Ileal bile acid transporter inhibitors in Alagille syndrome and Progressive Familial Intrahepatic Cholestasis: A systematic review into dose-response.

ABSTRACT: Ileal bile acid transporter inhibitors (IBATi), including maralixibat and odevixibat, are a novel approach to the treatment of paediatric cholestatic liver diseases, such as Alagille syndrome (ALGS) and different forms of progressive familial intrahepatic cholestasis (PFIC). Multiple phase 3 trials have demonstrated efficacy of IBATi in alleviating pruritus and decreasing serum bile acid (sBA) in a significant subset of patients. While most clinical IBATi trials included low-dose and high-dose treatment, a consistent dose-response effect has not been uniformly reported. This systematic review aims to assess the dose-response relationship for IBATi in patients with ALGS or PFIC, based on available pruritus, sBA and (non-)responder outcomes, identifying minimally effective dosage and explori

SUBMITTER: de Groot ADE 

PROVIDER: S-EPMC12746362 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature