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

Odevixibat, a reversible ileal bile acid transport (iBAT) 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.We describe three children with progressive cholestatic liver disease who developed refractory pruritus, who had a genotypic diagnosis of AKR1D1, ABCB4 variant, and PKHD1 and PKHD2 variants; all being variants of unknown significance as per the American College of Medical Genetics and Genomics guidelines.On Odevixibat there was a significant improvement in sBA (absolute change from baseline: −196 and −393 μmol/L) and pruritus in two children with heterozygous AKR1D1 and ABCB4 mutations. The child with ABCB4 variants was found to have features of sclerosing cholangitis along with a diagnosis of Crohn's disease, which represents the first reported usage of Odevixibat in such a case with good response. There was some reported improvement in the third child with PKHD1 and PKHD2 variants; however, we hypothesize that no sustained improvement could be due to severe and progressive nature of the disease. There were no side effects reported and it was well tolerated in all.We suggest that Odevixibat may be used as an adjunctive drug in refractory pruritus and could be started early in the course of disease if clinically and phenotypically indicated.