Predictors of 6-year event-free survival in alagille syndrome patients treated with maralixibat, an ileal bile acid transporter inhibitor.

Ronald J Sokol,Emmanuel M Gonzales,Binita M Kamath,Alastair Baker,Pamela Vig,Douglas B Mogul,Will Garner,Bettina E Hansen,Emmanuel Jacquemin,Richard J Thompson

Hepatology (Baltimore, Md.)（2023）

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摘要

Improvement in pruritus over 48 weeks and lower W48 bilirubin and sBA levels were associated with fewer events. These data may help identify potential markers of disease progression for MRX-treated ALGS patients.

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