WASHINGTON — In a dose-varied study of maralixibat, patients’ caregivers reported trends toward pruritis relief while the drug also demonstrated safety, according to a presenter at The Liver Meeting 2017.

“Although the pre-specified primary analyses of ItchRO were not all statistically significant, the data suggest that maralixibat was safe and may reduce pruritus in Alagille Syndrome,” Benjamin L. Shneider, MD, from Texas Children’s Hospital, said during the presidential plenary.

Shneider and colleagues used the Itch Reported Outcome (ItchRO) tool, a novel electronic diary in which caretakers report each day’s score from 0 to 4 – with 4 being mutilation of the skin, based upon the child’s itch-related symptoms overnight. This was supported by a clinical provider report as well.

Maralixibat delivery was in a dose escalation fashion for 5 weeks with stable dose treatment for an additional 8 weeks, Shneider said. Patients had follow-up with their provider at 4 weeks. Researchers compared the two highest tolerated doses of maralixibat vs. placeboand compared pooled doses to the placebo.

Participants received either placebo (n = 12), 70 µg/kg per day (n = 8), 140 µg/kg per day (n = 11) or 280 µg/kg per day (n = 6) and the primary endpoint was change in baseline to week 13 in observer-based ItchRO. The secondary endpoint was serum bile acids.

Shneider showed that the 70 µg/kg dose and the 140 µg/kg significantly impacted the ItchRO score (P = .032 and .014, respectively) at week 13 and, additionally, the 70 µg/kg dose significantly lowered serum bile acid (P = .016) at week 13. All other relationships were not significant, but patients often responded to the medication.

“Determination of optimal dosing and further assessments of safety and efficacy in children with cholestasis are warranted,” Shneider said. – by Katrina Altersitz


Shneider BL, et al. Abstract 144. Presented at: The Liver Meeting; Oct. 20-24, 2017; Washington, D.C.

Disclosure: Shneider reports consulting and holding stocks for Bristol Myers Squibb.


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