Potential Prognostic Markers Identified in Alagille Syndrome Guided Treatment

Total bilirubin and serum bile acid levels at week 48 of maralixibat (Livmarli™) treatment, as well as improvement in pruritus and age at which treatment was initiated, are associated with lower long-term rates of clinically important events and liver transplantation.

This is according to the results of an analysis examining predictors of long-term event-free survival and transplant-free survival in Alagille syndrome patients treated with maralixibat.

These findings identify potential prognostic markers that may help guide clinical decisions in Alagille syndrome patients treated with maralixibat, the researchers said.

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For the analysis, the researchers followed 76 patients with Alagille syndrome treated with maralixibat for up to 6 years. This was part of 3 long-term clinical trials.

The results showed that with over 6 years of maralixibat treatment, 16 of the 76 patients had a clinically important event. These included 10 liver transplantations, 3 decompensations, 2 deaths, and 1 surgical biliary diversion. The remaining 60 patients were event-free.

The researchers reported that variables that were predictive of event-free survival and transplant-free survival were total bilirubin levels, serum bile-acid levels, change in pruritus from baseline to week 48, and age of the patient at the start of the study.

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These results will be presented as a poster at the American Association for the Study of Liver Diseases annual meeting, which will take place between November 12 and 15, 2021. Ronald J. Sokol, MD, FAASLD, vice chair of Clinical and Translational Research in the Department of Pediatrics at the University of Colorado School of Medicine and Children’s Hospital Colorado will present the poster.

Maralixibat is an ileal bile acid transporter inhibitor developed by Mirum Pharmaceuticals and approved by the US Food and Drug Administration (FDA) to treat cholestatic pruritus in Alagille syndrome patients aged 1 year and older.