Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA.
Bettina E HansenShannon M VandrielPamela VigWill GarnerDouglas B MogulKathleen M LoomesDavid A PiccoliElizabeth B RandIrena JankowskaPiotr CzubkowskiDorota Gliwicz-MiedzińskaEmmanuel M GonzalesEmmanuel JacqueminJérôme BouligandLorenzo D'AntigaEmanuele NicastroHenrik ArnellBjörn FischlerÉtienne SokalTanguy DemaretSusan SiewMichael StormonSaul J KarpenRene RomeroNoelle H EbelJeffrey A FeinsteinAmin J RobertsHelen M EvansShikha S SundaramAlexander ChaidezWinita HardikarSahana ShankarRyan T FischerFlorence LacailleDominique DebrayHenry C LinM Kyle JensenCatalina JaramilloPalaniswamy KarthikeyanGiuseppe IndolfiHenkjan J VerkadeCatherine Larson-NathRuben E Quiros-TejeiraPamela L ValentinoMaria RogalidouAntal DezsőfiJames E SquiresKathleen SchwarzPier Luigi CalvoJesus Quintero BernabeuAndréanne N ZizzoGabriella NebbiaPinar BulutErmelinda Santos-SilvaRima FawazSilvia NastasioWikrom KarnsakulMaría Legarda TamaraCristina Molera BusomsDeirdre A KellyThomas Damgaard SandahlCarolina Jimenez-RiveraJesus M BanalesQuais MujawarLi-Ting LiHuiyu SheJian-She WangKyung Mo KimSeak Hee OhMaria Camila SanchezMaria Lorena CavalieriWay Seah LeeChristina HajinicolaouChatmanee LertudomphonwanitOrith Waisbourd-ZinmanCigdem ArikanSeema AlamElisa CarvalhoMelina MelereJohn EshunZerrin ÖnalDev M DesaiSabina WiecekRaquel Borges PintoVictorien M WoltersJennifer GarciaMarisa BerettaNanda KerkarJernej BreceljNathalie RockEberhard LurzNiviann BlondetUzma ShahRichard J ThompsonBinita M. Kamathnull nullPublished in: Hepatology (Baltimore, Md.) (2023)
This study demonstrates a novel application of a robust statistical method to evaluate outcomes in long-term intervention studies where placebo comparisons are not feasible, providing wide application for rare diseases. This comparison with real-world natural history data suggests that maralixibat improves event-free survival in patients with ALGS.