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

Autor: Hansen BE; Department of Hepatology, Toronto General Hospital University Health Network, Toronto, Ontario, Canada.; Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada.; Department of Epidemiology, Erasmus MC, Rotterdam, The Netherlands., Vandriel SM; Department of Paediatrics, Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada., Vig P; Department of Scientific and Medical Affairs, Mirum Pharmaceuticals, Inc., Foster City, California, USA., Garner W; Department of Scientific and Medical Affairs, Mirum Pharmaceuticals, Inc., Foster City, California, USA., Mogul DB; Department of Scientific and Medical Affairs, Mirum Pharmaceuticals, Inc., Foster City, California, USA., Loomes KM; Department of Pathology and Laboratory Medicine, Division of Gastroenterology, Hepatology and Nutrition, The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA., Piccoli DA; Department of Pathology and Laboratory Medicine, Division of Gastroenterology, Hepatology and Nutrition, The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA., Rand EB; Department of Pathology and Laboratory Medicine, Division of Gastroenterology, Hepatology and Nutrition, The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA., Jankowska I; Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics, The Children's Memorial Health Institute, Warsaw, Poland., Czubkowski P; Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics, The Children's Memorial Health Institute, Warsaw, Poland., Gliwicz-Miedzińska D; Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics, The Children's Memorial Health Institute, Warsaw, Poland., Gonzales EM; Department of Pediatric Hepatology and Liver Transplantation, 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; Department of Pediatric Hepatology and Liver Transplantation, 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; Department of Molecular Genetics, Pharmacogenetics and Hormonology, 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; Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Ospedale Papa Giovanni XXIII, Pediatric Hepatology, Gastroenterology and Transplantation, Bergamo, Italy., Nicastro E; Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Ospedale Papa Giovanni XXIII, Pediatric Hepatology, Gastroenterology and Transplantation, Bergamo, Italy., Arnell H; Department of Paediatric Gastroenterology, Hepatology and Nutrition, Astrid Lindgren Children's Hospital, Karolinska University Hospital and Department of Women's and Children's Health, Karolinska Institutet, Stockholm, Sweden., Fischler B; Department of Paediatric Gastroenterology, Hepatology and Nutrition, Astrid Lindgren Children's Hospital, Karolinska University Hospital and CLINTEC, Karolinska Institutet, Stockholm, Sweden., Sokal É; Department of Pediatric GI and Hepatology, Cliniques Universitaires Saint-Luc, Service De Gastroentérologie & Hépatologie Pédiatrique, Brussels, Belgium., Demaret T; Department of Pediatric GI and Hepatology, Cliniques Universitaires Saint-Luc, Service De Gastroentérologie & Hépatologie Pédiatrique, Brussels, Belgium., Siew S; Department of Gastroenterology, The Children's Hospital at Westmead, Sydney, New South Wales, Australia., Stormon M; Department of Gastroenterology, The Children's Hospital at Westmead, Sydney, New South Wales, Australia., Karpen SJ; Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology & Nutrition, Children's Healthcare of Atlanta & Emory University School of Medicine, Atlanta, Georgia, USA., Romero R; Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology & Nutrition, Children's Healthcare of Atlanta & Emory University School of Medicine, Atlanta, Georgia, USA., Ebel NH; Department of Pediatrics, Division of Gastroenterology, Stanford University School of Medicine, Palo Alto, California, USA., Feinstein JA; Department of Pediatrics (Cardiology), Stanford University School of Medicine, Lucile Packard Children's Hospital, Palo Alto, California, 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, Colorado, 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, Colorado, USA., Hardikar W; Department of Gastroenterology and Clinical Nutrition, Royal Children's Hospital, Melbourne, Victoria, Australia., Shankar S; Department of Pediatrics, Mazumdar Shaw Medical Center, Narayana Health, Bangalore, Karnataka, India., Fischer RT; Department of Gastroenterology, Children's Mercy Kansas City, Section of Hepatology, Kansas City, Missouri, USA., Lacaille F; Department of Pediatric Gastroenterology and Nutrition, Necker-Enfants Malades Hospital, University of Paris, Paris, France., Debray D; Department of Pediatric Gastroenterology and Hepatology, 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; Department of Pediatrics, Division of Pediatric Gastroenterology, Oregon Health and Science University, Portland, Oregon, USA., Jensen MK; Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology and Nutrition, Primary Children's Hospital, University of Utah, Salt Lake City, Utah, USA., Jaramillo C; Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology and Nutrition, Primary Children's Hospital, University of Utah, Salt Lake City, Utah, USA., Karthikeyan P; Department of Pediatrics, 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; Department of Pediatrics, Center for Liver, Digestive, and Metabolic Diseases, University Medical Center Groningen, Groningen, The Netherlands., Larson-Nath C; Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology, and Nutrition, University of Minnesota, Minneapolis, Minnesota, USA., Quiros-Tejeira RE; Department of Pediatrics, Children's Hospital & Medical Center and University of Nebraska Medical Center, Omaha, Nebraska, USA., Valentino PL; Department of Pediatrics, Gastroenterology & Hepatology Division, University of Washington, Seattle Children's Hospital, Seattle, Washington, USA., Rogalidou M; First Department of Pediatrics, Division of Gastroenterology & Hepatology, 'Agia Sofia' Children's Hospital, University of Athens, Athens, Greece., Dezsőfi A; First Department of Paediatrics, Semmelweis University, Budapest, Hungary., Squires JE; Department of Pediatrics, Division of Pediatric Gastroenterology and Hepatology, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, USA., Schwarz K; Department of Pediatrics, Division of Pediatric Gastroenterology, Rady Children's Hospital San Diego, University of California San Diego, San Diego, California, USA., Calvo PL; Department of Pediatrics, Pediatric Gastroenterology Unit, Regina Margherita Children's Hospital, Azienda Ospedaliera-Universitaria Citta' della Salute e della Scienza, Turin, Italy., Bernabeu JQ; Pediatric Hepatology and Liver Transplant Department, Hospital Universitari Vall d'Hebron, 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; Department of Paediatrics, Division of Paediatric Gastroenterology and Hepatology, London Health Sciences Centre, Children's Hospital, Western University, London, Ontario, Canada., Nebbia G; Department of Pediatric Hepatology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Servizio di Epatologia Pediatrica, Milan, Italy., Bulut P; Department of Pediatrics, Division of Pediatric Gastroenterology and Hepatology, Phoenix Children's Hospital, Phoenix, Arizona, USA., Santos-Silva E; Department of Pediatrics, Centro Hospitalar Universitário Do Porto, Pediatric Gastroenterology Unit, Porto, Portugal., Fawaz R; Department of Pediatrics, Yale University School of Medicine, New Haven, Connecticut, USA., Nastasio S; Department of Pediatrics, Division of Gastroenterology, Hepatology, & Nutrition, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts, US A., Karnsakul W; Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Tamara ML; Department of Pediatrics, Paediatric Gastroenterology Unit, Cruces University Hospital, Bilbao, Spain., Busoms CM; Department of Gastroenterology, Hepatology and Nutrition, Pediatric Gastroenterology Hepatology and Nutrition Unit, Hospital Sant Joan de Déu, Esplugues de Llobregat, Spain., Kelly DA; Department of Paediatric Hepatology, 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; Department of Paediatrics, Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada., Banales JM; Department of Hepatology and Gastroenterology, Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBERehd), Biodonostia Health Research Institute-Donostia University Hospital, Universidad del País Vasco (UPV/EHU), San Sebastián, Spain., Mujawar Q; Section of Pediatric Gastroenterology, Department of Pediatrics, University of Manitoba, Winnipeg, Manitoba, Canada., Li LT; Department of Pediatric Gastroenterology, Children's Hospital of Fudan University, The Center for Pediatric Liver Diseases, Shanghai, China., She H; Department of Pediatric Gastroenterology, Children's Hospital of Fudan University, The Center for Pediatric Liver Diseases, Shanghai, China., Wang JS; Department of Pediatric Gastroenterology, 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; Department of Pediatric Gastroenterology, Pediatric Gastroenterology and Hepatology Division, Hospital Italiano Buenos Aires, Buenos Aires, Argentina., Cavalieri ML; Department of Pediatric Gastroenterology, Pediatric Gastroenterology and Hepatology Division, Hospital Italiano Buenos Aires, Buenos Aires, Argentina., Lee WS; Department of Paediatrics, Faculty of Medicine, University of Malaya, Kuala Lumpur, Malaysia., Hajinicolaou C; Department of Paediatrics and Child Health, Division of Paediatric Gastroenterology, Chris Hani Baragwanath Academic Hospital, University of the Witwatersrand, Johannesburg, South Africa., Lertudomphonwanit C; Department of Pediatrics, Division of Gastroenterology, Ramathibodi Hospital Mahidol University, Bangkok, Thailand., Waisbourd-Zinman O; Department of Pediatrics, Schneider Children's Medical Center of Israel, Institute of Gastroenterology, Nutrition and Liver Diseases, Petah Tikva, Sackler Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel., Arikan C; Department of Pediatric Gastroenterology and Organ Transplant, Koç University School of Medicine, Istanbul, Turkey., Alam S; Department of Pediatric Hepatology, Institute of Liver and Biliary Sciences, New Delhi, India., Carvalho E; Pediatric Gastroenterology Department, Hospital de Base do Distrito Federal, Hospital da Criança de Brasília, Centro Universitário de Brasília, Brasília, DF, Brazil., Melere M; Departamento de Gastroenterologia e Hepatologia Pediátrica, 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, USA., 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, USA., Wiecek S; Department of Pediatrics, Medical University of Silesia in Katowice, Katowice, Poland., Pinto RB; Department of Pediatric Gastroenterology, 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, The Netherlands., Garcia J; Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology and Nutrition/Miami Transplant Institute, University of Miami, Miami, Florida, USA., Beretta M; Department of Pediatric Intensive Care, Wits Donald Gordon Medical Centre, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa., Kerkar N; Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology and Nutrition, University of Rochester Medical Center, Rochester, New York, USA., Brecelj J; Pediatric Gastroenterology, Hepatology and Nutrition, and Department of Pediatrics, Faculty of Medicine, University Medical Center Ljubljana, Ljubljana, Slovenia., Rock N; Department of Pediatrics, Gynecology, and Obstetrics, Division of Pediatric Specialties, Swiss Pediatric Liver Center, 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; Department of Pediatrics, Gastroenterology & Hepatology Division, University of Washington, Seattle Children's Hospital, Seattle, Washington, USA., Shah U; Department of Pediatrics, Harvard Medical School, Massachusetts General Hospital for Children, Boston, Massachusetts, USA., Thompson RJ; Department of Inflammation Biology, Institute of Liver Studies, King's College London, London, UK., Kamath BM; Department of Paediatrics, Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada.

