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Alagille Syndrome

IBAT inhibitors improve symptoms in children with inherited liver conditions

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Ileal bile acid transport inhibitors (IBATIs) inhibitors significantly improve pruritus and reduce serum bile acids in children with progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS) without increasing the risk of adverse events, according to a study.

The study reviewed 4 multicenter randomized controlled trials involving 215 patients with PFIC or ALGS. IBATIs significantly reduced pruritus scores, serum bile acids, and total bilirubin levels compared to placebo. More patients experienced clinically meaningful reductions in itch severity and improved bile acid response. IBATIs did not increase the risk of treatment-related adverse events.

The researchers concluded that IBATIs offer a promising treatment option with a favorable safety profile, though further studies are needed to determine optimal dosing and long-term benefits.

Reference
Imran M, Elsnhory AB, Ibrahim AA, et al. Efficacy and Safety of Ileal Bile Acid Transport Inhibitors in Inherited Cholestatic Liver Disorders: A Meta-analysis of Randomized Controlled Trials. J Clin Exp Hepatol. 2025;15(3):102462. doi: 10.1016/j.jceh.2024.102462. Epub 2024 Nov 21. PMID: 39802553; PMCID: PMC11720443.

Rare Disease 360® is the Official Media Partner and Official Publication of The Alagille Syndrome Alliance (Alagille.org).

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