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Updated: Jul 10, 2025
Isolation of Neonatal Extrahepatic Cholangiocytes
Published on: June 5, 2014
Paediatric research sets new standards for therapy in paediatric and adult cholestasis
Rebecca Jeyaraj1, Eamonn R Maher2, Deirdre Kelly3
1University College London Great Ormond Street Institute of Child Health, London, UK.
Insights
New ileal bile acid transporter (IBAT) inhibitors offer hope for children with Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC), significantly improving quality of life and potentially altering disease progression.
Area of Science:
- Hepatology and Gastroenterology
- Pediatric Liver Disease
- Drug Development
Background:
- Children with Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC) suffer from severe pruritus with limited treatment options.
- Genetic discoveries have illuminated the molecular underpinnings of cholestatic liver diseases.
Purpose of the Study:
- To review the development and impact of ileal bile acid transporter (IBAT) inhibitors for pediatric cholestatic pruritus.
- To discuss the potential of these novel therapies in Alagille syndrome and PFIC.
Main Methods:
- Review of recent clinical data and genetic research in pediatric cholestatic disorders.
- Focus on the application of maralixibat and odevixibat in Alagille syndrome and PFIC.
Main Results:
- Ileal bile acid transporter (IBAT) inhibitors, maralixibat and odevixibat, are now approved for cholestatic pruritus in children.
- These drugs improve quality of life and show potential for improved event-free survival.
Conclusions:
- Newly approved IBAT inhibitors represent a significant advancement in managing pediatric cholestatic pruritus.
- These therapies may benefit a broader range of pediatric and adult patients with gastrointestinal and liver conditions.
Abstract:
Children with Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC) experience debilitating pruritus, for which there have been few effective treatment options. In the past 2 years, the ileal bile acid transporter (IBAT) inhibitors maralixibat and odevixibat have been approved for the management of cholestatic pruritus in these individuals, representing an important step forward in improving their quality of life. Emerging data suggest these drugs might also improve event-free survival, therefore potentially altering the typical disease course currently seen in these disorders. This Review will discuss how genetic advances have clarified the molecular basis of cholestatic disorders, facilitating the development of new therapeutic options that have only been evaluated in children. We focus specifically on the newly licensed IBAT inhibitors for patients with Alagille syndrome and PFIC and explore the next steps for these drugs in relation to other paediatric and adult cholestatic disorders, recognising that they have the potential to benefit a wider group of patients with gastrointestinal and liver disease.
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