Odevixibat improves pruritus and bile acid level in Alagille syndrome: A case report

Core Tip

Core Tip: Alagille syndrome is a rare genetic disorder that affects the liver causing, cholestasis, jaundice and intractable pruritus which can significantly impair health-related quality of life. The treatment is mainly supportive with majority eventually requiring liver transplant. Ileal bile acid transporter inhibition is a novel therapeutic concept for cholestatic pruritus and cholestatic liver disease. We report the effects of odevixibat treatment in a patient Alagille syndrome over 12 months. This case demonstrates the use of ileal bile acid transporter inhibitors treating pruritus and improving patient quality of life and biochemical parameters. This allowed for the delay in the need for liver transplantation. It has potential to delay the need for liver transplantation. However more long-term studies are needed to help clinicians better understand the benefit of this new treatment.