# Pediatric Cholestatic Diseases in the Era of Ileal Bile Acid Transporter (IBAT) Inhibitors

## TL;DR

This paper reviews new drugs called IBAT inhibitors that help reduce itching in children with rare liver diseases by blocking bile acid reabsorption.

## Contribution

The paper provides a comprehensive review of the real-world efficacy and safety of IBAT inhibitors in treating cholestatic pruritus in children.

## Key findings

- IBAT inhibitors effectively reduce pruritus in PFIC and ALGS by lowering serum bile acid levels.
- Real-world data supports the safety and potential broader benefits of IBAT inhibitors in cholestatic diseases.
- Off-label use and adult experiences suggest IBAT inhibitors may be beneficial beyond their approved indications.

## Abstract

Cholestatic diseases in children represent 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 (BAs) lowers serum levels. This contributes to reducing cholestatic pruritus. Such a mechanism of action may also have a potential benefit in other cholestatic diseases and even in the consequences of chronic cholestasis. This is a narrative review of the literature, including the most recent communications, to summarize data on the efficacy and safety of IBATis in the treatment of pruritus in PFIC and ALGS in children, including a description of the latest results from their use in a real-world setting. Reports on off-label use and experiences in adults are also discussed. This review aims to help physicians understand the potential and limitations of these new drugs in the treatment of cholestatic pruritus.