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Ursodeoxycholic Acid Response Is Associated With Reduced Mortality in Primary Biliary Cholangitis With Compensated
Binu V John1, Nidah S Khakoo2, Kaley B Schwartz1
1Division of Hepatology, Bruce W Carter VA Medical Center, Miami, Florida, USA.
Insights
Response to ursodeoxycholic acid (UDCA) reduces liver complications and death in male primary biliary cholangitis (PBC) patients with cirrhosis. The greatest benefit was observed in those with portal hypertension.
Area of Science:
- Hepatology
- Clinical Medicine
- Pharmacology
Background:
- Primary biliary cholangitis (PBC) studies often under-represent male patients and those with cirrhosis.
- Ursodeoxycholic acid (UDCA) is a key treatment for PBC, but its effectiveness in specific patient groups requires further investigation.
Purpose of the Study:
- To assess the association between UDCA response and clinical outcomes in male patients with PBC and cirrhosis.
- To evaluate the impact of UDCA response on liver-related death or transplantation, hepatic decompensation, and hepatocellular carcinoma (HCC).
Main Methods:
- Retrospective cohort study of 501 male veterans with PBC and compensated cirrhosis.
- Utilized competing risk time-updating Cox proportional hazards models to analyze UDCA response.
- Follow-up included assessment of all-cause and liver-related mortality or transplantation, hepatic decompensation, and HCC development.
Main Results:
- UDCA responders showed significantly lower rates of hepatic decompensation and liver-related death or transplantation compared to partial responders.
- UDCA response was linked to a reduced risk of hepatic decompensation, all-cause death or transplantation, and liver-related death or transplantation.
- No significant association was found between UDCA response and HCC development. The effect was most pronounced in patients with portal hypertension.
Conclusions:
- UDCA response is associated with improved clinical outcomes, including reduced decompensation and mortality, in male patients with PBC cirrhosis.
- The study highlights the importance of considering UDCA response in managing PBC, particularly in male populations and those with portal hypertension.
Introduction:
Patients with cirrhosis and men have been under-represented in most studies examining the clinical benefit of response to ursodeoxycholic acid (UDCA) in primary biliary cholangitis (PBC). The aim of this study was to study the association of UDCA response and liver-related death or transplantation, hepatic decompensation, and hepatocellular carcinoma (HCC) in patients with PBC cirrhosis.
Methods:
We conducted a retrospective cohort study of veterans, predominantly men, with PBC and compensated cirrhosis to assess the association of UDCA response with the development of all-cause and liver-related mortality or transplantation, hepatic decompensation, and HCC using competing risk time-updating Cox proportional hazards models.
Results:
We identified 501 subjects with PBC and compensated cirrhosis, including 287 UDCA responders (1,692.8 patient-years [PY] of follow-up) and 214 partial responders (838.9 PY of follow-up). The unadjusted rates of hepatic decompensation (3.8 vs 7.9 per 100 PY, P < 0.0001) and liver-related death or transplantation (3.7 vs 6.2 per 100 PY, P < 0.0001) were lower in UDCA responders compared with partial responders. UDCA response was associated with a lower risk of hepatic decompensation (subhazard ratio [sHR] 0.54, 95% confidence interval [CI] 0.31-0.95, P = 0.03), death from any cause or transplantation (adjusted hazard ratio 0.49, 95% CI 0.33-0.72, P = 0.0002), and liver-related death or transplantation (sHR 0.40, 95% CI 0.24-0.67, P = 0.0004), but not HCC (sHR 0.39, 95% CI 0.60-2.55, P = 0.32). In a sensitivity analysis, the presence of portal hypertension was associated with the highest UDCA-associated effect.
Discussion:
UDCA response is associated with a reduction in decompensation, all-cause, and liver-related death or transplantation in a cohort of predominantly male patients with cirrhosis, with the highest benefit in patients with portal hypertension.
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