Experience of obeticholic acid use in the treatment of primary biliary cholangitis in a small group of patients

• Autores: Tania González Furelos, Iria Rodriguez Legazpi, Antonia Casás Martínez, Ignacio López de Ullibarri, Isaura Rodríguez Penín
• Localización: European journal of clinical pharmacy: atención farmacéutica, ISSN 2385-409X, Vol. 23, Nº. 4, 2021, págs. 239-243
• Idioma: inglés
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• Resumen

  • Objectives: Primary biliary cholangitis (PBC) is a rare, chronic, and severe autoimmune disease whose initial treatment is ursodeoxycholic acid (UDCA). The combination of UDCA with obeticholic acid (OCA) is a new therapeutic option for unresponsive patients to UDCA. The primary objective of this study was to assess the efficacy and tolerability of OCA in patients diagnosed with PBC in a health district with a population of 190,000.

    Method: An observational retrospective study was performed between January 2017 and March 2020. The assessment of efficacy and tolerability of OCA was performed based on demographic data, diagnosis, disease markers and adverse events. Descriptive summaries of quantitative data are expressed as means and ranges. An analysis of the longitudinal data on alkaline phosphatase (AP) and gamma-glutamyltranspeptidase (GGT) levels since the start of OCA treatment was addressed by linear mixed-effects modeling. The cost of treatment (€ patient per year) was also calculated.

    Results: A total of six patients with a mean age of 58.2 years (27-75 years) were included. The linear mixed-effects model fitted evidenced a significant effect of time under OCA treatment on the reduction of log AP and GGT levels. A 12.5% and 10.3% decrease of the mean of log AP and GGT levels, respectively, are estimated after one year since the start of treatment with OCA. The most frequent adverse event was pruritus, developed by 100% of patients. The direct cost of the therapy was €27,174/patient/year. A limitation of the study was the small sample size.

    Conclusions: In our population, OCA has demonstrated to be effective reducing mean values of AP and GGT, emerging as an alternative treatment with added therapeutic value