Primary biliary cholangitis: ursodeoxycholic acid improves liver transplant-free survival

  • Harms MH & al.
  • J Hepatol
  • 10 Apr 2019

  • curated by Sarfaroj Khan
  • UK Clinical Digest
Access to the full content of this site is available only to registered healthcare professionals. Access to the full content of this site is available only to registered healthcare professionals.

Takeaway

  • 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.

Study design

  • 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.

Key results

  • 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>

Limitations

  • Residual confounders.
  • Time-dependent bias.