{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,2]],"date-time":"2026-10-02T22:58:58Z","timestamp":1790981938577,"version":"4.1.0"},"reference-count":21,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"2","license":[{"start":{"date-parts":[[2022,10,13]],"date-time":"2022-10-13T00:00:00Z","timestamp":1665619200000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/ http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["lww.com","ovid.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2023,2]]},"abstract":"<jats:sec>\n                    <jats:title>Background and Aims:<\/jats:title>\n                    <jats:p>Alagille syndrome (ALGS) is a multisystem disorder, characterized by cholestasis. Existing outcome data are largely derived from tertiary centers, and real\u2010world data are lacking. This study aimed to elucidate the natural history of liver disease in a contemporary, international cohort of children with ALGS.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Approach and Results:<\/jats:title>\n                    <jats:p>\n                      This was a multicenter retrospective study of children with a clinically and\/or genetically confirmed ALGS diagnosis, born between January 1997 and August 2019. Native liver survival (NLS) and event\u2010free survival rates were assessed. Cox models were constructed to identify early biochemical predictors of clinically evident portal hypertension (CEPH) and NLS. In total, 1433 children (57% male) from 67 centers in 29 countries were included. The 10 and 18\u2010year NLS rates were 54.4% and 40.3%. By 10 and 18\u2009years, 51.5% and 66.0% of children with ALGS experienced \u22651 adverse liver\u2010related event (CEPH, transplant, or death). Children (&gt;6 and \u226412 months) with median total bilirubin (TB) levels between \u22655.0 and &lt;10.0 mg\/dl had a 4.1\u2010fold (95% confidence interval [CI], 1.6\u201310.8), and those \u226510.0 mg\/dl had an 8.0\u2010fold (95% CI, 3.4\u201318.4) increased risk of developing CEPH compared with those &lt;5.0 mg\/dl. Median TB levels between \u22655.0 and &lt;10.0 mg\/dl and &gt;10.0 mg\/dl were associated with a 4.8 (95% CI, 2.4\u20139.7) and 15.6 (95% CI, 8.7\u201328.2) increased risk of transplantation relative to &lt;5.0 mg\/dl. Median TB &lt;5.0 mg\/dl were associated with higher NLS rates relative to \u22655.0 mg\/dl, with 79% reaching adulthood with native liver (\n                      <jats:italic toggle=\"yes\">p<\/jats:italic>\n                      &lt;\u20090.001).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>In this large international cohort of ALGS, only 40.3% of children reach adulthood with their native liver. A TB &lt;5.0 mg\/dl between 6 and 12 months of age is associated with better hepatic outcomes. These thresholds provide clinicians with an objective tool to assist with clinical decision\u2010making and in the evaluation of therapies.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/hep.32761","type":"journal-article","created":{"date-parts":[[2022,8,29]],"date-time":"2022-08-29T05:54:02Z","timestamp":1661752442000},"page":"512-529","update-policy":"https:\/\/doi.org\/10.1097\/lww.0000000000001000","source":"Crossref","is-referenced-by-count":98,"title":["Natural history of liver disease in a large international cohort of children with Alagille syndrome: Results from the GALA study"],"prefix":"10.1097","volume":"77","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7157-2063","authenticated-orcid":false,"given":"Shannon M.","family":"Vandriel","sequence":"first","affiliation":[{"name":"Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children and the University of Toronto, Toronto, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Li\u2010Ting","family":"Li","sequence":"additional","affiliation":[{"name":"The Center for Pediatric Liver Diseases, Children's Hospital of Fudan University, Shanghai, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7634-7217","authenticated-orcid":false,"given":"Huiyu","family":"She","sequence":"additional","affiliation":[{"name":"The Center for Pediatric Liver Diseases, Children's Hospital of Fudan University, Shanghai, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jian\u2010She","family":"Wang","sequence":"additional","affiliation":[{"name":"The Center for Pediatric Liver Diseases, Children's Hospital of Fudan University, Shanghai, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa A.","family":"Gilbert","sequence":"additional","affiliation":[{"name":"Division of Genomic Diagnostics, Department of Pathology and Laboratory Medicine, The Children\u2019s Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irena","family":"Jankowska","sequence":"additional","affiliation":[{"name":"Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics, The