Biochemical responses to bezafibrate improve long-term outcome in asymptomatic patients with primary biliary cirrhosis refractory to UDCA

Background Bezafibrate is reported to have biochemical efficacy for patients with primary biliary cirrhosis (PBC) refractory to ursodeoxycholic acid (UDCA), yet the long-term effect is still unknown. In Japan, nationwide surveys of PBC have been performed since 1980. In the current study, we retrospectively examined whether response to bezafibrate treatment is associated with the long-term outcomes using this large-scale database. Methods Among 7,376 patients in the database, we enrolled patients who were treated with UDCA at 13–15 mg/kg per day and followed up for at least 2 years after diagnosis. Bezafibrate (400 mg/day) was administered in addition to UDCA when biochemical response to UDCA was not optimal. Response to bezafibrate treatment was determined by serum alanine transaminase (ALT) levels within 2–3 years or at the earliest point thereafter from the commencement of bezafibrate treatment. Results We enrolled 1,121 PBC patients, and the observational period was 6.1 ± 3.4 years. Among the PBC patients, 835 were asymptomatic, defined as no liver-related symptoms at the baseline. In asymptomatic PBC patients, multivariate analysis indicated that ALT response to bezafibrate treatment was significantly associated with the presence of liver-related symptoms at the end of observation [hazard ratio 1.46 (95 % confidence interval 1.01–2.13), P = 0.048]. The cumulative liver-related-symptoms-free rate of patients treated with bezafibrate and who had a normal ALT level was significantly higher than that of those treated with bezafibrate and who had an elevated ALT level ( P