Mirum Pharmaceuticals announced that data from LIVMARLI or maralixibat, oral solution clinical studies evaluating patients with Alagille syndrome, ALGS, were published in Hepatology. LIVMARLI is the first and only approved treatment for cholestatic pruritus in patients with Alagille syndrome three months of age and older. The overall findings from the retrospective analysis suggest that patients with ALGS treated with LIVMARLI who meet the identified prognostic parameters may have improved six-year survival and clinical outcomes. In the analysis, 46 pre-specified variables were evaluated as potential predictors of event-free and transplant-free survival from the LIVMARLI ALGS clinical program. Patients were assessed for up to six years; EFS was defined as absence of liver transplant , surgical biliary diversion SBD , hepatic decompensation, or death; TFS was absence of LT or death…”Complications of cholestasis, including refractory pruritus, are among the main indications for liver transplantation in patients with Alagille syndrome and many children will unfortunately require a liver transplant before adulthood,” said Ronald J. Sokol, MD, chief scientific officer, child health, Children’s Hospital Colorado. “This six-year analysis provides important insight into which markers predict transplant-free and event-free survival in those receiving LIVMARLI, and therefore can help guide medical management of patients receiving LIVMARLI.”
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