External validation of the United Kingdom‐primary biliary cholangitis risk scores of patients with primary biliary cholangitis treated with ursodeoxycholic acid

Abstract

The United Kingdom‐Primary Biliary Cholangitis (UK‐PBC) risk scores are a set of prognostic models that estimate the risk of end‐stage liver disease in patients with PBC at 5‐, 10‐ and 15‐year intervals. They have not been externally validated outside the United Kingdom. In this retrospective, external validation study, data were abstracted from outpatient charts and discrimination and calibration of the UK‐PBC risk scores were assessed. A total of 464 patients with PBC treated with ursodeoxycholic acid were included. The median diagnosis age was 52.4 years, and 88% were female patients. The cumulative incidence of events was 6%, 9%, and 15% at 5, 10, and 15 years, respectively. Concordance (c‐statistic) was 0.88, 0.85, and 0.84 using the 5‐, 10‐ and 15‐year risk scores, respectively, which was slightly lower than values observed in the United Kingdom validation cohort. Using the 5‐year risk score, more events were observed than predicted (25 versus 16.8; P = 0.046); using the 10‐year risk score, there was no difference between the observed and predicted number of events (35 versus 44.9; P = 0.14); conversely, using the 15‐year risk score, fewer events were observed than predicted (46 versus 67.5; P = 0.009). Limiting evaluation by the 15‐year UK‐PBC risk score to those with >10 years of follow‐up demonstrated no difference between observed and predicted events. Using the 5‐year risk score, patients within the highest quartile had statistically significant worse event‐free survival compared to the rest of the cohort: 82% versus 98% at 5 years, 73% versus 97% at 10 years, and 58% versus 93% at 15 years. Conclusion: In patients assessed at a North American tertiary medical center, the UK‐PBC risk score had excellent discrimination and was reasonably calibrated both in the short and long term. (Hepatology Communications 2018;2:676‐682)

EASL Clinical Practice Guidelines on non-invasive tests for evaluation of liver disease severity and prognosis – 2021 update

“…209 Both scores have been externally validated and were superior to qualitative criteria, and to MELD and Child-Pugh scores. [210][211][212]213 Further studies are needed to better define the applicability of the UK-PBC risk score in routine clinical practice.…”

Clinical Outcomes and Validation of Ursodeoxycholic Acid Response Scores in Patients with Korean Primary Biliary Cholangitis: A Multicenter Cohort Study

“…GLOBE and UK-PBC scores were developed to predict the risk of liver transplantation or liver-related mortality of PBC. [11][12][13] However, these scores use laboratory parameters after 1 year of UDCA treatment.…”

Risk stratification in primary sclerosing cholangitis: It’s time to move on from replicating imperfection and break the glass ceiling

“…12 Nevertheless, in the related cholestatic disorder primary biliary cholangitis, prognostic models demonstrate both highlevel discrimination as well as calibration accuracy. [14][15][16][17] This difference may relate to the fact serum ALP exhibits wider intra-individual variability between time-points in PSC, which is likely to impact the performance of any ALP-based stratification system such as the AMS. 18 Notably, the current publication points toward significant limitations to serum ALP as a biomarker, as well as raising questions surrounding its utility as a surrogate endpoint in PSC clinical trials.…”