Malattie colestatiche del bambino e i nuovi farmaci IBAT inibitori

Le résumé fourni par la source

Cholestatic diseases in children are a heterogeneous group of disorders that, with few exceptions, have no cure. For decades, off-label drugs and/or drugs with little evidence of efficacy have been used to treat pruritus or as supportive therapy. In recent years, a family of molecules known as bile acid transporter inhibitors (IBATis) has been developed, with two of these being approved for treating pruritus in progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS). Blocking the ileal reabsorption of bile acids (BA) lowers serum levels. This contributes to reducing cholestatic pruritus. This mechanism of action opens scenarios that speculatively may also include a potential benefit in other cholestatic diseases and even on the consequences of chronic cholestasis. This is a narrative review of the literature that includes the most recent communications and summarises data on efficacy and safety of IBATis in the treatment of pruritus in PFIC and ALGS in children. It also includes a description of the latest results from their use in a real-world setting. Reports on off-label use in adults and their feedbacks are also discussed. This review aims to help physicians understand the potential and limitations of these new drugs in the treatment of cholestatic pruritus.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.