Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA

Por: Hansen BE, Vandriel SM, Vig P, Garner W, Mogul DB, Loomes KM, Piccoli DA, Rand EB, Jankowska I, Czubkowski P, Gliwicz-Miedzinska D, Gonzales EM, Jacquemin E, Bouligand J, D'Antiga L, Nicastro E, Arnell H, Fischler B, Sokal É, Demaret T, Siew S, Stormon M, Karpen SJ, Romero R, Ebel NH, Feinstein JA, Roberts AJ, Evans HM, Sundaram SS, Chaidez A, Hardikar W, Shankar S, Fischer RT, Lacaille F, Debray D, Lin HC, Jensen MK, Jaramillo C, Karthikeyan P, Indolfi G, Verkade HJ, Larson-Nath C, Quiros-Tejeira RE, Valentino PL, Rogalidou M, Dezsofi A, Squires JE, Schwarz K, Calvo PL, Bernabeu JQ, Zizzo AN, Nebbia G, Bulut P, Santos-Silva E, Fawaz R, Nastasio S, Karnsakul W, Tamara ML, Molera C, Kelly D, Sandahl TD, Jimenez-Rivera C, Banales JM, Mujawar Q, Li LT, She H, Wang JS, Kim KM, Oh SH, Sanchez MC, Cavalieri ML, Lee WS, Hajinicolaou C, Lertudomphonwanit C, Waisbourd-Zinman O, Arikan C, Alam S, Carvalho E, Melere M, Eshun J, Önal Z, Desai DM, Wiecek S, Pinto RB, Wolters VM, Garcia J, Beretta M, Kerkar N, Brecelj J, Rock N, Lurz E, Blondet N, Shah U, Thompson RJ and Kamath BM

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Publicada: 1 jun 2024 Ahead of Print: 1 dic 2023

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Filiaciones:
Hansen BE:
 Toronto General Hospital University Health Network, Toronto, ON, Canada

 Institute of Health Policy, Management and Evaluation, Toronto, ON, Canada

 Department of Epidemiology, Erasmus MC, Rotterdam, Netherlands

Vandriel SM:
 The Hospital for Sick Children and the University of Toronto, Division of Gastroenterology, Hepatology and Nutrition, Toronto, ON, Canada

Vig P:
 Mirum Pharmaceuticals, Inc., Foster City, CA, USA

Garner W:
 Mirum Pharmaceuticals, Inc., Foster City, CA, USA

Mogul DB:
 Mirum Pharmaceuticals, Inc., Foster City, CA, USA

Loomes KM:
 The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Division of Gastroenterology, Hepatology and Nutrition, Philadelphia, PA, USA

Piccoli DA:
 The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Division of Gastroenterology, Hepatology and Nutrition, Philadelphia, PA, USA

Rand EB:
 The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Division of Gastroenterology, Hepatology and Nutrition, Philadelphia, PA, USA

Jankowska I:
 The Children's Memorial Health Institute, Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics, Warsaw, Poland

Czubkowski P:
 The Children's Memorial Health Institute, Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics, Warsaw, Poland

Gliwicz-Miedzinska D:
 The Children's Memorial Health Institute, Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics, Warsaw, Poland

Gonzales EM:
 Service d'Hépatologie et de Transplantation Hépatique Pédiatriques, Centre de Référence de l'Atrésie des Voies Biliaires et des Cholestases Génétiques (AVB-CG), FSMR FILFOIE, ERN RARE LIVER, Hôpital Bicêtre, AP-HP, Faculté de Médecine Paris-Saclay, Le Kremlin-Bicêtre, and Inserm U1193, Hépatinov, Université Paris-Saclay, Orsay, France

Jacquemin E:
 Service d'Hépatologie et de Transplantation Hépatique Pédiatriques, Centre de Référence de l'Atrésie des Voies Biliaires et des Cholestases Génétiques (AVB-CG), FSMR FILFOIE, ERN RARE LIVER, Hôpital Bicêtre, AP-HP, Faculté de Médecine Paris-Saclay, Le Kremlin-Bicêtre, and Inserm U1193, Hépatinov, Université Paris-Saclay, Orsay, France

