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Good long-term outcomes of primary sclerosing cholangitis in childhood
Anna Jerregård Skarby1, Thomas Casswall2, Annika Bergquist3
1Department of Medicine Huddinge, Karolinska Institutet, Department of Acute Geriatrics, Stroke and Palliative care, Nyköping Hospital Nyköping, Sweden.
Insights
Pediatric primary sclerosing cholangitis (PSC) shows good long-term survival, with 77% event-free survival at 10 years. A high SCOPE index at diagnosis indicates a higher risk for liver transplantation in children with PSC.
Area of Science:
- Hepatology
- Pediatric Gastroenterology
- Autoimmune Diseases
Background:
- Primary sclerosing cholangitis (PSC) is a rare, progressive liver disease often linked to inflammatory bowel disease (IBD).
- While typically diagnosed in adults, PSC also affects children, but long-term outcome data for pediatric cases are scarce.
- Understanding the natural history of pediatric PSC is crucial for patient management and future planning.
Purpose of the Study:
- To investigate the natural history of pediatric primary sclerosing cholangitis (PSC) in Sweden.
- To analyze long-term outcomes, including survival rates and complications, in children diagnosed with PSC.
- To identify factors influencing the prognosis of pediatric PSC.
Main Methods:
- A retrospective cohort study was conducted involving all children (<18 years) diagnosed with PSC between 2000 and 2015.
- Patients were followed until liver transplantation, death, or August 2021.
- Data analysis included event-free survival, transplant-free survival, and risk factor assessment using the SCOPE index and co-existing autoimmune hepatitis (AIH).
Main Results:
- 124 children with PSC were identified, with a median age of 14 years at diagnosis; 93% had IBD.
- Event-free survival was 91% at 5 years and 77% at 10 years. Transplant-free survival was 91% after 10 years.
- A high SCOPE index increased the risk of liver transplantation (HR 2.35), as did co-existing AIH (HR 2.85).
Conclusions:
- Pediatric PSC demonstrates a favorable prognosis within the first decade post-diagnosis.
- The SCOPE index at diagnosis is a significant predictor of a less favorable outcome, including the need for liver transplantation.
- These findings provide valuable insights for counseling healthcare professionals, young patients, and families affected by pediatric PSC.
Background & Aims:
Primary sclerosing cholangitis (PSC) is a rare progressive liver disease associated with inflammatory bowel disease (IBD). It is usually diagnosed in adults but can also present in children. Data on long-term outcomes of pediatric PSC are 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 developed in 13% (n = 16), biliary complications in 24% (n = 30), cholangiocarcinoma (CCA) in 0.8% (n = 1), while 13% (n = 16) underwent liver transplantation and three patients died. Transplant-free survival was 91% after 10 years. Individuals with a high SCOPE index at diagnosis had a 2.3-fold increased risk of requiring liver transplantation (hazard ratio 2.35, 95% CI 1.18-4.66, c-statistics = 0.70). Patients with an additional diagnosis of autoimmune hepatitis had slightly higher risk of reaching transplantation during follow-up (hazard ratio 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.
Impact And Implications:
Data on long-term outcome in pediatric primary sclerosing cholangitis bridging over to adulthood is limited. There is a great need among children with primary sclerosing cholangitis and their parents for more knowledge about the natural history of this disease and what they can expect from the future. We hope that the data presented in this study may help counsel health professionals, young individuals and families affected by this disease.
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