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Alagille Syndrome
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Good outcomes with different dose of IBAT inhibitors seen in patients with AGLS

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Pediatric patients with Alagille syndrome (ALGS) had a significant reduction in serum bile acid and pruritus when treated with different doses of maralixibat or odevixibat, according to a study.

In the systematic review and meta-analysis, 94 patients with ALGS with cholestasis from 4 studies treated with either maralixibat or odevixibat (intervention group) or placebo, were evaluated.

Patients in the intervention group had a significant reduction in sBAs levels (−75.8 μmol/L) and a significant improvement (1.8 points) in Itch-Reported Outcome scores. All quality-of-life assessments also noted significant improvements. An increase in alanine aminotransferase levels were found.

“Further trials may consider spacing the dosing frequency to reduce the gastrointestinal side effects while still retaining the positive effects on pruritus and sBAs,” the authors concluded.

Reference
Muntaha HST, Munir M, Sajid SH, et al. Ileal Bile Acid Transporter Blockers for Cholestatic Liver Disease in Pediatric Patients with Alagille Syndrome: A Systematic Review and Meta-Analysis. J Clin Med. 2022;11(24):7526. doi: 10.3390/jcm11247526. PMID: 36556142; PMCID: PMC9784790.

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