Natural history of liver disease in a large international cohort of children with Alagille syndrome: Results from the GALA study.
Shannon Marie VandrielLi-Ting LiHuiyu SheJian-She WangMelissa A GilbertIrena JankowskaPiotr CzubkowskiDorota Gliwicz-MiedzińskaEmmanuel M GonzalesEmmanuel JacqueminJérôme BouligandNancy B SpinnerKathleen M LoomesDavid A PiccoliLorenzo D'AntigaEmanuele NicastroEtienne M SokalTanguy DemaretNoelle H EbelJeffrey A FeinsteinRima FawazSilvia NastasioFlorence LacailleDominique DebrayHenrik ArnellBjörn FischlerSusan SiewMichael StormonSaul J KarpenRene RomeroKyung Mo KimWoo Yim BaekWinita HardikarSahana ShankarAmin J RobertsHelen M EvansM Kyle JensenMarianne KavanShikha S SundaramAlexander ChaidezPalaniswamy KarthikeyanMaria Camila SanchezMaria Lorena CavalieriHenkjan J VerkadeWay Seah LeeJames E SquiresChristina HajinicolaouChatmanee LertudomphonwanitRyan T FischerCatherine Larson-NathYael Mozer-GlassbergCigdem ArikanHenry C LinJesus Quintero BernabeuSeema AlamDeirdre A KellyElisa CarvalhoCristina Targa FerreiraGiuseppe IndolfiRuben E Quiros-TejeiraPinar BulutPier Luigi CalvoZerrin ÖnalPamela L ValentinoDev M DesaiJohn EshunMaria RogalidouAntal DezsőfiSabina WiecekGabriella NebbiaRaquel Borges PintoVictorien M WoltersMaría Legarda TamaraAndréanne N ZizzoJennifer GarciaKathleen SchwarzMarisa BerettaThomas Damgaard SandahlCarolina Jimenez-RiveraNanda KerkarJernej BreceljQuais MujawarNathalie RockCristina Molera BusomsWikrom KarnsakulEberhard LurzErmelinda Santos-SilvaNiviann BlondetLuis BujandaUzma ShahRichard J ThompsonBettina E HansenBinita M Kamathnull nullPublished in: Hepatology (Baltimore, Md.) (2022)
In this large international cohort of ALGS, only 40.3% of children reach adulthood with their native liver. A TB <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-making and in the evaluation of therapies.