Dr Corpechot and colleagues from Paris, France used a multistate modeling approach to assess the effect of ursodeoxycholic acid (UDCA) therapy on the natural course of primary biliary cirrhosis (PBC), which remains controversial.
The researchers included a total of 262 patients with primary biliary cirrhosis who had received 13–15 mg/kg ursodeoxycholic acid daily for a mean of 8 years (range, 1–22 years).
The research teams analyzed data using a multistate Markov model, with histologic stage progression, death, and orthotopic liver transplantation (OLT) as main end points.
Survival without orthotopic liver transplantation was compared with that predicted by the updated Mayo model and with the expected survival in the control population.
|Overall survival rates were 92% at 10 years and 82% at 20 years|
45 patients developed cirrhosis, 20 underwent orthotopic liver transplantation, and 16 died by the censor date.
Ten deaths were due to liver disease.
The overall survival rates were 92% at 10 years and 82% at 20 years.
Survival rates without orthotopic liver transplantation were 84% and 66% at 10 and 20 years, respectively, which were slightly lower than the survival rate of an age- and sex-matched control population but better than the spontaneous survival rate as predicted by the updated Mayo model.
The survival rate of patients in stage 1 and 2 was similar to that in the control population, whereas the probability of death or orthotopic liver transplantation remained significantly increased in treated patients in late histologic stages.
Treatment with ursodeoxycholic acid alone normalizes the survival rate of patients with primary biliary cirrhosis when given at early stages.
However, there is a continued need for new therapeutic options in patients with advanced disease.