Analysis of predictive response scores to treatment with ursodeoxycholic acid in patients with primary biliary cholangitis

Abstract

Background and objectives: Primary biliary cholangitis (PBC) is an autoimmune disease that affects the small bile ducts. The only treatments currently approved in our country are ursodeoxycholic acid (UDCA) and obeticholic acid. Different indices evaluate the response after one year of treatment. The aim of our study was to evaluate the different predictive scores and prognostic factors of response to UDCA.

Material and methods: Retrospective single-centre study in which clinical and analytical data of patients diagnosed with PBC were collected from January 1987 to December 2015. The response after one year of treatment was evaluated using the different response scores and their concordance degree using the Kappa index. The area under the receiver operating characteristic curve (AUROC) was calculated to determine the predictive capacity of the scores. Likewise, the prognostic factors of response to treatment were analysed.

Results: We included 153 patients. The bivariate analysis showed a statistically significant relationship between the initial high levels of alkaline phosphatase and cholesterol and the poor response to treatment. The best AUROC was in Paris-I score (0.81). The concordance between the different scores was low. The GLOBE score was valid to evaluate the prognosis.

Conclusion: Basal alkaline phosphatase and cholesterol were predictors of poor outcome. The best predictive qualitative score in our cohort patients was Paris-I. There was a poor concordance between the different predictive scores. GLOBE score is valid to evaluate prognosis.

Keywords: Colangitis biliar primaria; Factores pronósticos; Historia natural; Natural history; Predictive scores; Primary biliary cholangitis; Prognostic factors; Ursodeoxycholic acid; Ácido ursodeoxicólico; Índices predictivos.