Two risk stratification models, GLOBE and UK-PBC, performed well in predicting future complications related to cirrhosis in patients with primary biliary cholangitis (PBC), according to study results published in The American Journal of Gastroenterology.
Several response criteria for ursodeoxycholic acid (UDCA), including the Paris I, Paris II, Barcelona, and Toronto criteria, were developed from single-center cohorts. Newer models from the Global PBC Study Group (GLOBE) and the UK-PBC Research Group (UK-PBC) were created from cohorts with larger patient populations and use combinations of UDCA response and features that indicate liver disease stage.
This international, multicenter study included data from 1746 individuals with PBC who received treatment with UDCA between 1997 and 2017. Treatment took place in 25 facilities in Canada, Europe, and the United States. Inclusion criteria were treatment with UDCA for more than a year following diagnosis of PBC, as well as data to assess risk and biochemical response criteria. The primary outcome was development of decompensated cirrhosis during treatment. The GLOBE and UK-PBC risk scoring systems were assessed for performance, as were UDCA response criteria.
Among the participants, 171 experienced a clinically significant adverse outcome: 59% (n=101) experienced ascites, 23% (n=40) variceal bleeding, 9% (n=15) hepatic encephalopathy, and 9% (n=15) a combination of complications. At 5, 10, and 15 years, event-free survival rates were 95%, 85%, and 77%, respectively.
According to the age-specific GLOBE score thresholds, 247 patients were at high risk for adverse outcomes. Of this group, 137 developed an adverse event, resulting in a sensitivity of 93% and a specificity of 83% in the GLOBE model. Compared with UDCA response criteria, the GLOBE and UK-PBC models performed better at predicting complications related to cirrhosis. With each standard deviation increase in score, the hazard ratio for the UK-PBC model was 3.39 (95% CI, 3.10-3.72; P <.001) and 5.05 (95% CI, 4.43-5.74; P <.001) for the GLOBE model. Both models performed well, with a C-statistic of 0.93 (95% CI, 0.91-0.95) in the GLOBE and 0.94 (95% CI, 0.91-0.96) in the UK-PBC model.
Study researchers concluded that “risk-predicting models (GLOBE and UK-PBC) are superior to UDCA response criteria for predicting disease outcome in PBC. Both the GLOBE and the UK-PBC risk scores had excellent performance for predicting adverse events in our study population. Future prospective and multicenter studies may further validate and explore the prognostic relevance of the GLOBE and the UK-PBC risk scoring systems.”
Efe C, Taşçilar K, Henriksson I, et al. Validation of risk scoring systems in ursodeoxycholic acid-treated patients with primary biliary cholangitis. Am J Gastroenterol. 2019;114(7):1101-1108.