Bouligand J:
 Service de Génétique Moléculaire, Pharmacogénétique et Hormonologie, Hôpitaux Universitaires Paris-Saclay, Assistance PubliqueHôpitaux de Paris, Centre Hospitalier Universitaire de Bicêtre, Le Kremlin-Bicêtre, France

D'Antiga L:
 Ospedale Papa Giovanni XXIII, Pediatric Hepatology, Gastroenterology and Transplantation, Bergamo, Italy

Nicastro E:
 Ospedale Papa Giovanni XXIII, Pediatric Hepatology, Gastroenterology and Transplantation, Bergamo, Italy

Arnell H:
 Astrid Lindgren Children's Hospital, Department of Paediatric Gastroenterology, Hepatology and Nutrition, Karolinska University Hospital and Department of Women's and Children's Health, Karolinska Institutet, Stockholm, Sweden

Fischler B:
 Astrid Lindgren Children's Hospital, Department of Paediatric Gastroenterology, Hepatology and Nutrition, Karolinska University Hospital and CLINTEC, Karolinska Institutet, Stockholm, Sweden

Sokal É:
 Cliniques Universitaires Saint-Luc, Service De Gastroentérologie & Hépatologie Pédiatrique, Brussels, Belgium

Demaret T:
 Cliniques Universitaires Saint-Luc, Service De Gastroentérologie & Hépatologie Pédiatrique, Brussels, Belgium

Siew S:
 The Children's Hospital at Westmead, Department of Gastroenterology, Sydney, NSW, Australia

Stormon M:
 The Children's Hospital at Westmead, Department of Gastroenterology, Sydney, NSW, Australia

Karpen SJ:
 Children's Healthcare of Atlanta & Emory University School of Medicine, Division of Pediatric Gastroenterology, Hepatology & Nutrition, Atlanta, Georgia

Romero R:
 Children's Healthcare of Atlanta & Emory University School of Medicine, Division of Pediatric Gastroenterology, Hepatology & Nutrition, Atlanta, Georgia

Ebel NH:
 Division of Gastroenterology, Department of Pediatrics, Stanford University School of Medicine, Palo Alto, CA, USA

Feinstein JA:
 Department of Pediatrics (Cardiology), Stanford University School of Medicine, Lucile Packard Children's Hospital, Palo Alto, CA, USA

Roberts AJ:
 Starship Child Health, Department of Paediatric Gastroenterology, Auckland, New Zealand

Evans HM:
 Starship Child Health, Department of Paediatric Gastroenterology, Auckland, New Zealand

Sundaram SS:
 Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado School of Medicine, Aurora, CO, USA

Chaidez A:
 Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado School of Medicine, Aurora, CO, USA

Hardikar W:
 Royal Children's Hospital, Department of Gastroenterology and Clinical Nutrition, Melbourne, Vic, Australia

Shankar S:
 Mazumdar Shaw Medical Center, Narayana Health, Bangalore, India

Fischer RT:
 Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology, Kansas City, MO, USA

Lacaille F:
 Department of Pediatric Gastroenterology, and Nutrition, Necker-Enfants Malades Hospital, University of Paris, Paris, France

Debray D:
 Pediatric liver unit, National Reference Centre for Rare Pediatric Liver Diseases (Biliary Atresia and Genetic Cholestasis), FILFOIE, ERN RARE LIVER, Necker-Enfants Malades Hospital, University of Paris, Paris, France

Lin HC:
 Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics, Portland, OR, USA

Jensen MK:
 University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition, Primary Children's Hospital, Salt Lake City, UT, USA

Jaramillo C:
 University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition, Primary Children's Hospital, Salt Lake City, UT, USA

Karthikeyan P:
 Leeds Teaching Hospitals NHS Trust, Leeds Children's Hospital, Leeds, UK

Indolfi G:
 Department Neurofarba, University of Florence and Meyer Children's University Hospital, Paediatric and Liver Unit, Florence, Italy

