Early biochemical response to ursodeoxycholic acid predicts symptom development in patients with asymptomatic primary biliary cirrhosis.

PURPOSE: Among patients with asymptomatic primary biliary cirrhosis (a-PBC), a substantial portion ultimately develop symptoms suggestive of liver injury. Prognostic variables to distinguish patients likely to become symptomatic from patients who will remain asymptomatic need to be identified. We examined the impact of biochemical response to ursodeoxycholic acid in the development of symptoms in patients with a-PBC.

METHODS

Subjects comprised 83 patients with a-PBC treated using ursodeoxycholic acid (UDCA). All patients were followed regularly every 1-3 months. Response to treatment with UDCA was defined as a decrease in gamma-glutamyl transpeptidase (GGT) > or = 70% of pretreatment or normal levels from 6 months after start of treatment.

RESULTS

During the follow-up period (62.1 +/- 52.7 months), 12 patients (14.5%) developed liver-related symptoms. Incidence of the development of liver-related symptoms was significantly higher in UDCA non-responders than in responders (p < 0.001). Multivariate analysis showed that response to UDCA (improvement of GGT) represents an independent factor for predicting symptom development in patients with a-PBC.

CONCLUSIONS

Patients with a-PBC showing lack of biochemical response to UDCA by 6 months after treatment commencement should be considered for further treatments.