Ten-year survival in ursodeoxycholic acid-treated patients with primary biliary cirrhosis. The UDCA-PBC Study Group.

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.