Alagille Syndrome
Conference Roundup

Prognostic markers in maralixibat treated ALGS may guide clinical decision-making

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Total bilirubin (TB) and total serum bile acids (sBA) 48 weeks from the first dose of maralixibat (MRX) in patients with Alagille syndrome (ALGS) were associated with lower long-term rates of clinically important events and transplantation, according to a presentation at the American Association for the Study of Liver Diseases annual congress, The Liver Meeting. Improvement in pruritus and the patient’s age at the start of MRX treatment were also associated with lower rates.

In this analysis, 76 patients with ALGS treated with MRX in 3 long-term clinical trials were followed for up to 6 years. Over the course of the study, 60 patients remained event-free and 16 had events, including liver transplantation (n = 10), decompensation (n = 3), death (n = 2), and surgical biliary diversion (n = 1).

At week 48, patients with TB of <6.5 had a 6-year EFS of 90% compared with 43% for patients with a TB of ≥6.5 mg/dL. At week 48, patients with sBA <200 had a 6-year EFS of 85% compared with 49% for patients with a sBA ≥200 µmol/L.

A >1-point reduction on the ItchRO (Obs) scale used to measure pruritus from baseline to week 48 was a significant predictor of EFS. The 6-year EFS for patients with a >1-point reduction was 88% compared with 57% for patients with ≤1-point reduction

Patients who started treatment with MRX before the age of 36 months were at a higher risk for an event.

Sokol R, et al. Predictors of 6-Year Event-Free Survival in Patients with Alagille Syndrome Treated with maralixibat, an IBAT Inhibitor. Presented at: The Liver Meeting 2021.