ReviewLiver international : official journal of the International Association for the Study of the Liver2020
Potential of ileal bile acid transporter inhibition as a therapeutic target in Alagille syndrome and progressive familial intrahepatic cholestasis.
Review in Liver international : official journal of the International Association for the Study of the Liver, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers, 2 of them syntheses that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
34 citing papers in PubMed, 2 syntheses or guidelines pooled it, 76 citations in OpenAlex.
- Efficacy of ileal bile acid transport inhibitors in children with Alagille syndrome: a meta-analysis.European journal of pediatrics · 2025Pooled it
- The burden of Alagille syndrome: uncovering the potential of emerging therapeutics - a comprehensive systematic literature review.Journal of comparative effectiveness research · 2025Pooled it
- Pretreatment serum bile acid composition and predictability of subsequent response to odevixibat in patients with bile salt export pump (BSEP) deficiency.Hepatology (Baltimore, Md.) · 2026Trial
- Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.The lancet. Gastroenterology & hepatology · 2024Trial
- Reversal of surgical biliary diversion with ileal bile acid transport inhibitors: A new chapter in progressive familiar intrahepatic cholestasis type 1 management?JPGN reports · 2026Article
- New percentiles of gamma-glutamyl transferase serum levels in infants and the role of breastfeeding.BMC pediatrics · 2026Article
- Alagille syndrome caused by p.L2014Vfs*10 in NOTCH2: a case report and review of the literature.Journal of medical case reports · 2026Review
- Ileal bile acid transporter inhibitors for chronic constipation in the elderly.World journal of gastrointestinal pharmacology and therapeutics · 2026Article
- Maralixibat for the treatment of severe xanthomas in two children with Alagille syndrome: Case reports.JPGN reports · 2026Article
- ATP-binding Cassette Transporter Defects and Their Roles in Hepatic Diseases.Journal of clinical and translational hepatology · 2026Review
- Real-world use of maralixibat in biliary atresia: a case series.Frontiers in pediatrics · 2026Article
- The history and future of bile acid therapies.Journal of hepatology · 2025Article
- Treatment of Pruritus With Maralixibat in Early-Stage Chronic Graft Dysfunction of a Child With Alagille Syndrome.Cureus · 2025Article
- Primary sclerosing cholangitis.Nature reviews. Disease primers · 2025Review
- Pruritus in Chronic Cholestatic Liver Diseases, Especially in Primary Biliary Cholangitis: A Narrative Review.International journal of molecular sciences · 2025Review
- Genotypes and different clinical variants between children and adults in progressive familial intrahepatic cholestasis: a state-of-the-art review.Orphanet journal of rare diseases · 2025Review
- Practical Considerations for Odevixibat Treatment in Patients with Progressive Familial Intrahepatic Cholestasis: A Single-Center Case Series.Journal of clinical medicine · 2024Article
- Clinical, pathological and genetic characteristics of 17 unrelated children with Alagille Syndrome.BMC pediatrics · 2024Article
- Transporter Proteins as Therapeutic Drug Targets-With a Focus on SGLT2 Inhibitors.International journal of molecular sciences · 2024Review
- Odevixibat treatment in a child with hypoplastic left heart syndrome and severe cholestatic pruritus: a case report.Frontiers in pediatrics · 2024Article
Corrections and comments
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Authors and funding
4 authors at 4 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC) are rare, inherited cholestatic liver disorders that manifest in infants and children and are associated with impaired bile flow (ie cholestasis), pruritus and potentially fatal liver disease. There are no effective or approved pharmacologic treatments for these diseases (standard medical treatments are supportive only), and new, noninvasive options would be valuable. Typically, bile acids undergo biliary secretion and intestinal reabsorption (ie enterohepatic circulation). However, in these diseases, disrupted secretion of bile acids leads to their accumulation in the liver, which is thought to underlie pruritus and liver-damaging inflammation. One approach to reducing pathologic bile acid accumulation in the body is surgical biliary diversion, which interrupts the enterohepatic circulation (eg by diverting bile acids to an external stoma). These procedures can normalize serum bile acids, reduce pruritus and liver injury and improve quality of life. A novel, nonsurgical approach to interrupting the enterohepatic circulation is inhibition of the ileal bile acid transporter (IBAT), a key molecule in the enterohepatic circulation that reabsorbs bile acids from the intestine. IBAT inhibition has been shown to reduce serum bile acids and pruritus in trials of paediatric cholestatic liver diseases. This review explores the rationale of inhibition of the IBAT as a therapeutic target, describes IBAT inhibitors in development and summarizes the current data on interrupting the enterohepatic circulation as treatment for cholestatic liver diseases including ALGS and PFIC.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.