- In patients with primary biliary cholangitis (PBC), treatment with ursodeoxycholic acid (UDCA) is associated with prolonged liver transplantation (LT)-free survival, irrespective of disease stages and observed biochemical response to UDCA.
Why this matters
- Findings imply a strong recommendation for all patients with PBC to use UDCA.
- This international cohort study included 3529 UDCA-treated patients and 373 untreated patients using data from the Global PBC Study Group.
- Primary endpoint: composite endpoint (LT and all-cause mortality).
- Funding: Intercept Pharmaceuticals and Foundation for Liver and Gastrointestinal Research, Netherlands.
- During a median follow-up of 7.8 years, 721 UDCA-treated patients and 145 untreated patients died or underwent LT.
- The incidence of composite endpoint was lower in UDCA-treated patients vs untreated patients (23.21 vs 58.81 per 1000 person-years; P<.001>
- After inverse probability of treatment weighting adjustment, the cumulative LT-free survival was higher in UDCA-treated patients at 5 years (90.8% vs 81.0%; P<.001 and years vs p compared with untreated patients.>
- The survival benefits were statistically significant in early, intermediate and advanced stages of PBC (P<.0001 for all>
- Patients with inadequate response to UDCA had a significantly lower risk for composite endpoint vs untreated patients (adjusted HR [aHR], 0.56; P<.001 but the favourable lt-free survival opposed to those without therapy was strong in udca responders p>
- Residual confounders.
- Time-dependent bias.