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Maralixibat (Livmarli™) treatment improves the health-related quality of life of patients with Alagille syndrome, according to a study that will be presented this month at the virtual annual meeting of the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition. 

The study by Binita Kamath, MD, from the Division of Gastroenterology, Hepatology, and Nutrition at the University of Toronto in Canada retrospectively analyzed 48-week data from a phase 2 clinical trial called ICONIC. That was an open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period for children with Alagille syndrome, aged 1 to 18 years, the aim of which was to evaluate the safety and efficacy of maralixibat.

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The present study evaluated the pruritus response of maralixibat using the validated caregiver-reported Itch Reported Outcome (ItchRO) severity assessment tool as well as patients’ health-related quality of life using the Pediatric Quality of Life Inventory Generic Core (PedsQL).

The results showed that most patients (74%) achieved an ItchRO treatment response. Those who achieved a response had a greater health-related quality of life compared to those who did not achieve a response. 

Using statistical analyses, the researchers found that the ItchRO treatment response was associated with a clinically meaningful improvement in health-related quality of life at week 48. 

Sleep-related items in PedsQL such as difficulty sleeping, feeling tired, excessive sleeping, difficulty sleeping through the night, feeling tired upon waking, and taking a lot of naps were significantly changed at week 48 in responders compared to nonresponders.

“This analysis on pruritus, and its association with sleep disturbance and fatigue, warrants further exploration,” the researchers concluded.

One of the main symptoms of Alagille syndrome is pruritus, which occurs along with jaundice and xanthomas and can have a great impact on patients’ health-related quality of life.

Maralixibat is an oral solution to treat cholestatic pruritus in patients with Alagille syndrome aged 1 year and older.


Kamath B, Goldstein A, Howard R, et al. Response to treatment with maralixibat in Alagille syndrome is associated with improved health-related quality of life. Poster presented at: the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition Annual Meeting 2021: December 12-18, 2021; Virtual.

Safety and efficacy study of LUM001 (maralixibat) with a drug withdrawal period in participants with Alagille syndrome (ALGS) (ICONIC). US National Library of Medicine. Last updated July 14, 2021. Accessed December 9, 2021.