Real-world results of the use of obeticholic acid in the treatment of primary biliary cholangitis

Introduction: Primary biliary cholangitis (PBC) is a chronic and progressive auto immune disease. Patients with PBC have been treated for a long time only with ursodeoxycholic acid (UDCA), though not all patients responded or tolerated it. Nowadays, a secondline drug has been approved for the treatment of patients with PBC who fail to first-line UDCA therapy, showing improvements on liver biochemistry. Thus, we want to describe our experience in realworld clinical practice. The main aim of the treatment is to increase transplantfree survival, while the secondary objectives are to slow down the liver disease progression and to diminish PBCassociated symptoms. Method: Prospective study which evaluates the efficacy and safety of obeticholic acid (OCA) in a realworld subset of patients. The primary aim was to assess changes in alkaline phosphatase (ALP). The secondary objectives were to iden tify changes in alanine aminotransferase (ALT), aspartate aminotransferase (AST), gammaglutamyl transpeptidase (GGT), albumin and bilirubin, the reason for start ing the treatment, the dose of obeticholic acid, drug interactions, the adverse effects detected and the budgetary impact at our hospital. Result: We enrolled 14 patients, 85.7% women with a mean age of 54 ±8 years. 28.6% of patients had liver fibrosis stage 0-1. The mean duration of obeticholic acid therapy was 14 ±5 months, with >90% adherence. All patients maintained dual therapy with UDCA. 42.9% of patients received 5 mg/day of obeticholic acid, and in 57.1% the dose was titrated to 10 mg/day after six months of treat ment due to lack of response. All patients reduced their ALP values and 92.9% reduced their ALT. The mean percentages of reduction were 34% for ALP, 36% for ALT, 27% for AST, 2% for albumin and 44% for GGT. After starting treat ment, pruritus was worsened in three patients, interrupting the therapy in only one case. The cost of obeticholic acid treatment for PBC is €26,449/patient/year, and the treatment of these patients involved a cost of €264,490, which was the 3.1% of the total budget of the Gastrointestinal Department in 2019. Conclusion: Obeticholic acid is a drug with a high budgetary impact that has shown similar effectiveness in the real world to that achieved in clinical trials, improving ALP and ALT levels in primary biliary cholangitis patients