Publikováno v: Hepatology (Baltimore, Md.) [Hepatology] 2024 Jun 01; Vol. 79 (6), pp. 1279-1292. Date of Electronic Publication: 2023 Dec 25.

Impact of Share 35 liver transplantation allocation in Australia and New Zealand.

Autor: Fink MA; Department of Surgery, Austin Health, The University of Melbourne, Melbourne, Victoria, Australia.; Victorian Liver Transplant Unit, Austin Health, Melbourne, Victoria, Australia.; Australia and New Zealand Liver and Intestinal Transplant Registry, Melbourne, Australia., Gow PJ; Victorian Liver Transplant Unit, Austin Health, Melbourne, Victoria, Australia., McCaughan GW; University of Sydney, Sydney, New South Wales, Australia.; Liver Injury and Cancer, Centenary Institute, Camperdown, New South Wales, Australia.; Australian National Liver Transplant Unit, Royal Prince Alfred Hospital, Sydney, Australia., Hodgkinson P; Queensland Liver Transplant Service, Princess Alexandra Hospital and Queensland Children's Hospital, Brisbane, Australia., Chen J; Flinders Medical Centre, Adelaide, South Australia, Australia., McCall J; University of Auckland, Auckland, New Zealand.; New Zealand Liver Transplant Service, Auckland City Hospital, Auckland, New Zealand., Jaques B; Western Australian Liver Transplant Unit, Sir Charles Gairdner Hospital, Perth, Western Australia, Australia., Crawford M; University of Sydney, Sydney, New South Wales, Australia.; Australian National Liver Transplant Unit, Royal Prince Alfred Hospital, Sydney, Australia., Strasser SI; University of Sydney, Sydney, New South Wales, Australia.; Australian National Liver Transplant Unit, Royal Prince Alfred Hospital, Sydney, Australia., Hardikar W; Gastroenterology and Clinical Nutrition Department Royal Children's Hospital, Melbourne, Victoria, Australia., Brooke-Smith M; Flinders Medical Centre, Adelaide, South Australia, Australia., Starkey G; Victorian Liver Transplant Unit, Austin Health, Melbourne, Victoria, Australia., Jeffrey GP; Western Australian Liver Transplant Unit, Sir Charles Gairdner Hospital, Perth, Western Australia, Australia.; Medical School, The University of Western Australia, Perth, Western Australia, Australia., Gane E; University of Auckland, Auckland, New Zealand.; New Zealand Liver Transplant Service, Auckland City Hospital, Auckland, New Zealand., Stormon M; University of Sydney, Sydney, New South Wales, Australia.; Australian National Liver Transplantation Service, Children's Hospital at Westmead, Sydney, New South Wales, Australia., Evans H; University of Auckland, Auckland, New Zealand.; Department of Paediatric Gastroenterology, Starship Child Health, Auckland, New Zealand., Tallis C; Queensland Liver Transplant Service, Princess Alexandra Hospital and Queensland Children's Hospital, Brisbane, Australia., Byrne AJ; Australia and New Zealand Liver and Intestinal Transplant Registry, Melbourne, Australia., Jones RM; Department of Surgery, Austin Health, The University of Melbourne, Melbourne, Victoria, Australia.; Victorian Liver Transplant Unit, Austin Health, Melbourne, Victoria, Australia.

Publikováno v: Clinical transplantation [Clin Transplant] 2024 Jan; Vol. 38 (1), pp. e15203. Date of Electronic Publication: 2023 Dec 13.

Natural history of liver disease in a large international cohort of children with Alagille syndrome: Results from the GALA study.