Children's Memorial Health Institute, Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Piotr","family":"Czubkowski","sequence":"additional","affiliation":[{"name":"Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics, The Children's Memorial Health Institute, Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dorota","family":"Gliwicz\u2010Miedzi\u0144ska","sequence":"additional","affiliation":[{"name":"Department of Gastroenterology, Hepatology, Nutrition Disturbances and Pediatrics, The Children's Memorial Health Institute, Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1546-3193","authenticated-orcid":false,"given":"Emmanuel M.","family":"Gonzales","sequence":"additional","affiliation":[{"name":"Pediatric Hepatology and Liver Transplantation Unit, National Reference Centre for Rare Pediatric Liver Diseases (Biliary Atresia and Genetic Cholestasis), FILFOIE, ERN RARE LIVER, Bic\u00eatre Hospital, AP\u2010HP and Inserm U1193, Hepatinov, Universit\u00e9 Paris\u2010Saclay, Le Kremlin\u2010Bic\u00eatre, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emmanuel","family":"Jacquemin","sequence":"additional","affiliation":[{"name":"Pediatric Hepatology and Liver Transplantation Unit, National Reference Centre for Rare Pediatric Liver Diseases (Biliary Atresia and Genetic Cholestasis), FILFOIE, ERN RARE LIVER, Bic\u00eatre Hospital, AP\u2010HP and Inserm U1193, Hepatinov, Universit\u00e9 Paris\u2010Saclay, Le Kremlin\u2010Bic\u00eatre, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J\u00e9r\u00f4me","family":"Bouligand","sequence":"additional","affiliation":[{"name":"Service de G\u00e9n\u00e9tique Mol\u00e9culaire, Pharmacog\u00e9n\u00e9tique et Hormonologie, H\u00f4pitaux Universitaires Paris\u2010Saclay, Assistance Publique\u2010H\u00f4pitaux de Paris, Centre Hospitalier Universitaire de Bic\u00eatre, Le Kremlin\u2010Bic\u00eatre, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nancy B.","family":"Spinner","sequence":"additional","affiliation":[{"name":"Division of Genomic Diagnostics, Department of Pathology and Laboratory Medicine, The Children\u2019s Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen M.","family":"Loomes","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology, Hepatology and Nutrition, The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David A.","family":"Piccoli","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology, Hepatology and Nutrition, The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7150-3148","authenticated-orcid":false,"given":"Lorenzo","family":"D'Antiga","sequence":"additional","affiliation":[{"name":"Pediatric Hepatology, Gastroenterology and Transplantation, Ospedale Papa Giovanni XXIII, Bergamo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emanuele","family":"Nicastro","sequence":"additional","affiliation":[{"name":"Pediatric Hepatology, Gastroenterology and Transplantation, Ospedale Papa Giovanni XXIII, Bergamo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5597-4708","authenticated-orcid":false,"given":"\u00c9tienne","family":"Sokal","sequence":"additional","affiliation":[{"name":"Service De Gastroent\u00e9rologie & H\u00e9patologie P\u00e9diatrique, Cliniques Universitaires Saint\u2010Luc, Brussels, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tanguy","family":"Demaret","sequence":"additional","affiliation":[{"name":"Service De Gastroent\u00e9rologie & H\u00e9patologie P\u00e9diatrique, Cliniques Universitaires Saint\u2010Luc, Brussels, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Noelle H.","family":"Ebel","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology, Department of Pediatrics, Stanford University School of Medicine, Palo Alto, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey A.","family":"Feinstein","sequence":"additional","affiliation":[{"name":"Department of Pediatrics (Cardiology), Stanford University School of Medicine, Lucile Packard Children's Hospital, Palo Alto, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rima","family":"Fawaz","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Yale University School of Medicine, New Haven, Connecticut, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0200-5326","authenticated-orcid":false,"given":"Silvia","family":"Nastasio","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology, Hepatology, & Nutrition, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Florence","family":"Lacaille","sequence":"additional","affiliation":[{"name":"Department of Pediatric Gastroenterology, and Nutrition, Necker\u2010Enfants Malades Hospital, University of Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dominique","family":"Debray","sequence":"additional","affiliation":[{"name":"Pediatric