Background and aims: The therapeutic potential of ursodeoxycholic acid (UDCA) treatment in primary biliary cirrhosis (PBC) remains controversial. In addition, relatively few data have been reported on the outcome of patients who have been treated long term. The aim of the present study was to document long-term survival of a prospectively followed large cohort of UDCA treated patients in comparison to that predicted by the Mayo model and of a matched control cohort of the Dutch population.
Methods: Two hundred ninety-seven patients were included and followed during a median period of 68 (range 3-126) months until death or the end of the study.
Results: Survival free of transplantation (1 yr 99.7%, 5 yr 87%, and 10 yr 71%) was significantly better than predicted by the Mayo model (p= 0.01). However, for patients with abnormal serum bilirubin and/or albumin concentrations at entry, observed and predicted survival did not significantly differ. Compared with survival for a standardized cohort of the Dutch population, observed survival for the total group was significantly decreased (p= 0.0003); for noncirrhotic patients and patients with normal entry bilirubin and albumin concentrations survival was comparable. Serum bilirubin and albumin concentrations were the prognostic factors most consistently associated with survival.
Conclusions: A 10-year prognosis for most UDCA-treated patients with PBC, i.e., those with a normal bilirubin and albumin concentration, is comparable to that of a matched general population. Our finding that observed survival was significantly better than predicted by the Mayo model may suggest that this model did not accurately predict prognosis in our cohort. Alternatively, this finding indicates an important therapeutic effect of long-term UDCA treatment in PBC, particularly in patients with noncirrhotic, nonadvanced disease.