Ursodeoxycholic acid therapy and liver transplant-free survival in patients with primary biliary cholangitis.

Authors:
Maren H Harms
Maren H Harms
Erasmus University Medical Center Rotterdam
Christophe Corpechot
Christophe Corpechot
Service d'Hépatologie
Douglas Thorburn
Douglas Thorburn
Queen Elizabeth Hospital
United Kingdom
Harry L A Janssen
Harry L A Janssen
North Dakota State University
Keith D Lindor
Keith D Lindor
Mayo Clinic
United States
Gideon M Hirschfield
Gideon M Hirschfield
University of Birmingham
Birmingham | United States
Albert Pares, MD, PhD
Albert Pares, MD, PhD
Hospital Clinic, University of Barcelona
Professor
Internal Medicine, Hepatology
Barcelona | Spain

J Hepatol 2019 Apr 11. Epub 2019 Apr 11.

Department of Gastroenterology and Hepatology, Erasmus University Medical Center, Rotterdam, The Netherlands. Electronic address:

Background & Aims: The clinical efficacy of ursodeoxycholic acid (UDCA) in primary biliary cholangitis (PBC) remains subject to debate as definitive randomized controlled trials are lacking. We aimed to determine whether UDCA prolongs liver transplant (LT)-free survival in patients with PBC.

Methods: This international cohort study included patients from the Global PBC Study Group database, originating from 8 countries in Europe and North America. Both UDCA-treated and untreated patients were included. LT and death were assessed as a combined endpoint through Cox regression analyses, with inverse probability treatment weighting (IPTW).

Results: In the 3,902 patients included, the mean (SD) age was 54.3 (11.9) years, 3,552 patients (94.0%) were female, 3,529 patients (90.4%) were treated with UDCA and 373 patients (9.6%) were not treated. The median (interquartile range) follow-up was 7.8 (4.1-12.1) years. In total, 721 UDCA-treated patients and 145 untreated patients died or underwent LT. After IPTW, the 10-year cumulative LT-free survival was 79.7% (95% CI 78.1-81.2) among UDCA-treated patients and 60.7% (95% CI 58.2-63.4) among untreated patients (p <0.001). UDCA was associated with a statistically significant reduced risk of LT or death (hazard ratio 0.46, 95% CI 0.40-0.52; p <0.001). The hazard ratio remained statistically significant in all stages of disease. Patients classified as inadequate biochemical responders after 1 year of UDCA had a lower risk of LT or death than patients who were not treated (adjusted hazard ratio 0.56; 95% CI 0.45-0.69; p <0.001).

Conclusion: The use of UDCA improves LT-free survival among patients with PBC, regardless of the disease stage and the observed biochemical response. These findings support UDCA as the current universal standard of care in PBC.

Lay Summary: In this international multicenter study of 3,902 patients with primary biliary cholangitis, we found that treatment with ursodeoxycholic acid is associated with prolonged liver transplant-free survival. This association was significant, irrespective of sex, age, or disease stage. The survival benefit remained statistically significant in patients with an incomplete biochemical response to ursodeoxycholic acid therapy.

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http://dx.doi.org/10.1016/j.jhep.2019.04.001DOI Listing
April 2019
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