4CPS-199 Bezafibrate for primary biliary cholangitis: efficacy, safety, and efficiency of an off-label use protocol in real-world practice

<h3>Background and Importance</h3> Primary biliary cholangitis (PBC) is an autoimmune disease affecting bile ducts. Ursodeoxycholic acid (UDCA) is first-line therapy, but around 40% of patients do not respond. Obeticholic acid (OCA), approved as second-line therapy, is under review. Fibrates, used off-label, have shown potential as an alternative. <h3>Aim and Objectives</h3> This study evaluates the effectiveness and safety of bezafibrate as a second-line treatment for PBC. Additionally, it assesses the economic impact of protocolising fibrate use. <h3>Material and Methods</h3> An observational, retrospective study was conducted in a tertiary hospital by a multidisciplinary team of pharmacists and hepatologists. Patients diagnosed with PBC who did not respond to UDCA and receiving treatment with bezafibrate as a second-line therapy were included. Variables collected included alkaline phosphatase (ALP), alanine aminotransferase (ALT), total bilirubin, liver stiffness and steatosis using FibroScan, adverse effects, and treatment discontinuations. Data were collected pre-treatment, at 12 months, and at the last follow-up until June 2024. Effectiveness was assessed by comparing biochemical variables before and after treatment, as well as changes in FibroScan values. Safety was evaluated based on adverse effects and treatment discontinuations. Wilcoxon paired tests were used to compare values before and after treatment, and 95% confidence intervals were calculated for percentages (STATA17). Patients meeting POISE study inclusion criteria, therefore being candidates to treament with OCA, were included for a cost-minimisation analysis. It compared OCA (5–10 mg) costs with bezafibrate (400 mg). <h3>Results</h3> In 57 patients, at 12 months, ALP decreased by 81 IU/L, ALT by 14 IU/L, and total bilirubin by 0.12 mg/dL (all p &lt; 0.05). 63% of patients achieved normal ALP levels at the last follow-up. No significant changes were observed in FibroScan results. Adverse effects were reported in 11% of patients, with 7% discontinuing. The cost-minimisation analysis included 23 patients. The annual cost of bezafibrate was €67, compared to €27,507 for OCA (difference in annual cost resulted in €27,440 per patient/year and total savings €631,138 during the first year of follow-up). <h3>Conclusion and Relevance</h3> Bezafibrate is an effective and safe second-line therapy for PBC, achieving significant biochemical improvements and maintaining disease control. The cost-minimisation analysis highlights substantial economic savings when bezafibrate is protocolised, supporting its integration into clinical practice. <h3>References and/or Acknowledgements</h3> https://www.nejm.org/doi/10.1056/NEJMoa1714519?url_ver=Z39.88-2003&amp;rfr_id=ori:rid:crossref.org&amp;rfr_dat=cr_pub%20%200www.ncbi.nlm.nih.gov https://www.nejm.org/doi/10.1056/NEJMoa1509840?url_ver=Z39.88-2003&amp;rfr_id=ori:rid:crossref.org&amp;rfr_dat=cr_pub%20%200www.ncbi.nlm.nih.gov <h3>Conflict of Interest</h3> No conflict of interest