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Alagille Syndrome

Study shows maralixibat’s positive impact on Alagille syndrome outcomes

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By comparing 6-year outcomes from maralixibat trials with real-world data from the Global ALagille Alliance (GALA) study, researchers demonstrated that maralixibat significantly improved event-free survival (EFS) in patients with Alagille syndrome (ALGS) compared to those in the GALA cohort.

The maralixibat trials involved 84 patients with ALGS who received treatment for up to 6 years while the comparative cohort aligned GALA study with key maralixibat eligibility criteria resulting in a filtered cohort of 469 participants.

The comparison focused on EFS, defined as the time to the first occurrence of portal hypertension manifestations, surgical biliary diversion, liver transplant, or death. The analysis, demonstrated a significantly better EFS in the maralixibat cohort compared to the GALA cohort (hazard ratio 0.305; 95% CI, 0.189-0.491; P < 0.0001). Sensitivity analyses and adjustments for covariates, including age, total bilirubin, gamma-glutamyl transferase, and alanine aminotransferase, were applied, revealing consistent findings.

An innovative aspect of the study involved the use of maximum likelihood estimation to align disease severity between the 2 cohorts at the initiation of maralixibat. Despite the routine exclusion of serum bile acids from the GALA filtering criteria, multiple sensitivity and subgroup analyses, including serum bile acids availability, supported the robustness of the results.

Reference
Hansen BE, Vandriel SM, Vig P, et al; Global ALagille Alliance (GALA) Study Group. Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA. Hepatology. 2023 Dec 25. doi: 10.1097/HEP.0000000000000727. Epub ahead of print. PMID: 38146932.

 

Rare Disease 360® is the Official Media Partner and Official Publication of The Alagille Syndrome Alliance (Alagille.org).

 

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