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Alagille Syndrome
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Maralixibat improves health-related quality of life in children with ALGS

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Significant improvements in pruritus in children with Alagille syndrome ( ALGS) treated with maralixibat are clinically meaningful and are associated with improved health-related quality of life (HRQoL), according to a study.

Using data from 27 patients with ALGS with moderate-to-severe pruritus who were enrolled in the ICONIC trial, a Phase 2 study with a 4-week double-blind, placebo-controlled, randomized drug withdrawal period, researchers analyzed the impact of treatment response maralixibat. The clinically meaningful response was defined as a ≥1-point reduction in Itch-Reported Outcome (Observer) (ItchRO[Obs]) score, from baseline to week 48.

At week 48, 74% (n = 20) an ItchRO(Obs) treatment response. The mean (standard deviation) change in Multidimensional Fatigue Score was +25.8 (23.0) and -3.1 (19.8) for responders and non-responders, respectively. Non-statistically significant mean changes were seen for the Pediatric Quality of Life Inventory Generic Core and Family Impact scores.

Reference
Kamath BM, Goldstein A, Howard R, et al. Maralixibat Treatment Response in Alagille Syndrome Is Associated with Improved Health-Related Quality of Life. J Pediatr. 2022;S0022-3476(22)00791-0. doi: 10.1016/j.jpeds.2022.09.001. Epub ahead of print. PMID: 36096175.

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