Maralixibat is an alternative to liver transplantation in children with Alagille syndrome

Abstract

Alagille syndrome is a genetic multisystem disease in which one of the key symptoms that significantly impairs quality of life is cholestatic pruritus. Until recently the only treatment opportunity was liver transplantation. In 2021 the new drug maralixibat (Marixibat) was approved and registered in the United States. This article presents the first experience of using this medication in Russia. The patient stopped itching, significantly reduced the serum bile acids and also improved physical development within 7 months of therapy. Our first experience of using maralixibat in Russia, as well as the available literature data indicates a significant improvement in the quality of life of patients and allows us to consider this drug as an alternative to liver transplantation.