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

Publication Title

Journal of hepatology

Authors

Maren H Harms
Henk R van Buuren
Christophe Corpechot
Douglas Thorburn
Harry L A Janssen
Keith D Lindor
Gideon M Hirschfield
Albert Parés
Annarosa Floreani
Marlyn J Mayo
Pietro Invernizzi
Pier Maria Battezzati
Frederik Nevens
Cyriel Y Ponsioen
Andrew L Mason
Kris V Kowdley, Liver Care Network and Organ Care Research, Swedish Medical Center, 1124 Columbia Street, Suite 600, Seattle, WA, 98104, USAFollow
Willem J Lammers
Bettina E Hansen
Adriaan J van der Meer

Document Type

Article

Publication Date

4-11-2019

Keywords

Cholestasis; Clinical trials; Mortality; Patient management; Treatment; UDCA, transplantation

Abstract

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

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.

Clinical Institute

Digestive Health

Specialty/Research Institute

Hepatology