https://pubmed.ncbi.nlm.nih.gov/32464706/

Hepatology. 2020 May 28.
doi: 10.1002/hep.31393. Online ahead of print.
The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children

Abstract

Background & aims: Disease progression in children with primary sclerosing cholangitis (PSC) is variable. Prognostic and risk-stratification tools exist for adult-onset PSC, but not for children. We aimed to create a tool that accounts for the biochemical and phenotypic features and early disease stage of pediatric PSC.

Approach & results: We used retrospective data from the Pediatric PSC Consortium. The training cohort contained 1012 patients from 40 centers. We generated a multivariate risk index (SCOPE index) that contained total bilirubin, albumin, platelet count, gamma glutamyltransferase and cholangiography to predict a primary outcome of liver transplantation or death (TD), and a broader secondary outcome that included portal hypertensive, biliary and cancer complications termed hepatobiliary complications (HBC). The model stratified patients as low, medium or high risk based on progression to TD at rates of <1, 3, and 9% annually, and to HBC at rates of 2, 6, and 13% annually, respectively (p<0.001). C-statistics to discriminate outcomes at 1 and 5 years was 0.95 and 0.82 for TD, and 0.80 and 0.76 for HBC, respectively. Baseline hepatic fibrosis stage was worse with increasing risk score, with extensive fibrosis in 8% of the lowest vs. 100% with the highest risk index, p<0.001. The model was validated in 240 children from 11 additional centers and performed well.

Conclusions: The SCOPE index is the first pediatric-specific prognostic tool for PSC. It uses routinely-obtained, objective data to predict a complicated clinical course. It correlates strongly with biopsy-proven liver fibrosis. SCOPE can be used with families for shared decision making on clinical care based on a patient's individual risk, and to account for variable disease progression when designing future clinical trials.