A Time-Dependent Prognostic Score (Helsinki Primary Sclerosing Cholangitis Score) to Predict Disease Outcomes in Primary Sclerosing Cholangitis.

BACKGROUND & AIMS: Primary sclerosing cholangitis is a chronic, progressive liver disease with a highly variable course. Improved tools are urgently needed to identify patients with primary sclerosing cholangitis at risk of rapid progression to end-stage liver disease or cholangiocarcinoma. This study aimed to develop an easy-to-use, disease-specific, time-dependent prognostic score (HelPSCore) incorporating a composite endpoint of hepatobiliary malignancy, transplantation, and liver-related mortality.

METHODS

Multicenter prospective study consisting of derivation and validation cohorts. The derivation cohort included 1016 consecutive, prospectively collected patients with primary sclerosing cholangitis from Helsinki University Hospital, Finland. The time-weighted area under the curve for laboratory values was calculated, with variables selected based on predictive performance using Liu's optimal cutoff point estimation. The validation cohort (n = 467) included patients from Sweden (n = 415) and the Netherlands (n = 52). HelPSCore was developed using variables demonstrating highest area under the curve for the composite endpoint and the score coefficients were derived based on cumulative incidence estimates of the composite endpoint obtained via the Cox regression.

RESULTS

The hazard ratios for the endpoint increased progressively and demonstrated robust predictive accuracy over 25 years. The overall correct classification rate was 86%. The area under the curve for the derivation and validation cohorts was 0.82 and 0.83, respectively, for predicting the composite endpoint. Compared to the Amsterdam-Oxford Model (0.61), UK-PSC risk score (0.61-0.72), Mayo Risk Score (0.69), and Model for End-stage Liver Disease score (0.61), HelPSCore showed improved predictive ability in both the short- and long-term.

CONCLUSIONS

HelPSCore provides a straightforward and reliable tool for short- and long-term primary sclerosing cholangitis prognosis. This enables the identification of high-risk patients who may benefit from intensified monitoring and tailored therapy.