UDCA Use Linked to Lower Cancer Risk in Primary Biliary Cholangitis, Large EHR Study Finds
Ursodeoxycholic acid (UDCA), the standard first-line therapy for primary biliary cholangitis (PBC), was associated with a significantly lower risk of several cancers in a large, propensity-matched cohort study using real-world electronic health record data. The findings address longstanding uncertainty about cancer risk in patients with PBC treated with UDCA.
Investigators analyzed data from the TriNetX Research Network, which includes records from more than 156 million patients worldwide. From this dataset, more than 48,000 adults with PBC were identified. After applying eligibility criteria and 1:1 propensity score matching, 6,057 UDCA-treated patients were compared with 6,057 nonusers. Patients had no prior cancer and were followed for cancer outcomes occurring at least 1 year after treatment initiation or diagnosis.
UDCA treatment was associated with a significantly reduced risk of gastrointestinal cancers overall, with a hazard ratio of 0.59 compared with nonusers. Reductions were also observed for liver cancer (HR, 0.54) and breast cancer among women (HR, 0.36). In contrast, UDCA use was not associated with a statistically significant reduction in colorectal cancer risk (HR, 0.54; 95% CI, 0.25–1.20). Results were consistent across multiple analytic approaches, including sensitivity analyses using alternative index dates.
The authors concluded that “UDCA treatment was associated with significantly lower risks of gastrointestinal cancer, liver cancer, and breast cancer but not colorectal cancer in patients with PBC.” They noted that the study’s strengths included its large sample size, robust confounder control, and minimized immortal time bias.
The potential implications extend beyond disease control. According to the authors, the findings “suggest a potential chemopreventive effect of UDCA beyond its hepatic benefits.” While the mechanisms remain uncertain, they proposed that anti-inflammatory, antiproliferative, and cytoprotective effects may contribute.
The authors emphasized that prospective studies are needed to confirm these observations, particularly for liver and breast cancer.
Reference
Su L, Patel PJ, Shu X. Use of ursodeoxycholic acid and cancer risk for patients with primary biliary cholangitis. JAMA Network Open. 2025;8(12):e2550907. doi.org/10.1001/jamanetworkopen.2025.50907
