Improvement in sequelae of chronic liver disease in patients with Alagille syndrome treated with maralixibat

Maralixibat (MRX) is an orally administered, small‐molecule ileal bile acid transporter (IBAT) inhibitor used in the treatment of cholestatic pruritus in conditions such as Alagille syndrome (ALGS). MRX's beneficial effects on cholestatic pruritus are well described, however its effects on patients’ clinical manifestations of hepatic disease are less well known. We report two cases of patients with ALGS treated with MRX who demonstrated stabilization of their esophageal varices and xanthomas, respectively. Our findings demonstrate the potential role of MRX in improving the extrahepatic manifestations of liver disease in patients with ALGS. Further studies are needed to understand the physiologic mechanisms driving these changes.