Alagille Syndrome
Conference Roundup

Real-world data may help assess long-term outcomes in ALGS clinical research

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The potential use of real-world data to analyze long-term outcomes in interventional studies when utilizing placebo is not possible was discussed during a NASPGHAN 2022 presentation of data from a study comparing patients with Alagille syndrome (ALGS) treated with maralixibat for up to 6 years and an external control cohort using real-world data

Data from the external cohort was derived from the Global Alagille Alliance (GALA) Study Group, which is an aggregated global natural history clinical database established for ALGS.

“Long-term, randomized controlled trials with definitive clinical outcomes are difficult, if not impossible, to conduct in rare diseases,” Bettina E. Hansen, PhD said during the presentation, but use of real-world data as a control arm may be possible to better assess long-term outcomes.

Demographic characteristics and disease characteristics were well-balanced between the GALA control cohorot (n = 469) and the maralixibat cohort (n = 84).

In this 6-year analysis, a 70% reduction in clinical outcomes with maralixibat treatment was found compared with natural history in patients with ALGS, as well as highly statistically significant improvements in transplant-free survival.

Hansen BE, et al. Maralixibat-Treated Patients with Alagille Syndrome Demonstrate Improved Event-Free Survival in a Natural History Comparison with Patients from the GALA Database: Application of Real-World Evidence Analytics. Presented at NASPGHAN 2022.

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