Bezafibrate for primary biliary cholangitis: A number needed to treat analysis

Area of Science:

• Hepatology
• Clinical Pharmacology
• Biostatistics

Background:

• Primary biliary cholangitis (PBC) is a chronic liver disease.
• Ursodeoxycholic acid (UDCA) is a standard treatment for PBC.
• The clinical benefit of bezafibrate (BZF) in PBC requires further quantification.

Purpose of the Study:

• To assess the number needed to treat (NNT) with BZF to prevent liver transplantation (LT) or death in PBC patients.
• To evaluate BZF's efficacy based on alkaline phosphatase (ALP) levels after UDCA treatment.

Main Methods:

• NNT calculated using LT-free survival data from the Dutch PBC Cohort Study.
• Hazard ratios for BZF from the Japanese Cohort (Tanaka et al.) were applied.
• NNT estimated for 5-year (NNT5y) and 10-year (NNT10y) timeframes.

Main Results:

• Overall NNT5y was 15 and NNT10y was 7 to prevent one LT or death.
• For patients with ALP 1.0-1.5xULN at 1 year, NNT5y was 24 and NNT10y was 10.
• For patients with ALP ≥3.0xULN, NNT5y was 7 and NNT10y was 4.

Conclusions:

• Bezafibrate demonstrates strong projected efficacy in preventing major clinical endpoints in PBC.
• BZF offers significant clinical benefit even in patients with a favorable ALP response (1.0-1.5xULN) to UDCA.
• Off-label use of BZF may be beneficial for UDCA-treated PBC patients who do not achieve ALP normalization.