UDCA treatment reduces biliary tract cancer in primary sclerosing cholangitis

Ursodeoxycholic acid treatment correlated with reduced mortality, need for liver transplantation and biliary tract cancer in patients with primary sclerosing cholangitis, according to a presenter at The Liver Meeting Digital Experience.

“UDCA was likely to be associated with a reduction with biliary tract cancer but not consistently significant, partly due to small sample size,” Toshihiko Arizumi, MD, an assistant professor in the department of internal medicine at Teikyo University School of Medicine, said during his presentation.

Arizumi and colleagues identified 325 patients with primary sclerosing cholangitis. Data included were sex, age at diagnosis, blood tests and fibrosis and fibrosis-4 (FIB-4) index at diagnosis, presence of any symptom at diagnosis, history/presence of inflammatory bowel disease, treatment with UDCA and/or bezafibrate, development of biliary tract cancer and history of liver transplantation.

Arizumi said the median observational period was 5.1 years.

In total, 278 patients received UDCA and 78 patients received bezafibrate. Investigators reported 57 patients died, 24 patients underwent liver transplantation and 26 patients developed biliary tract cancer during observation. UDCA treatment correlated with an improvement of liver transplantation-free survival (adjusted HR = 0.467; 95% CI. 0.28-0.778) and a decrease in development of biliary tract cancer (aHR = 0.324; 95% CI, 0.135-0.778).

Results from an inverse probability treatment weighting-adjusted model showed a similar correlation between UDCA treatment and liver transplantation-free survival (aHR = 0 429; 95% CI, 0.245-0.753). However, the correlation did not remain regarding biliary tract cancer (aHR = 0.418; 95%CI, 0.158-1.104).

“We believe these findings definitely provide the most robust evidence to show clinical benefit of UDCA in PSC for the time being, and this will encourage more treatment with UDCA in patients with PSC,” Arizumi said in an AASLD press release. “Nevertheless, we don’t consider that this cohort has enough statistical power due to its medium sample size. A large-scale cohort with an international collaboration is required to produce more convincing evidence of UDCA in PSC.”