Verkade HJ:
 University Medical Center Groningen, Department of Pediatrics, Center for Liver, Digestive, and Metabolic Diseases, Groningen, The Netherlands

Larson-Nath C:
 University of Minnesota, Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Minneapolis, MN, USA

Quiros-Tejeira RE:
 Children's Hospital & Medical Center and University of Nebraska Medical Center, Department of Pediatrics, Omaha, NE, USA

Valentino PL:
 Gastroenterology & Hepatology Division, Department of Pediatrics, University of Washington, Seattle Children's Hospital, Seattle, WA, USA

Rogalidou M:
 Division of Gastroenterology & Hepatology, "Agia Sofia" Children's Hospital, First Department of Pediatrics, University of Athens, Athens, Greece

Dezsofi A:
 First Department of Paediatrics, Semmelweis University, Budapest, Hungary

Squires JE:
 University of Pittsburgh School of Medicine, Division of Pediatric Gastroenterology and Hepatology, Department of Pediatrics, Pittsburgh, PA, USA

Schwarz K:
 University of California San Diego, Rady Children's Hospital San Diego, Division of Pediatric Gastroenterology, San Diego, CA, USA

Calvo PL:
 Pediatric Gastroenterology Unit, Regina Margherita Children's Hospital, Azienda Ospedaliera-Universitaria Citta' della Salute e della Scienza, Turin, Italy

Bernabeu JQ:
 Hospital Universitari Vall d'Hebron, Pediatric Hepatology and Liver Transplant Department, Barcelona, Spain

 Department of Liver and Gastrointestinal Diseases, Biodonostia Health Research Institute - Donostia University Hospital, University of the Basque Country (UPV/EHU), San Sebastian, Spain

Zizzo AN:
 Children's Hospital, London Health Sciences Centre, Division of Paediatric Gastroenterology and Hepatology, Western University, London, ON, Canada

Nebbia G:
 Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Servizio di Epatologia Pediatrica, Milan, Italy

Bulut P:
 Phoenix Children's Hospital, Division of Pediatric Gastroenterology and Hepatology, Phoenix, AZ, USA

Santos-Silva E:
 Centro Hospitalar Universitário Do Porto, Pediatric Gastroenterology Unit, Porto, Portugal

Fawaz R:
 Yale University School of Medicine, Department of Pediatrics, New Haven, CT, USA

Nastasio S:
 Boston Children's Hospital and Harvard Medical School, Division of Gastroenterology, Hepatology, & Nutrition, Boston, MA, USA

Karnsakul W:
 Johns Hopkins University School of Medicine, Department of Pediatrics, Baltimore, MD, USA

Tamara ML:
 Paediatric Gastroenterology Unit, Cruces University Hospital, Bilbao, Spain

Molera C:
 Pediatric Gastroenterology Hepatology and Nutrition Unit, Hospital Sant Joan de Déu, Esplugues de Llobregat, Spain

Kelly D:
 Liver Unit, Birmingham Women's & Children's Hospital NHS Trust and University of Birmingham, Birmingham, UK

Sandahl TD:
 Department of Hepatology and Gastroenterology, Aarhus University Hospital, Aarhus, Denmark

Jimenez-Rivera C:
 Children's Hospital of Eastern Ontario, Division of Gastroenterology, Hepatology and Nutrition, Ottawa, ON, Canada

Banales JM:
 Department of Hepatology and Gastroenterology, Biodonostia Health Research Institute - Donostia University Hospital, Universidad del País Vasco (UPV/EHU), Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBERehd), San Sebastián, Spain

Mujawar Q:
 University of Manitoba, Section of Pediatric Gastroenterology, Department of Pediatrics, Winnipeg, MB, Canada

Li LT:
 Children's Hospital of Fudan University, The Center for Pediatric Liver Diseases, Shanghai, China

She H:
 Children's Hospital of Fudan University, The Center for Pediatric Liver Diseases, Shanghai, China

Wang JS:
 Children's Hospital of Fudan University, The Center for Pediatric Liver Diseases, Shanghai, China

