Maralixibat improves health-related quality of life in pediatric patients with Alagille Syndrome

A recent study demonstrated that the ileal bile acid transporter inhibitor maralixibat improves health-related quality of life (HRQoL) in children with Alagille syndrome. This study was published in The Journal of Pediatrics.

The ICONIC trial was a phase 2 study, having a 4-week double-blind, placebo-controlled, randomized drug withdrawal period. The study included 27 children having Alagille syndrome with moderate-to-severe pruritus. From baseline to week 48, the treatment response to maralixibat was noted using Itch-Reported Outcome (Observer) score. The HRQoL was assessed based on the certain scale scores that included Pediatric Quality of Life Inventory Generic Core, Family Impact, and Multidimensional Fatigue scale scores.

At week 48, twenty patients attained an Itch-Reported Outcome (Observer) treatment score response. The mean (SD) change in Multidimensional Fatigue score was higher was higher for responders over non-responders. The Pediatric Quality of Life Inventory Generic Core and Multidimensional Fatigue scores showed smaller and non-statistically significant point estimates.

Based on the results of the study, it can be concluded that maralixibat shows significant improvement in pruritis, thereby enhancing the quality of life in the affected children.