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Alagille Syndrome
Conference Roundup

Maralixibat response associated with health-related quality of life improvements in ALGS

Posted on January 5, 2022

Children with Alagille syndrome (ALGS) who responded to treatment with maralixibat experienced improvements in health-related quality of life (HRQoL) measures, according to data from a post-hoc analysis of the ICONIC study presented at the North American Society for Pediatric Gastroenterology Hepatology and Nutrition Annual Meeting.

In this analysis, 27 patients with ALGS treated with 380 µg/kg per day of maralixibat were followed from baseline through 48 weeks. At 48 weeks, 74% (n = 20) had ≥1 point reduction in caregiver Itch-Reported Outcome.

Compared with non-responders, patients who responded to treatment showed improvement in HRQoL measures including, an increase of 13.9 points in Multidimensional Fatigue Total Scale Score from baseline to 48 weeks; larger changes in 6 sleep-related items; and an average increase of 8.8 points on the PedsQL Generic Core Total Scale Score.

Reference
Kamath BM, et al. Response to treatment with maralixibat in Alagille syndrome is associated with improved health-related quality of life. Presented at: North American Society for Pediatric Gastroenterology Hepatology and Nutrition Annual Meeting (Virtual).

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