Corpechot C et al. (2005) The effect of ursodeoxycholic acid therapy on the natural course of primary biliary cirrhosis. Gastroenterology 128: 297–303

Ursodeoxycholic acid (UDCA) is the only drug approved for treating primary biliary cirrhosis, but recent meta-analyses have cast doubt on its long-term benefits. Corpechot and colleagues have examined this in their recent prospective study.

The team used a multistate Markov model to allow the analysis of incomplete disease history data in 262 patients with primary biliary cirrhosis, all of whom received UDCA daily for a mean period of 8 years. Observed and predicted data on histologic stage progression, death, and orthotopic liver transplantation were similar, indicating that the model was valid.

When given in the early histologic stages of the disease, UDCA therapy appeared to improve survival; predicted 10-year and 20-year survival rates for patients in the first two stages of the disease were similar to those of an age-matched and sex-matched control population. Furthermore, survival was markedly better than predicted by the updated Mayo model. Patients in the third stage of the disease, however, were at significantly increased risk of death or orthotopic liver transplantation compared with the control population.

The study highlights the important impact of UDCA therapy when given early in the course of primary biliary cirrhosis, and emphasizes the need for new therapies for those in the advanced stages of the disease.