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Using Multi-fluorinated Bile Acids and In Vivo Magnetic Resonance Imaging to Measure Bile Acid Transport
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Pediatric Cholestatic Diseases in the Era of Ileal Bile Acid Transporter (IBAT) Inhibitors

Marco Sciveres1, Silvio Veraldi1, Francesco Cirillo2

  • 1Hepatology and Liver Transplantation, IRCCS Bambino Gesù Paediatric Hospital, 00165 Rome, Italy.

Pediatric Reports
|February 20, 2026
PubMed

Insights

Bile acid transporter inhibitors (IBATis) offer new hope for treating cholestatic pruritus in children with rare liver diseases like PFIC and ALGS. These drugs effectively lower bile acid levels, reducing itch and showing potential for broader applications.

Area of Science:

  • Pediatric Gastroenterology and Hepatology
  • Pharmacology
  • Drug Development

Background:

  • Cholestatic liver diseases in children are often incurable, with limited treatment options for symptoms like pruritus.
  • Historically, off-label medications with unproven efficacy were used for managing cholestatic pruritus.
  • Bile acid transporter inhibitors (IBATis) represent a novel therapeutic class targeting bile acid reabsorption.

Purpose of the Study:

  • To review the efficacy and safety of IBATis in pediatric cholestatic pruritus.
  • To summarize real-world data on IBATis for progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS).
  • To explore the potential of IBATis in other cholestatic conditions and in adults.

Main Methods:

  • Narrative literature review.
  • Inclusion of recent communications and real-world data.
  • Analysis of efficacy and safety data for IBATis in pediatric cholestasis.

Main Results:

  • Two IBATis are approved for treating pruritus in PFIC and ALGS in children.
  • IBATis lower serum bile acid levels by inhibiting ileal reabsorption, thereby reducing cholestatic pruritus.
  • Emerging data suggest potential benefits in other cholestatic diseases and adult populations.

Conclusions:

  • IBATis demonstrate significant efficacy in managing pruritus associated with PFIC and ALGS in children.
  • These inhibitors offer a promising therapeutic avenue for cholestatic pruritus, with expanding applications.
  • Understanding the potential and limitations of IBATis is crucial for clinical decision-making in pediatric cholestasis.

Keywords:
Alagille syndromeIBAT inhibitorPFICcholestatic prurituspediatric cholestasis diseases

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