Real-world use of maralixibat in biliary atresia: a case series

Biliary atresia (BA) is the most common cause of cholestasis in infants, and pruritus may be a prominent and debilitating complication. Maralixibat is the first US Food and Drug Administration-approved drug for the treatment of cholestatic pruritus in children with Alagille syndrome and has been subsequently approved for treatment of progressive familial intrahepatic cholestasis as well. Several patients with BA have received maralixibat as part of a compassionate use program. We report the use of maralixibat for the treatment of cholestatic pruritus in BA for six patients. Demographics, past medical history, laboratory markers, and medications were collected. Pruritus was assessed using the Clinician Scratch Scale (CSS) at baseline and last clinical follow-up. All patients reported improved CSS with no increased usage of other antipruritic medications, and the medication was well tolerated.