Ten-year survival in ursodeoxycholic acid–treated patients with primary biliary cirrhosis



Ursodeoxycholic acid (UDCA) treatment has been shown to increase survival without orthotopic liver transplantation (OLT) in patients with primary biliary cirrhosis (PBC) at 4 years. Whether this beneficial effect was maintained over the long term remained to be established. In a large cohort of UDCA-treated patients with PBC, we aimed to determine the 10-year outcome of these patients using two endpoints: (1) survival without OLT, and (2) survival. The cohort was comprised of 225 patients with PBC treated with UDCA (13-15 mg/kg/d) monitored from the beginning of treatment until time of last follow-up, OLT, or death. Because of the absence of a control group, survival without OLT was compared with survival predicted by the Mayo model (first 7 years), and observed 10-year survival with an estimation of survival of a standardized control cohort of the French population. Observed survival without OLT of UDCA-treated patients was significantly higher (P < .04) than survival predicted by the Mayo model. Observed survival was significantly lower (P < .01) than survival predicted from the French population. Observed survival of noncirrhotic patients was not different (P > .9) from that of the French control population but survival of cirrhotic patients was significantly lower (P < .0001). Twenty-two patients died; 13 patients died of hepatic causes and 4 patients died after OLT. In conclusion, survival without OLT among patients treated with UDCA for PBC is higher than that of untreated patients, as predicted by the Mayo model. Ten-year survival among UDCA-treated patients is slightly lower than that of an age- and sex-matched general population, the difference mainly being explained by mortality among cirrhotic patients