Validation of Risk Scoring Systems in Ursodeoxycholic Acid–Treated Patients With Primary Biliary Cholangitis

Abstract

INTRODUCTION: Risk stratification based on biochemical variables is a useful tool for monitoring ursodeoxycholic acid (UDCA)-treated patients with primary biliary cholangitis (PBC). Several UDCA response criteria and scoring systems have been proposed for risk prediction in PBC, but these have not been validated in large external cohorts. METHODS: We performed a study on data of 1746 UDCA-treated patients with PBC from 25 centers in Europe, United States, and Canada. The prognostic performance of the risk scoring systems (GLOBE and UK-PBC) and the UDCA response criteria (Barcelona, Paris I, Paris II, Rotterdam, and Toronto) were evaluated. We regarded cirrhosis-related complications (ascites, variceal bleeding, and/or hepatic encephalopathy) as clinical end points. RESULTS: A total of 171 patients reached a clinical end point during a median 7 years (range 1–16 years) of follow-up. The 5-, 10- and 15-year adverse outcome–free survivals were 95%, 85%, and 77%. The GLOBE and UK-PBC scores predicted cirrhosis-related complications better than the UDCA response criteria. The hazard ratio (HR) for a 1 standard deviation increase was HR 5.05 (95% confidence interval (CI): 4.43–5.74, P < 0.001) for the GLOBE score and HR 3.39 (95% CI: 3.10–3.72, P < 0.001) for the UK-PBC score. Overall, the GLOBE and UK-PBC risk scores showed similar and excellent prognostic performance (C-statistic, 0.93; 95% CI: 0.91%–95% vs 0.94; 95% CI: 0.91%–0.96%). DISCUSSION: In our international, multicenter PBC cohort, the GLOBE and UK-PBC risk scoring systems were good predictors of future cirrhosis-related complications.

The association of histological progression with biochemical response to ursodeoxycholic acid in primary biliary cholangitis

“…Several studies have validated the ability of Globe and UK-PBC risk scores to predict clinical outcomes in patients with PBC. [41][42][43] Absolute alkaline phosphatase and total bilirubin criteria at 1 year after the UDCA therapy have been used as a more robust predictor of hard endpoints such as liver transplantation or deaths of patients with PBC. 35 More recently, Zhang et al reported that earlier biochemical response to UDCA at 3 or 6 months was devised to identify patients at risk of poor outcomes.…”

Clinical Updates in Primary Biliary Cholangitis: Trends, Epidemiology, Diagnostics, and New Therapeutic Approaches

“…50,69,70 Both the GLOBE and UK-PBC risk scoring systems were found to be predictive of cirrhosis-related complications based on a recent multicenter Turkish study. 71 Table 2 provides a review of current prognostication models.…”

A nomogram based on pretreatment clinical parameters for the prediction of inadequate biochemical response in primary biliary cholangitis

“…Moreover, there are no clear criteria that can reliably predict the response to UDCA. 15 Therefore, there were limitations to drawing conclusions from a single biochemical response criterion, which may lead to the misclassification of the clinical effect of UDCA in PBC patients. In this study, the Paris I criteria combined with the Barcelona criteria were defined as the study endpoints.…”

“…6,[12][13][14] There are significant differences in the criteria for evaluating the UDCA response in PBC patients. 15 Given the potential risk of misidentification, the comprehensive evaluation of biochemical response can be superior to the use of a single criterion and could be helpful in risk stratification. In this study, we sought to retrospectively collect pretreatment clinical parameters, with the aim of identifying potential nonresponders to UDCA (defined by combined biochemical response criteria), using these clinical parameters to develop and validate a nomogram to facilitate decision-making.…”