Bezafibrate Shows Meaningful Long-Term Benefit in PBC : J Hepatol | June 2026

Introduction:

Ursodeoxycholic acid (UDCA) remains the first-line treatment for primary biliary cholangitis (PBC), yet a substantial proportion of patients fail to achieve complete biochemical response. Persistent elevation of alkaline phosphatase (ALP) after UDCA therapy is associated with increased risks of liver transplantation and liver-related mortality. Bezafibrate has emerged as an effective adjunctive therapy in patients with incomplete biochemical response, but its clinical benefit has often been reported using relative risk measures that can be difficult to interpret in routine practice.

Problem Statement:

While previous studies have demonstrated favorable effects of bezafibrate on biochemical markers and long-term outcomes, clinicians require more clinically meaningful measures to understand the magnitude of benefit. The number needed to treat (NNT) provides an intuitive assessment of how many patients need treatment to prevent major clinical events such as transplantation or death.

Summary:

This study evaluated the projected clinical benefit of bezafibrate in patients with PBC who continued to have elevated ALP levels despite UDCA therapy. Using data from a large national PBC cohort and previously validated treatment-effect estimates, the investigators calculated the number needed to treat to prevent liver transplantation or death. The analysis demonstrated a substantial long-term benefit of bezafibrate, with relatively few patients requiring treatment to prevent one major adverse liver outcome. Importantly, the benefit became more pronounced over longer follow-up periods and was greatest among patients with higher residual ALP levels, reflecting their increased baseline risk. However, clinically meaningful benefits were also observed in patients with only modest ALP elevation after one year of UDCA treatment, suggesting that the therapeutic value of bezafibrate extends beyond traditionally high-risk populations. These findings provide a practical perspective on the magnitude of benefit associated with bezafibrate and strengthen the rationale for its use as an adjunctive therapy in patients with incomplete biochemical response to UDCA. From a clinical standpoint, the results support earlier consideration of bezafibrate in appropriate patients and highlight its potential to reduce progression to liver transplantation and liver-related mo