Liver Unit, National Reference Centre for Rare Pediatric Liver Diseases (Biliary Atresia and Genetic Cholestasis), FILFOIE, ERN RARE LIVER, Necker\u2010Enfants Malades Hospital, University of Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Henrik","family":"Arnell","sequence":"additional","affiliation":[{"name":"Department of Paediatric Gastroenterology, Hepatology and Nutrition, Astrid Lindgren Children's Hospital, Karolinska University Hospital, Stockholm, Sweden"},{"name":"Department of Women's and Children's Health, Karolinska Institutet, Stockholm, Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bj\u00f6rn","family":"Fischler","sequence":"additional","affiliation":[{"name":"Department of Paediatric Gastroenterology, Hepatology and Nutrition, Astrid Lindgren Children's Hospital, Karolinska University Hospital, Stockholm, Sweden"},{"name":"CLINTEC, Karolinska Institutet, Stockholm, Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan","family":"Siew","sequence":"additional","affiliation":[{"name":"Department of Gastroenterology, The Children's Hospital at Westmead, Sydney, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Stormon","sequence":"additional","affiliation":[{"name":"Department of Gastroenterology, The Children's Hospital at Westmead, Sydney, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saul J.","family":"Karpen","sequence":"additional","affiliation":[{"name":"Division of Pediatric Gastroenterology, Hepatology & Nutrition, Children's Healthcare of Atlanta & Emory University School of Medicine, Atlanta, Georgia, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rene","family":"Romero","sequence":"additional","affiliation":[{"name":"Division of Pediatric Gastroenterology, Hepatology & Nutrition, Children's Healthcare of Atlanta & Emory University School of Medicine, Atlanta, Georgia, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kyung Mo","family":"Kim","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Woo Yim","family":"Baek","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Winita","family":"Hardikar","sequence":"additional","affiliation":[{"name":"Department of Gastroenterology and Clinical Nutrition, Royal Children's Hospital, Melbourne, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sahana","family":"Shankar","sequence":"additional","affiliation":[{"name":"Mazumdar Shaw Medical Center, Narayana Health, Bangalore, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amin J.","family":"Roberts","sequence":"additional","affiliation":[{"name":"Department of Paediatric Gastroenterology, Starship Child Health, Auckland, New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Helen M.","family":"Evans","sequence":"additional","affiliation":[{"name":"Department of Paediatric Gastroenterology, Starship Child Health, Auckland, New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. Kyle","family":"Jensen","sequence":"additional","affiliation":[{"name":"Division of Pediatric Gastroenterology, Hepatology and Nutrition, University of Utah, Salt Lake City, Utah, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marianne","family":"Kavan","sequence":"additional","affiliation":[{"name":"Division of Pediatric Gastroenterology, Hepatology and Nutrition, University of Utah, Salt Lake City, Utah, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shikha S.","family":"Sundaram","sequence":"additional","affiliation":[{"name":"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"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander","family":"Chaidez","sequence":"additional","affiliation":[{"name":"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"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Palaniswamy","family":"Karthikeyan","sequence":"additional","affiliation":[{"name":"Leeds Teaching Hospitals NHS Trust, Leeds Children's Hospital, Leeds, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Camila","family":"Sanchez","sequence":"additional","affiliation":[{"name":"Pediatric Gastroenterology and Hepatology Division, Hospital Italiano Buenos Aires, Buenos Aires, Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Lorena","family":"Cavalieri","sequence":"additional","affiliation":[{"name":"Pediatric Gastroenterology and Hepatology Division, Hospital Italiano Buenos Aires, Buenos Aires, Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Henkjan J.","family":"Verkade","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Center for Liver, Digestive, and Metabolic Diseases, University Medical Center Groningen, Groningen, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Way Seah","family":"Lee","sequence":"additional","affiliation":[{"name":"Faculty of Medicine, Department of Paediatrics, University