Background: Primary Sclerosing Cholangitis (PSC) is a rare progressive liver disease

associated with inflammatory bowel disease (IBD). There is a high risk for

development of cirrhosis and need of liver transplantation, cholangiocarcinoma

(CCA) and colorectal cancer. PSC is usually diagnosed in adults but can also present

in children and data on long-term outcome in pediatric PSC is scarce. Our aim was to

study initial presentation of pediatric PSC, describe long-term outcome related to

disease phenotype and to validate the prognostic tool Sclerosing Cholangitis

Primary Sclerosing Cholangitis in childhood – initial presentation, natural

history and long-term outcome

Outcomes in Pediatrics (SCOPE) index. Methods: This is a cohort study including all

consecutive children (<18 years), diagnosed with PSC between Jan 2000 – Dec

2015 at the Paediatric Liver Unit, a tertiary referral center, at Karolinska University

Hospital, Stockholm. Clinical, radiological, histological, and biochemical data were

collected. Patients were followed from diagnosis until death or last date of follow-up

(31st August 2021). Transplant-free survival was calculated with the Kaplan-Meiermethod.

The SCOPE index was calculated at diagnosis, dividing patients into low,

medium or high risk for adverse liver events within 5 years. Results: We included 124

patients with a median age of 13 (5.3-17.8) at diagnosis. Median follow-up time was

13 years (5.7-21.6). The majority (60%) were boys, IBD was present in 93%,

ulcerative colitis (UC) in 55% (n=63), Crohn´s disease (CD) in 37% (n=42) and IBD-U

in 8% (n=9). Overlap with autoimmune hepatitis (AIH) was present in 37% (n=35).

SCOPE index was low in 45% (n=47), medium in 38% (n=39) and high in 17%

(n=18) at diagnosis. During follow-up portal hypertension developed in 16% (n=20),

biliary complications in 26% (n=30). One patient developed CCA 15 years after

diagnosis and three patients died during follow-up, one liver related. Twelve percent

(n=15) were liver transplanted. Transplant-free survival was 91% after 10 years.

Conclusions: The prognosis of PSC in children is good with a transplant-free survival

of over 90 % after 10 years. A high SCOPE index at diagnosis was associated with a

less favorable outcome.