Natural history of primary sclerosing cholangitis in childhood

Background & Aims Primary sclerosing cholangitis (PSC) is a rare progressive liver disease associated with inflammatory bowel disease (IBD), usually diagnosed in adults but can also present in children. Data on long-term outcome in pediatric PSC is limited. Our aim was to study the natural history of pediatric PSC in Sweden. Methods: This is a cohort study, including all children (<18 years), diagnosed with PSC between January 2000 and December 2015 at the Pediatric Liver Unit at Karolinska University Hospital, Stockholm. Patients were followed until liver transplantation, death or last date of follow-up (August 2021). Results We identified 124 children with a median age of 14 (1.9–17.8) years at PSC diagnosis. Sixty percent were boys, 93% had IBD. Median follow-up time was 13 years (5.7–21.6). Overall event-free survival in the cohort was 91% (95% CI 0.84–0.95) at 5 years and 77% (95% CI 0.68–0.84) at 10 years after diagnosis. Autoimmune hepatitis (AIH) was present in 31% (n=39). Portal hypertension (PHT) developed in 13% (n=16), biliary complications in 24% (n=30), cholangiocarcinoma (CCA) in 0.8% (n=1), 13% (n=16) were liver transplanted and three patients died. Transplant-free survival was 91% after 10 years. Individuals with a high SCOPE index at diagnosis had 2.3 timed increased risk of liver transplantation, HR 2.35 (95% CI 1.18–4.66), c-statistics=0.70. Patients with an additional AIH diagnosis had slightly higher risk of reaching transplantation during follow-up, HR 2.85 (95% CI 1.06–7.67), p=0.038. Conclusions Children diagnosed with PSC have a good prognosis during the first decade after diagnosis. A high SCOPE index at diagnosis was associated with a less favorable outcome.