Autor: Vandriel SM; Division of Gastroenterology, Hepatology and Nutrition , The Hospital for Sick Children and the University of Toronto , Toronto , Canada., Li LT; The Center for Pediatric Liver Diseases , Children's Hospital of Fudan University , Shanghai , China., She H; The Center for Pediatric Liver Diseases , Children's Hospital of Fudan University , Shanghai , China., Wang JS; The Center for Pediatric Liver Diseases , Children's Hospital of Fudan University , Shanghai , China., Gilbert MA; Division of Genomic Diagnostics , Department of Pathology and Laboratory Medicine , The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine , Philadelphia , Pennsylvania , USA., Jankowska I; Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics , The Children's Memorial Health Institute , Warsaw , Poland., Czubkowski P; Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics , The Children's Memorial Health Institute , Warsaw , Poland., Gliwicz-Miedzińska D; Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics , The Children's Memorial Health Institute , Warsaw , Poland., Gonzales EM; Pediatric Hepatology and Liver Transplantation Unit , National Reference Centre for Rare Pediatric Liver Diseases (Biliary Atresia and Genetic Cholestasis) , FILFOIE, ERN RARE LIVER, Bicêtre Hospital, AP-HP and Inserm U1193, Hepatinov, Université Paris-Saclay , Le Kremlin-Bicêtre , France., Jacquemin E; Pediatric Hepatology and Liver Transplantation Unit , National Reference Centre for Rare Pediatric Liver Diseases (Biliary Atresia and Genetic Cholestasis) , FILFOIE, ERN RARE LIVER, Bicêtre Hospital, AP-HP and Inserm U1193, Hepatinov, Université Paris-Saclay , Le Kremlin-Bicêtre , France., Bouligand J; Service de Génétique Moléculaire, Pharmacogénétique et Hormonologie, Hôpitaux Universitaires Paris-Saclay , Assistance Publique-Hôpitaux de Paris , Centre Hospitalier Universitaire de Bicêtre , Le Kremlin-Bicêtre , France., Spinner NB; Division of Genomic Diagnostics , Department of Pathology and Laboratory Medicine , The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine , Philadelphia , Pennsylvania , USA., Loomes KM; Division of Gastroenterology, Hepatology and Nutrition , The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine , Philadelphia , Pennsylvania , USA., Piccoli DA; Division of Gastroenterology, Hepatology and Nutrition , The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine , Philadelphia , Pennsylvania , USA., D'Antiga L; Pediatric Hepatology, Gastroenterology and Transplantation , Ospedale Papa Giovanni XXIII , Bergamo , Italy., Nicastro E; Pediatric Hepatology, Gastroenterology and Transplantation , Ospedale Papa Giovanni XXIII , Bergamo , Italy., Sokal É; Service De Gastroentérologie & Hépatologie Pédiatrique , Cliniques Universitaires Saint-Luc , Brussels , Belgium., Demaret T; Service De Gastroentérologie & Hépatologie Pédiatrique , Cliniques Universitaires Saint-Luc , Brussels , Belgium., Ebel NH; Division of Gastroenterology , Department of Pediatrics , Stanford University School of Medicine , Palo Alto , California , USA., Feinstein JA; Department of Pediatrics (Cardiology) , Stanford University School of Medicine , Lucile Packard Children's Hospital , Palo Alto , California , USA., Fawaz R; Department of Pediatrics , Yale University School of Medicine , New Haven , Connecticut , USA., Nastasio S; Division of Gastroenterology, Hepatology, & Nutrition , Boston Children's Hospital and Harvard Medical School , Boston , Massachusetts , 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., Arnell H; Department of Paediatric Gastroenterology, Hepatology and Nutrition , Astrid Lindgren Children's Hospital, Karolinska University Hospital , Stockholm , Sweden.; Department of Women's and Children's Health , Karolinska Institutet , Stockholm , Sweden., Fischler B; Department of Paediatric Gastroenterology, Hepatology and Nutrition , Astrid Lindgren Children's Hospital, Karolinska University Hospital , Stockholm , Sweden.; CLINTEC , Karolinska Institutet , Stockholm , Sweden., Siew S; Department of Gastroenterology , The Children's Hospital at Westmead , Sydney , Australia., Stormon M; Department of Gastroenterology , The Children's Hospital at Westmead , Sydney , Australia., Karpen SJ; Division of Pediatric Gastroenterology, Hepatology & Nutrition , Children's Healthcare of Atlanta & Emory University School of Medicine , Atlanta , Georgia , USA., Romero R; Division of Pediatric Gastroenterology, Hepatology & Nutrition , Children's Healthcare of Atlanta & Emory University School of Medicine , Atlanta , Georgia , USA., Kim KM; Department of Pediatrics , University of Ulsan College of Medicine , Asan Medical Center Children's Hospital , Seoul , Republic of Korea., Baek WY; Department of Pediatrics , University of Ulsan College of Medicine , Asan Medical Center Children's Hospital , Seoul , Republic of Korea., Hardikar W; Department of Gastroenterology and Clinical Nutrition , Royal Children's Hospital , Melbourne , Australia., Shankar S; Mazumdar Shaw Medical Center , Narayana Health , Bangalore , India., Roberts AJ; Department of Paediatric Gastroenterology , Starship Child Health , Auckland , New Zealand., Evans HM; Department of Paediatric Gastroenterology , Starship Child Health , Auckland , New Zealand., Jensen MK; Division of Pediatric Gastroenterology, Hepatology and Nutrition , University of Utah , Salt Lake City , Utah , USA., Kavan M; Division of Pediatric Gastroenterology, Hepatology and Nutrition , University of Utah , Salt Lake City , Utah , USA., 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 , Colorado , 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 , Colorado , USA., Karthikeyan P; Leeds Teaching Hospitals NHS Trust , Leeds Children's Hospital , Leeds , UK., Sanchez MC; Pediatric Gastroenterology and Hepatology Division , Hospital Italiano Buenos Aires , Buenos Aires , Argentina., Cavalieri ML; Pediatric Gastroenterology and Hepatology Division , Hospital Italiano Buenos Aires , Buenos Aires , Argentina., Verkade HJ; Department of Pediatrics , Center for Liver, Digestive, and Metabolic Diseases , University Medical Center Groningen , Groningen , The Netherlands., Lee WS; Faculty of Medicine, Department of Paediatrics , University of Malaya , Kuala Lumpur , Malaysia., Squires JE; Division of Pediatric Gastroenterology and Hepatology , Department of Pediatrics , University of Pittsburgh School of Medicine , Pittsburgh , Pennsylvania , USA., Hajinicolaou C; Division of Paediatric Gastroenterology , Department of Paediatrics and Child Health , Chris Hani Baragwanath Academic Hospital , University of the Witwatersrand , Johannesburg , South Africa., Lertudomphonwanit C; Division of Gastroenterology , Department of Pediatrics , Ramathibodi Hospital Mahidol University , Bangkok , Thailand., Fischer RT; Department of Gastroenterology, Section of Hepatology , Children's Mercy Kansas City , Kansas City , Missouri , USA., Larson-Nath C; Division of Pediatric Gastroenterology, Hepatology, and Nutrition , University of Minnesota , Minneapolis , Minnesota , USA., Mozer-Glassberg Y; Institute of Gastroenterology, Nutrition and Liver Diseases , Schneider Children's Medical Center of Israel , Petah Tikva , Israel., Arikan C; Department of Pediatric Gastroenterology and Organ Transplant , Koc University School of Medicine , Istanbul , Turkey., Lin HC; Division of Pediatric Gastroenterology , Department of Pediatrics , Oregon Health and Science University , Portland , Oregon , USA., Bernabeu JQ; Pediatric Hepatology and Liver Transplant Department , Hospital Universitari Vall d'Hebron , Barcelona , Spain., Alam S; Department of Pediatric Hepatology , Institute of Liver and Biliary Sciences , New Delhi , India., Kelly DA; Liver Unit , Birmingham Women's & Children's Hospital NHS Trust, University of Birmingham , Birmingham , UK., Carvalho E; Pediatric Gastroenterology Department , Hospital da Criança de Brasília , Centro Universitário de Brasília , Brasília , Brazil., Ferreira CT; Pediatric Gastroenterology Service , Hospital da Criança Santo Antôni, Universidade Federal de Ciências da Saúde de Porto Alegre , Complexo Hospitalar Santa Casa , Porto Alegre , RS , Brazil., Indolfi G; Paediatric and Liver Unit , Department Neurofarba , University of Florence and Meyer Children's University Hospital , Florence , Italy., Quiros-Tejeira RE; Department of Pediatrics , Children's Hospital & Medical Center and University of Nebraska Medical Center , Omaha , Nebraska , USA., Bulut P; Division of Pediatric Gastroenterology and Hepatology , Phoenix Children's Hospital , Phoenix , USA., Calvo PL; Pediatric Gastroenterology Unit , Regina Margherita Children's Hospital , Azienda Ospedaliera-Universitaria Citta' della Salute e della Scienza , Turin , Italy., Önal Z; Pediatric Gastroenterology, Hepatology and Nutrition Department, Istanbul Medical Faculty , Istanbul University , Istanbul , Turkey., Valentino PL; Department of Pediatrics , Yale University School of Medicine , New Haven , Connecticut , USA., Desai DM; Solid Organ Transplant Department , Children's Health - Children's Medical Center , Dallas , Texas , USA., Eshun J; Department of Pediatric Gastroenterology , Le Bonheur Children's Hospital, The University of Tennessee Health Science Center , Memphis , Texas , USA., Rogalidou M; Division of Gastroenterology & Hepatology, First Department of Pediatrics , 'Agia Sofia' Children's Hospital , University of Athens , Athens , Greece., Dezsőfi A; First Department of Paediatrics , Semmelweis University , Budapest , Hungary., Wiecek S; Department of Pediatrics , Medical University of Silesia in Katowice , Katowice , Poland., Nebbia G; Servizio di Epatologia Pediatrica , Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico , Milan , Italy., 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 , The Netherlands., Tamara ML; Paediatric Gastroenterology Unit , Cruces University Hospital , Bilbao , Spain., Zizzo AN; Division of Paediatric Gastroenterology and Hepatology , London Health Sciences Centre, Children's Hospital , Western University , London , Ontario , Canada., Garcia J; Division of Pediatric Gastroenterology, Hepatology and Nutrition, Miami Transplant Institute , University of Miami , Miami , Florida , USA., Schwarz K; Division of Pediatric Gastroenterology , University of California San Diego, Rady Children's Hospital San Diego , San Diego , California , USA., Beretta M; Faculty of Health Sciences, Wits Donald Gordon Medical Centre , University of the Witwatersrand , Johannesburg , South Africa., Sandahl TD; Department of Hepatology and Gastroenterology , Aarhus University Hospital , Aarhus , Denmark., Jimenez-Rivera C; Division of Gastroenterology, Hepatology and Nutrition , Children's Hospital of Eastern Ontario , Ottawa , Canada., Kerkar N; Division of Pediatric Gastroenterology, Hepatology and Nutrition , Department of Pediatrics , University of Rochester Medical Center , Rochester , New York , USA., Brecelj J; Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics , Faculty of Medicine , University Medical Center Ljubljana , Ljubljana , Slovenia., Mujawar Q; Section of Pediatric Gastroenterology , Department of Pediatrics , University of Manitoba , Winnipeg , Canada., Rock N; Department of Paediatrics, Gynaecology and Obstetrics , Geneva University Hospitals, University of Geneva , Geneva , Switzerland., Busoms CM; Pediatric Gastroenterology Hepatology and Nutrition Unit , Hospital Sant Joan de Déu , Esplugues de Llobregat , Spain., Karnsakul W; Department of Pediatrics , Johns Hopkins University School of Medicine , Baltimore , Maryland , USA., Lurz E; Department of Pediatrics , Dr. von Hauner Children's Hospital , University Hospital , LMU Munich , Munich , Germany., Santos-Silva E; Pediatric Gastroenterology Unit , Centro Hospitalar Universitário Do Porto , Porto , Portugal., Blondet N; Gastroenterology and Hepatology , Seattle Children's Hospital , Seattle , Washington , USA., Bujanda L; 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., Shah U; Harvard Medical School , Massachusetts General Hospital for Children , Boston , Massachusetts , USA., Thompson RJ; Institute of Liver Studies , King's College London , London , UK., Hansen BE; Toronto General Hospital University Health Network , Toronto , Ontario , Canada.; Institute of Health Policy, Management and Evaluation , Toronto , Ontario , Canada., Kamath BM; Division of Gastroenterology, Hepatology and Nutrition , The Hospital for Sick Children and the University of Toronto , Toronto , Canada.

Publikováno v: Hepatology (Baltimore, Md.) [Hepatology] 2023 Feb 01; Vol. 77 (2), pp. 512-529. Date of Electronic Publication: 2022 Oct 13.

Genotypic and phenotypic spectrum of infantile liver failure due to pathogenic TRMU variants.

Autor: Vogel GF, Mozer-Glassberg Y, Landau YE, Schlieben LD, Prokisch H, Feichtinger RG, Mayr JA, Brennenstuhl H, Schröter J, Pechlaner A, Alkuraya FS, Baker JJ, Barcia G, Baric I, Braverman N, Burnyte B, Christodoulou J, Ciara E, Coman D, Das AM, Darin N, Della Marina A, Distelmaier F, Eklund EA, Ersoy M, Fang W, Gaignard P, Ganetzky RD, Gonzales E, Howard C, Hughes J, Konstantopoulou V, Kose M, Kerr M, Khan A, Lenz D, McFarland R, Margolis MG, Morrison K, Müller T, Murayama K, Nicastro E, Pennisi A, Peters H, Piekutowska-Abramczuk D, Rötig A, Santer R, Scaglia F, Schiff M, Shagrani M, Sharrard M, Soler-Alfonso C, Staufner C, Storey I, Stormon M, Taylor RW, Thorburn DR, Teles EL, Wang JS, Weghuber D, Wortmannd S

Publikováno v: Genetics in medicine : official journal of the American College of Medical Genetics [Genet Med] 2023 Jun; Vol. 25 (6), pp. 100828. Date of Electronic Publication: 2023 Apr 13.

Split‐graft liver transplantation from an adult donor with an unrecognized UCD to a pediatric and adult recipient.

Autor: Lee, C. H.¹, Stormon, M. O.¹, Ellaway, C.², Shun, A.³, Thomas, G.³, Nair, P.⁴, O'Neill, J.⁵, Shakel, N.⁶

Publikováno v: Pediatric Transplantation. Feb2018, Vol. 22 Issue 1, p1-1. 4p.