Pediatric Hematology and Immunology, Medical University Graz, Graz, Austria., Duckers G; Department of Pediatrics, Helios Children's Hospital, Krefeld, Germany., Pai SY; Pediatrics, Boston Children's Hospital, Boston, Mass; Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Mass., Dvorak CC; Pediatric Allergy, Immunology and Bone Marrow Transplant, University of California-San Francisco Benioff Children's Hospital, San Francisco, Calif., Ehl S; Center for Chronic Immunodeficiency, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany., Locatelli F; Department of Pediatric Hematology-Oncology, University of Pavia, Istituto di Ricovero e Cura a Carattere Scientifico, Bambino Gesù Children's Hospital, Rome, Italy., Goldman F; Pediatrics, University of Alabama, Birmingham, Ala., Gennery AR; Institute of Cellular Medicine, Newcastle University and Children's Bone Marrow Transplant Unit, Great North Children's Hospital, Newcastle Upon Tyne, United Kingdom., Cowan MJ; Pediatric Allergy, Immunology and Bone Marrow Transplant, University of California-San Francisco Benioff Children's Hospital, San Francisco, Calif., Roncarolo MG; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, Calif., Bacchetta R; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, Calif. Electronic address: roasb@stanford.edu.

مؤلفون مشاركون: Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT)

المصدر: The Journal of allergy and clinical immunology [J Allergy Clin Immunol] 2018 Mar; Vol. 141 (3), pp. 1036-1049.e5. Date of Electronic Publication: 2017 Dec 11.

نوع المنشور: Clinical Trial; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Mosby Country of Publication: United States NLM ID: 1275002 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-6825 (Electronic) Linking ISSN: 00916749 NLM ISO Abbreviation: J Allergy Clin Immunol Subsets: MEDLINE

مواضيع طبية MeSH: Diarrhea*/genetics , Diarrhea*/immunology , Diarrhea*/mortality , Diarrhea*/therapy , Forkhead Transcription Factors*/genetics , Forkhead Transcription Factors*/immunology , Genetic Diseases, X-Linked*/genetics , Genetic Diseases, X-Linked*/immunology , Genetic Diseases, X-Linked*/mortality , Genetic Diseases, X-Linked*/therapy , Hematopoietic Stem Cell Transplantation* , Immunosuppression Therapy* , Mutation*, Diabetes Mellitus, Type 1/*congenital , Immune System Diseases/*congenital, Adolescent ; Adult ; Allografts ; Child ; Child, Preschool ; Diabetes Mellitus, Type 1/genetics ; Diabetes Mellitus, Type 1/immunology ; Diabetes Mellitus, Type 1/mortality ; Diabetes Mellitus, Type 1/therapy ; Disease-Free Survival ; Female ; Follow-Up Studies ; Humans ; Immune System Diseases/genetics ; Immune System Diseases/immunology ; Immune System Diseases/mortality ; Immune System Diseases/therapy ; Infant ; Male ; Retrospective Studies ; Survival Rate

SCR Disease Name: Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome