Rifaximin in Pediatric Hepatic Encephalopathy: First Trial, Ammonia Miss

This first-ever pediatric trial of rifaximin for hepatic encephalopathy provides safety, pharmacokinetic, and neurocognitive data that support off-label use in children with shunt-related hyperammonemia, though the ammonia endpoint was not met.

rifaximinrifamycin antibiotichepatic encephalopathyhyperammonemiapediatriccongenital portosystemic shuntopen-label trialpharmacokineticsgut microbiotaJapan Gastroenterology / Hepatology

Background Information

Rifaximin shows promise in pediatric hepatic encephalopathy treatment.

Hepatic encephalopathy (HE) significantly affects children's health, and rifaximin's role in treatment needs clear evaluation, especially regarding efficacy, safety, and dosage. Rifaximin treatment led to significant improvements in mental state and cognitive tests in pediatric patients with HE, but changes in blood ammonia concentration were not statistically significant. This study shows rifaximin's safety and efficacy profiles in children are comparable to adults, supporting its potential use in pediatric HE management. Rifaximin may improve mental state in children with hepatic encephalopathy, though biochemical changes are minimal.

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Authors: Okajima H, Tagawa M, Fujisawa M, Ueda K

Citation: Okajima H, Tagawa M, Fujisawa M, et al. Efficacy, safety, and pharmacokinetics of rifaximin in pediatric patients with hepatic encephalopathy in Japan: An open-label study. Hepatology communications. 2026;10(8). doi:10.1097/HC9.0000000000001002

Article Links: PubMed · Journal / DOI

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