换一批
换一批Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.
第一作者：
Federica,Barzaghi
第一单位：
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.
作者：
Federica,Barzaghi [1]
;
Laura Cristina,Amaya Hernandez [2]
;
Benedicte,Neven [3]
;
Silvia,Ricci [4]
;
Zeynep Yesim,Kucuk [5]
;
Jack J,Bleesing [5]
;
Zohreh,Nademi [6]
;
Mary Anne,Slatter [6]
;
Erlinda Rose,Ulloa [7]
;
Anna,Shcherbina [8]
;
Anna,Roppelt [8]
;
Austen,Worth [9]
;
Juliana,Silva [10]
;
Alessandro,Aiuti [11]
;
Luis,Murguia-Favela [12]
;
Carsten,Speckmann [13]
;
Magda,Carneiro-Sampaio [14]
;
Juliana Folloni,Fernandes [15]
;
Safa,Baris [16]
;
Ahmet,Ozen [16]
;
Elif,Karakoc-Aydiner [16]
;
Ayca,Kiykim [16]
;
Ansgar,Schulz [17]
;
Sandra,Steinmann [17]
;
Lucia Dora,Notarangelo [18]
;
Eleonora,Gambineri [19]
;
Paolo,Lionetti [20]
;
William Thomas,Shearer [21]
;
Lisa R,Forbes [21]
;
Caridad,Martinez [22]
;
Despina,Moshous [3]
;
Stephane,Blanche [3]
;
Alain,Fisher [3]
;
Frank M,Ruemmele [23]
;
Come,Tissandier [23]
;
Marie,Ouachee-Chardin [24]
;
Frédéric,Rieux-Laucat [25]
;
Marina,Cavazzana [26]
;
Waseem,Qasim [27]
;
Barbarella,Lucarelli [28]
;
Michael H,Albert [29]
;
Ichiro,Kobayashi [30]
;
Laura,Alonso [31]
;
Cristina,Diaz De Heredia [31]
;
Hirokazu,Kanegane [32]
;
Anita,Lawitschka [33]
;
Jong Jin,Seo [34]
;
Marta,Gonzalez-Vicent [35]
;
Miguel Angel,Diaz [35]
;
Rakesh Kumar,Goyal [36]
;
Martin G,Sauer [37]
;
Akif,Yesilipek [38]
;
Minsoo,Kim [39]
;
Yesim,Yilmaz-Demirdag [39]
;
Monica,Bhatia [40]
;
Julie,Khlevner [41]
;
Erick J,Richmond Padilla [42]
;
Silvana,Martino [43]
;
Davide,Montin [43]
;
Olaf,Neth [44]
;
Agueda,Molinos-Quintana [45]
;
Justo,Valverde-Fernandez [46]
;
Arnon,Broides [47]
;
Vered,Pinsk [48]
;
Antje,Ballauf [49]
;
Filomeen,Haerynck [50]
;
Victoria,Bordon [50]
;
Catharina,Dhooge [50]
;
Maria Laura,Garcia-Lloret [51]
;
Robbert G,Bredius [52]
;
Krzysztof,Kałwak [53]
;
Elie,Haddad [54]
;
Markus Gerhard,Seidel [55]
;
Gregor,Duckers [49]
;
Sung-Yun,Pai [56]
;
Christopher C,Dvorak [57]
;
Stephan,Ehl [58]
;
Franco,Locatelli [28]
;
Frederick,Goldman [59]
;
Andrew Richard,Gennery [6]
;
Mort J,Cowan [57]
;
Maria-Grazia,Roncarolo [2]
;
Rosa,Bacchetta [60]
;
Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT)
作者单位：
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.
[1]
Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, Calif.
[2]
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.
[3]
Pediatric Immunology, "Anna Meyer" Children's Hospital, Florence, Italy.
[4]
Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
[5]
Institute of Cellular Medicine, Newcastle University and Children's Bone Marrow Transplant Unit, Great North Children's Hospital, Newcastle Upon Tyne, United Kingdom.
[6]
Pediatrics, Boston Children's Hospital, Boston, Mass.
[7]
Department of Immunology, Research and Clinical Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russia.
[8]
Department of Immunology and Gene Therapy, Great Ormond Street Hospital, London, United Kingdom.
[9]
Department of Stem Cell Transplantation, Great Ormond Street Hospital, London, United Kingdom.
[10]
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.
[11]
Division of Clinical Immunology and Allergy, Department of Pediatrics, Hospital for Sick Children, Toronto, Ontario, Canada.
[12]
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.
[13]
Department of Pediatrics, Faculdade de Medicina da Universidade de São Paulo and Hospital Albert Einstein, São Paulo, Brazil.
[14]
Stem Cell Transplantation Unit, Instituto da Criança, Faculdade de Medicina da Universidade de São Paulo and Hospital Albert Einstein, São Paulo, Brazil.
[15]
Pediatric Allergy and Immunology, Marmara University, Istanbul, Turkey.
[16]
Department of Pediatrics, University Medical Center, Ulm, Germany.
[17]
Pediatric Onco-Hematology and Bone Marrow Transplant (BMT) Unit, Children's Hospital, Spedali Civili, Brescia, Italy.
[18]
Department of Hematology-Oncology: Bone Marrow Transplant (BMT) Unit, "Anna Meyer" Children's Hospital, Florence, Italy.
[19]
Gastroenterology Unit, University of Florence, Department of "NEUROFARBA": Section of Child's Health, "Anna Meyer" Children's Hospital, Florence, Italy.
