The use of maralixibat in children with Alagille syndrome is associated with better health-related quality of life outcomes, researchers reported in a study published in The Journal of Pediatrics.

Studies have demonstrated that children with this disease have much lower health-related quality of life scores than healthy age-matched individuals. One of the more unpleasant symptoms they experience is pruritus, which can be severe enough to negatively affect their emotional and physical well-being. Eventually, many children with Alagille syndrome will require a liver transplant to relieve intractable pruritus.

A landmark study, named ICONIC, demonstrated that patients with Alagille syndrome who received maralixibat experienced significant improvements in pruritus. “This is the first agent to demonstrate significant, durable, and clinically meaningful improvements in pruritus among children with [Alagille syndrome],” the authors of the present study wrote. 

Continue Reading

The present study used data from that study to investigate the impact of maralixibat on health-related quality of life parameters among children with Alagille syndrome. 

Read more about Alagille syndrome etiology 

The research team obtained data from the ICONIC study, which included participants from 9 locations in Australia and Europe. Participants must have had a clinical diagnosis of Alagille syndrome and been under the age of 18 years. The study analyzed health-related quality of life scores at baseline and at week 48 using the Pediatric Quality of Life Inventory™ 4.0 Generic Core Scales. 

Patients taking maralixibat reported an increase in health-related quality of life scores across all domains evaluated. In addition, this study established a clear relationship between the extent of pruritus and health-related quality of life scores; furthermore, it showed that the treatment of pruritus using drugs such as maralixibat could significantly improve quality of life. 

“As pruritus is a leading indication for liver transplant among patients with [Alagille syndrome], ongoing evaluation of outcomes in this pediatric population should include assessment of the future impact of treatment on the burden of liver transplantation,” the authors of the study wrote.


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. Published online September 10, 2022. doi:10.1016/j.jpeds.2022.09.001