Biochemical response to ursodeoxycholic acid predicts long-term outcome in Japanese patients with primary biliary cirrhosis.

Aim:   There is an ongoing need for predictors of long-term outcomes for patients with primary biliary cirrhosis (PBC). Biochemical response to ursodeoxycholic acid (UDCA) has been introduced to predict development of symptoms by our group (Ehime criteria) and to predict long-term outcomes in Western countries (Paris, Barcelona and Rotterdam criteria). The aim of this study was to evaluate whether these criteria are also useful to predict long-term outcomes in Japanese patients with PBC.

Methods:   A retrospective chart review was conducted for 227 Japanese patients with PBC. Patients taking UDCA with an observation period of more than 6 months were included in the study. Data collection included demographics, biochemical and serological markers, and histological stage. Four different criteria regarding biochemical response to UDCA were compared and evaluated.

Results:   In total, 138 patients met the inclusion criteria and underwent analysis. Using the Ehime criteria, the transplant-free survival rate was significantly higher in responders than in non-responders (P = 0.010). The Paris criteria also predicted long-term outcomes in our population (P = 0.003), whereas the Barcelona and Rotterdam criteria showed no such association (P = 0.282 and P = 0.553, respectively).

Conclusion:   Good biochemical response to UDCA according to the Ehime and Paris criteria is associated with long-term outcome in Japanese patients with PBC and allows identification of non-responders who may benefit from further trials. Finally, Ehime criteria should be validated in a different patient cohort.