Primary sclerosing cholangitis in children: a single-center experience and evaluation of prognostic markers

Pediatric primary sclerosing cholangitis (PSC) is a rare, chronic liver disease often associated with unfavorable outcomes. However, reliable prognostic markers in children remain limited. This observational cohort study aimed to identify clinical and laboratory markers associated with a severe liver disease course and to characterize the clinical progression of pediatric PSC.

We conducted a retrospective analysis of children with PSC treated at our tertiary transplant center between January 2011 and June 2023. The study evaluated laboratory markers [gamma-glutamyl transferase (GGT), total bilirubin, the aspartate aminotransferase (AST)-to-platelet ratio index (APRI), and the Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) index] at diagnosis, as well as GGT normalization within 1 year. In addition, we assessed clinical factors including sex, age at diagnosis, PSC subtype, presence of inflammatory bowel disease (IBD), and features of autoimmune hepatitis (AIH), in relation to the occurrence of severe complications. Severe complications were defined as biliary or portal complications, cholangiocarcinoma, liver transplant listing, or liver-related death.

Sixty patients with PSC (40.0% female) were followed for a median of 3.8 years [interquartile range (IQR), 1.5-8.7 years]. IBD was present in 81.7% of cases, and 43.3% had features of AIH. The median age at diagnosis was 12 years (IQR, 9-15 years). Severe complications occurred in 17% of patients, including biliary complications (6.7%), portal complications (8.3%), cholangiocarcinoma (1.7%), and liver transplant listing (6.7%). No liver-related deaths were recorded. Event-free survival was 88.3% at 5 years and 71.5% at 10 years. No statistically significant differences in outcomes were observed based on the SCOPE index calculated at time of diagnosis or GGT normalization within 1 year. Similarly, elevated total bilirubin, GGT, and APRI index at diagnosis were not associated with worse outcomes.

Neither the SCOPE index at diagnosis nor GGT normalization within 1 year predicted severe liver disease course. No clinical factors (sex, age at diagnosis, presence of IBD, features of AIH, or PSC subtype) or laboratory markers were found to reliably predict a severe disease course. Overall, the long-term prognosis in this pediatric cohort was generally favorable.