of Malaya, Kuala Lumpur, Malaysia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6979-8987","authenticated-orcid":false,"given":"James E.","family":"Squires","sequence":"additional","affiliation":[{"name":"Division of Pediatric Gastroenterology and Hepatology, Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christina","family":"Hajinicolaou","sequence":"additional","affiliation":[{"name":"Division of Paediatric Gastroenterology, Department of Paediatrics and Child Health, Chris Hani Baragwanath Academic Hospital, University of the Witwatersrand, Johannesburg, South Africa"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chatmanee","family":"Lertudomphonwanit","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology, Department of Pediatrics, Ramathibodi Hospital Mahidol University, Bangkok, Thailand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2576-4694","authenticated-orcid":false,"given":"Ryan T.","family":"Fischer","sequence":"additional","affiliation":[{"name":"Department of Gastroenterology, Section of Hepatology, Children's Mercy Kansas City, Kansas City, Missouri, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Larson\u2010Nath","sequence":"additional","affiliation":[{"name":"Division of Pediatric Gastroenterology, Hepatology, and Nutrition, University of Minnesota, Minneapolis, Minnesota, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yael","family":"Mozer\u2010Glassberg","sequence":"additional","affiliation":[{"name":"Institute of Gastroenterology, Nutrition and Liver Diseases, Schneider Children's Medical Center of Israel, Petah Tikva, Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cigdem","family":"Arikan","sequence":"additional","affiliation":[{"name":"Department of Pediatric Gastroenterology and Organ Transplant, Koc University School of Medicine, Istanbul, Turkey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Henry C.","family":"Lin","sequence":"additional","affiliation":[{"name":"Division of Pediatric Gastroenterology, Department of Pediatrics, Oregon Health and Science University, Portland, Oregon, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesus Quintero","family":"Bernabeu","sequence":"additional","affiliation":[{"name":"Pediatric 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Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina Targa","family":"Ferreira","sequence":"additional","affiliation":[{"name":"Pediatric Gastroenterology Service, Hospital da Crian\u00e7a Santo Ant\u00f4ni, Universidade Federal de Ci\u00eancias da Sa\u00fade de Porto Alegre, Complexo Hospitalar Santa Casa, Porto Alegre, RS, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giuseppe","family":"Indolfi","sequence":"additional","affiliation":[{"name":"Paediatric and Liver Unit, Department Neurofarba, University of Florence and Meyer Children's University Hospital, Florence, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruben E.","family":"Quiros\u2010Tejeira","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Children's Hospital & Medical Center and University of Nebraska Medical Center, Omaha, Nebraska, 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Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Victorien M.","family":"Wolters","sequence":"additional","affiliation":[{"name":"Department of Pediatric Gastroenterology, University Medical Center Utrecht, Utrecht, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mar\u00eda Legarda","family":"Tamara","sequence":"additional","affiliation":[{"name":"Paediatric Gastroenterology Unit, Cruces University Hospital, Bilbao, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6350-2057","authenticated-orcid":false,"given":"Andr\u00e9anne N.","family":"Zizzo","sequence":"additional","affiliation":[{"name":"Division of Paediatric Gastroenterology and Hepatology, London Health Sciences Centre, Children's Hospital, Western University, London, Ontario, 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and Gastroenterology, Aarhus University Hospital, Aarhus, Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carolina","family":"Jimenez\u2010Rivera","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Eastern Ontario, Ottawa, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nanda","family":"Kerkar","sequence":"additional","affiliation":[{"name":"Division of Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, University of Rochester Medical Center, Rochester, New York, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jernej","family":"Brecelj","sequence":"additional","affiliation":[{"name":"Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Faculty of Medicine, University Medical Center Ljubljana, Ljubljana, 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