[20]
Department of Pediatrics, Section of Immunology Allergy Rheumatology, Baylor College of Medicine Texas Children's Hospital, Houston, Tex.
[21]
Department of Pediatric Hematology and Oncology, Baylor College of Medicine Texas Children's Hospital, Houston, Tex.
[22]
Pediatric Gastroenterology unit, Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris Descartes-Université Sorbonne Paris Cité, Paris, France.
[23]
Hematology Unit, Robert Debré Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.
[24]
Institut national de la santé et de la recherche médicale (INSERM) UMR 1163, Laboratory of Immunogenetics of Pediatric Autoimmune Disease, Paris, France.
[25]
Biotherapy Department, Necker-Enfants Malades University Hospital, Paris Descartes -Université Sorbonne Paris Cité, Institut Imagine, Paris, France.
[26]
Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
[27]
Department of Pediatric Hematology-Oncology, University of Pavia, Istituto di Ricovero e Cura a Carattere Scientifico, Bambino Gesù Children's Hospital, Rome, Italy.
[28]
Pediatric Hematology-Oncology, Dr. von Hauner Children's hospital, Ludwig-Maximilians Universität, Munich, Germany.
[29]
Center for Pediatric Allergy and Rheumatology, KKR Sapporo Medical Center, Sapporo, Japan.
[30]
Paediatric Haematology and Oncology, Hospital Universitario Vall D'Hebron, Barcelona, Spain.
[31]
Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan.
[32]
St. Anna Children's Hospital, Medical University Vienna, Vienna, Austria.
[33]
Pediatrics, University of Ulsan College of Medicine and Asan Medical Center, Seoul, Korea.
[34]
Hematopoietic Stem Cell Transplantation Unit, Pediatric Department, Children's University Hospital Niño Jesús, Madrid, Spain.
[35]
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.
[36]
Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany.
[37]
Pediatric Stem Cell Transplantation Unit, Medicalpark Hospital Goztepe and Antalya Hospitals, Antalya, Turkey.
[38]
Pediatric Allergy, Immunology and Rheumatology, Columbia Medical Center, New York, NY.
[39]
Pediatric Hematology, Oncology and Stem Cell Transplantation, Columbia Medical Center, New York, NY.
[40]
Pediatric Gastroenterology, Hepatology, and Nutrition, Columbia Medical Center, New York, NY.
[41]
Pediatric Endocrinology, National Children's Hospital, San Jose, Israel.
[42]
Division of Immunology and Rheumatology, Department of Paediatric Infectious Diseases, University of Turin, Regina Margherita Children's Hospital, Turin, Italy.
[43]
Department of Paediatric Infectious Diseases, Rheumatology and Immunodeficiency, Instituto de Biomedicina de Sevilla/CSIC/Universidad de Sevilla, Seville, Spain.
[44]
Department of Pediatric Hematology, Instituto de Biomedicina de Sevilla/CSIC/Universidad de Sevilla, Seville, Spain.
[45]
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.
[46]
Pediatric Immunology Clinic, Soroka University Medical Center, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer Sheva, Israel.
[47]
Pediatric Ambulatory Care Unit, Soroka University Medical Center, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer Sheva, Israel.
[48]
Department of Pediatrics, Helios Children's Hospital, Krefeld, Germany.
[49]
Department of Pediatrics, Center for Primary Immunodeficiencies, Ghent University Hospital, Ghent, Belgium.
[50]
Division of Pediatric Allergy, Immunology and Rheumatology, University of California-Los Angeles School of Medicine, Los Angeles, Calif.
[51]
Pediatric Immunology, Infections and Stem Cell Transplantation (SCT), Leiden University Medical Center, Leiden, The Netherlands.
[52]
Department of Pediatric Hematology, Oncology and Bone Marrow Transplant (BMT) Unit, Wroclaw Medical University, Wroclaw, Poland.
[53]
Department of Pediatrics, Saint Justine Hospital, University of Montreal, Montreal, Quebec, Canada.
[54]
Division of Pediatric Hematology-Oncology, Research Unit Pediatric Hematology and Immunology, Medical University Graz, Graz, Austria.
[55]
Pediatrics, Boston Children's Hospital, Boston, Mass; Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Mass.
[56]
Pediatric Allergy, Immunology and Bone Marrow Transplant, University of California-San Francisco Benioff Children's Hospital, San Francisco, Calif.
[57]
Center for Chronic Immunodeficiency, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
[58]
Pediatrics, University of Alabama, Birmingham, Ala.
[59]
Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, Calif. Electronic address: roasb@stanford.edu.
[60]
主题词
青少年(Adolescent);成年人(Adult);儿童(Child);儿童, 学龄前(Child, Preschool);糖尿病, 1型(Diabetes Mellitus, Type 1);腹泻(Diarrhea);无病生存(Disease-Free Survival);女(雌)性(Female);随访研究(Follow-Up Studies);叉头转录因子类(Forkhead Transcription Factors);遗传性疾病, X连锁(Genetic Diseases, X-Linked);造血干细胞移植(Hematopoietic Stem Cell Transplantation);人类(Humans);免疫系统疾病(Immune System Diseases);婴儿(Infant);男(雄)性(Male);突变(Mutation);回顾性研究(Retrospective Studies);存活率(Survival Rate)
DOI
10.1016/j.jaci.2017.10.041
PMID
29241729
发布时间
2022-01-29
- 浏览6
The Journal of allergy and clinical immunology
The Journal of allergy and clinical immunology
1036-1049.e5页
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