Odevixibat as an adjunctive treatment for refractory pruritus in rare variants of cholestatic liver disease.

Objectives: Odevixibat, a reversible ileal bile acid transport inhibitor, has been shown to reduce serum bile acids (sBA) and pruritus mostly in children with progressive familial intrahepatic cholestasis (PFIC) 1 and 2 in clinical trials and case reports. There are currently no published case reports or series describing its use in rare variants of cholestatic liver disease.

Methods: We describe three children with progressive cholestatic liver disease who developed refractory pruritus, who had a genotypic diagnosis of , variant, and and variants; all being variants of unknown significance as per the American College of Medical Genetics and Genomics guidelines.