Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA.

ABSTRACT:

Background and aims

Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is an approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the G lobal AL agille A lliance (GALA) study.

Approach and results

Maralixibat trials comprise 84 patients with ALGS with up to 6 years of treatment. GALA contains retrospective data from 1438 participants. GALA was filtered to align with key maralixibat eligibility criteria, yield

SUBMITTER: Hansen BE 

PROVIDER: S-EPMC11095900 | biostudies-literature | 2024 Jun

REPOSITORIES: biostudies-literature