A new study has determine that clinical parameters, including serum bile acids (sBA), total bilirubin, and change in pruritus after maralixibat (MRX) treatment, in children with Alagille syndrome (ALGS) predict disease progression.

The study, published in Hepatology, evaluated event-free and transplant-free survival in the patients.

“In this analysis, we examined clinical outcomes for up to 6 years of treatment in participants with ALGS enrolled in MRX clinical trials,” the authors wrote. “Our primary aim was to identify predictors of event-free survival (absence of surgical biliary division, hepatic decompensation, liver transplant, or death) and transplant-free survival (absence of liver transplant or death) in children with ALGS treated with MRX.”

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MRX is the first drug approved by the US Food and Drug Administration for the treatment of cholestatic pruritus in patients with ALGS. It is an ileal bile acid transporter whose mechanism of action is to reduce the reuptake of bile acids in the ilium, which ultimately increases fecal excretion and reduces total sBA levels.

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The research team assessed 76 pediatric patients with AGLS undergoing treatment with MRX over a 48-week period and followed them for up to 6 years. The aim, in addition to identifying predictors of disease progression, was to establish cutoff points for response to treatment.

The results showed that sBA, total bilirubin, and change in pruritus levels were associated with improvements in event-free and transplant-free survival, and that those patients with clinically meaningful responses to MRX may have better survival and long-term outcomes, including liver transplant and surgical biliary division.

In terms of cutoff points, the data revealed an sBA threshold of 200 µmol/L at 48 weeks as an important predictor of event-free and transplant-free survival.

The authors concluded that the data obtained identify important prognostic biomarkers that can help guide clinical decision-making and treatment selection for pediatric patients with ALGS who are undergoing treatment with MRX.


Sokol RJ, Gonzales, EM, Binita KM, et al. Predictors of 6-year event-free survival in alagille syndrome patients treated with maralixibat, an ileal bile acid transporter inhibitor. Hepatology. Published online June 7, 2023. doi:10.1097/HEP.0000000000000502