{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T17:52:27Z","timestamp":1783014747368,"version":"3.54.6"},"reference-count":12,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2025,1,17]],"date-time":"2025-01-17T00:00:00Z","timestamp":1737072000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Liver International"],"published-print":{"date-parts":[[2025,2]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Alagille syndrome (ALGS) is a multisystem cholestatic disorder. Maralixibat is approved for the treatment of cholestatic pruritus in ALGS with limited data in adults.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Participants were included if they received \u2265\u20092 doses of maralixibat at age \u2265\u200916\u2009years in one of the three previously published maralixibat ALGS clinical trials.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Eleven initiated treatment &lt;\u200916\u2009years old (median age, 13.0) with a median follow\u2010up of 4.1\u2009years. Three participants began maralixibat at \u2265\u200916\u2009years old, with a median follow\u2010up of 3.8\u2009years. Participants starting maralixibat at age &lt;\u200916 had minimal\u2010to\u2010no itch (change from baseline [CFB]: \u22121.8; <jats:italic>p<\/jats:italic>\u2009=\u20090.002) persisting throughout the study. Serum bile acids (sBA) decreased (CFB: 29\u2009\u03bcmol\/L; <jats:italic>p<\/jats:italic>\u2009=\u20090.03), persisting throughout the study. Participants starting maralixibat \u2265\u200916\u2009years old had pruritus improvements (CFB: \u22122.8, \u22120.6, \u22121.0). One had a large decrease in sBA (CFB: \u2212112\u2009\u03bcmol\/L), and two had small increases (CFB: 8, 11\u2009\u03bcmol\/L). Maralixibat was well tolerated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Participants receiving maralixibat in adolescence demonstrated improvements in pruritus and sBA, which persisted through young adulthood.<\/jats:p><\/jats:sec>","DOI":"10.1111\/liv.16201","type":"journal-article","created":{"date-parts":[[2025,1,17]],"date-time":"2025-01-17T09:56:31Z","timestamp":1737107791000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Maralixibat Reduces Serum Bile Acids and Improves Cholestatic Pruritus in Adolescents With Alagille Syndrome"],"prefix":"10.1111","volume":"45","author":[{"given":"Gideon","family":"Hirschfield","sequence":"first","affiliation":[{"name":"The Autoimmune and Rare Liver Disease Programme, Division of Gastroenterology and Hepatology Toronto General Hospital  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7157-2063","authenticated-orcid":false,"given":"Shannon M.","family":"Vandriel","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology, Hepatology and Nutrition The Hospital for Sick Children and the University of Toronto  Toronto Ontario Canada"},{"name":"Mirum Pharmaceuticals Inc.  Foster City California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Douglas B.","family":"Mogul","sequence":"additional","affiliation":[{"name":"Mirum Pharmaceuticals Inc.  Foster City California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marshall","family":"Baek","sequence":"additional","affiliation":[{"name":"Mirum Pharmaceuticals Inc.  Foster City California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pamela","family":"Vig","sequence":"additional","affiliation":[{"name":"Mirum Pharmaceuticals Inc.  Foster City California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-2023-304X","authenticated-orcid":false,"given":"Binita M.","family":"Kamath","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology, Hepatology and Nutrition The Hospital for Sick Children and the University of Toronto  Toronto Ontario Canada"},{"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"}]}],"member":"311","published-online":{"date-parts":[[2025,1,17]]},"reference":[{"key":"e_1_2_14_2_1","doi-asserted-by":"publisher","DOI":"10.1097\/mpg.0000000000001958"},{"issue":"1","key":"e_1_2_14_3_1","first-page":"S20","article-title":"Surgical Biliary Diversion Is Associated With an Increased Risk of Liver Transplantation or Death in Alagille Syndrome","volume":"78","author":"Vandriel S. M.","year":"2023","journal-title":"Hepatology"},{"key":"e_1_2_14_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/hep.32761"},{"key":"e_1_2_14_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/hep4.1468"},{"key":"e_1_2_14_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/hep4.1244"},{"key":"e_1_2_14_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/s0140\u20106736(21)01256\u20103"},{"key":"e_1_2_14_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/hep4.1992"},{"key":"e_1_2_14_9_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40271\u2010017\u20100266\u20104"},{"key":"e_1_2_14_10_1","doi-asserted-by":"publisher","DOI":"10.1097\/hc9.0000000000000153"},{"key":"e_1_2_14_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.62028"},{"key":"e_1_2_14_12_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12072\u2010023\u201010578\u2010x"},{"key":"e_1_2_14_13_1","doi-asserted-by":"publisher","DOI":"10.1097\/HEP.0000000000000727"}],"container-title":["Liver International"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/liv.16201","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,4,9]],"date-time":"2025-04-09T08:03:39Z","timestamp":1744185819000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/liv.16201"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,1,17]]},"references-count":12,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2025,2]]}},"alternative-id":["10.1111\/liv.16201"],"URL":"https:\/\/doi.org\/10.1111\/liv.16201","archive":["Portico"],"relation":{},"ISSN":["1478-3223","1478-3231"],"issn-type":[{"value":"1478-3223","type":"print"},{"value":"1478-3231","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,1,17]]},"assertion":[{"value":"2024-07-25","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-11-25","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-01-17","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e16201"}}