Ileal bile acid transporter inhibitors in paediatric cholestatic liver disease: a class-effect systematic review and meta-analysis
Abstract
Aim: Ileal bile acid transporter inhibitors are approved for cholestatic pruritus in progressive familial intrahepatic cholestasis and Alagille syndrome. Previous meta-analyses covered inherited disease only and did not test whether the class effect is constant across diseases. We estimated the effect of these inhibitors versus placebo across the three paediatric cholestatic diseases. Methods: We searched MEDLINE, Embase, Web of Science and CENTRAL from inception to 5 June 2026 for double-blind, placebo-controlled trials of odevixibat or maralixibat in children with cholestatic liver disease; only parallel-group trials entered the primary pool. Main outcomes were change in serum bile acids and pruritus score. Mean differences and risk ratios were pooled with restricted maximum likelihood random-effects models and Hartung-Knapp confidence intervals, with prediction intervals and I2, in R. Results: Nineteen reports were included; five trials (290 children) entered the synthesis. These inhibitors lowered serum bile acids by 103.0 µmol/L (95% confidence interval −164.7 to −41.3; I2 47.9%; prediction interval −217.3 to 11.3) and pruritus by 0.92 points (−1.26 to −0.59; I2 0%). Pruritus response was more frequent with treatment (risk ratio 2.61, 1.32 to 5.15). Odevixibat and maralixibat did not differ. Alanine aminotransferase fell in progressive familial intrahepatic cholestasis and rose in Alagille syndrome. Serious adverse events matched placebo. Conclusion: Ileal bile acid…
How strong is the evidence?
Evidence Hub reads this as a meta-analysis. Its evidence level is 1 of 5 (Highest · pooled evidence). Pooled evidence from several studies sits at the top of the evidence pyramid. Reported size: 290 participants or records.
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