Kim KM:
 Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, South Korea

Oh SH:
 Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, South Korea

Sanchez MC:
 Hospital Italiano Buenos Aires, Pediatric Gastroenterology and Hepatology Division, Buenos Aires, Argentina

Cavalieri ML:
 Hospital Italiano Buenos Aires, Pediatric Gastroenterology and Hepatology Division, Buenos Aires, Argentina

Lee WS:
 Faculty of Medicine, Department of Paediatrics, University of Malaya, Kuala Lumpur, Malaysia

Hajinicolaou C:
 Division of Paediatric Gastroenterology, Chris Hani Baragwanath Academic Hospital, Department of Paediatrics and Child Health, University of the Witwatersrand, Johannesburg, South Africa

Lertudomphonwanit C:
 Ramathibodi Hospital Mahidol University, Division of Gastroenterology, Department of Pediatrics, Bangkok, Thailand

Waisbourd-Zinman O:
 Schneider Children's Medical Center of Israel, Institute of Gastroenterology, Nutrition and Liver Diseases, Petah Tikva, Sackler Faculty of Medicine, Tel-Aviv University, Israel

Arikan C:
 Koc University School of Medicine, Department of Pediatric Gastroenterology and Organ Transplant, Istanbul, Turkey

Alam S:
 Institute of Liver and Biliary Sciences, Department of Pediatric Hepatology, New Delhi, India

Carvalho E:
 Pediatric Gastroenterology Department, Hospital de Base do Distrito Federal, Hospital da Crianca de Brasília, Centro Universitário de Brasília, Brasília, DF, Brazil

Melere M:
 Pediatric Gastroenterology Service, Hospital da Criança Santo Antônio, Universidade Federal de Ciências da Saúde de Porto Alegre, Complexo Hospitalar Santa Casa, Porto Alegre, RS, Brazil

Eshun J:
 Department of Pediatric Gastroenterology, Le Bonheur Children's Hospital and The University of Tennessee Health Science Center, Memphis, Tennessee

Önal Z:
 Pediatric Gastroenterology, Hepatology and Nutrition Department, Istanbul University Istanbul Medical Faculty, Istanbul, Turkey

Desai DM:
 Solid Organ Transplant Department, Children's Health - Children's Medical Center, Dallas, Texas

Wiecek S:
 Medical University of Silesia in Katowice, Department of Pediatrics, Katowice, Poland

Pinto RB:
 Division of Pediatric Gastroenterology of Hospital da Criança Conceição do Grupo Hospitalar Conceição, Porto Alegre, RS, Brazil

Wolters VM:
 Department of Pediatric Gastroenterology, University Medical Center Utrecht, Utrecht, Netherlands

Garcia J:
 Division of Pediatric Gastroenterology, Hepatology and Nutrition/Miami Transplant Institute, University of Miami, Miami, Florida

Beretta M:
 Faculty of Health Sciences, Wits Donald Gordon Medical Centre, University of the Witwatersrand, Johannesburg, South Africa

Kerkar N:
 University of Rochester Medical Center, Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology and Nutrition, Rochester, NY, USA

Brecelj J:
 University Medical Center Ljubljana, Pediatric Gastroenterology, Hepatology and Nutrition, and Department of Pediatrics, Faculty of Medicine, Ljubljana, Slovenia

Rock N:
 Swiss Pediatric Liver Center, Division of Pediatric Specialties, Department of Pediatrics, Gynecology, and Obstetrics, University Hospitals Geneva and University of Geneva, Geneva, Switzerland

Lurz E:
 Department of Pediatrics, Dr. von Hauner Children's Hospital, University Hospital, LMU Munich, Munich, Germany

Blondet N:
 Gastroenterology & Hepatology Division, Department of Pediatrics, University of Washington, Seattle Children's Hospital, Seattle, WA, USA

Shah U:
 Harvard Medical School, Massachusetts General Hospital for Children, Boston, MA, USA

Thompson RJ:
 Institute of Liver Studies, King's College London, London, UK

Kamath BM:
 The Hospital for Sick Children and the University of Toronto, Division of Gastroenterology, Hepatology and Nutrition, Toronto, ON, Canada