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Primary sclerosing cholangitis in children: a long-term follow-up study
Ariel E Feldstein1, Jean Perrault, Mounif El-Youssif
1Division of Gastroenterology and Hepatology, Department of Pediatric and Adolescent Medicine, Mayo Medical School, Clinic and Foundation, Rochester, MN 55905, USA.
Insights
Primary sclerosing cholangitis (PSC) in children significantly reduces survival, with a median transplant-free survival of 12.7 years. Medical therapies offer only temporary symptom relief, not long-term outcome improvement.
Area of Science:
- Pediatric Gastroenterology
- Hepatology
- Autoimmune Diseases
Background:
- Primary sclerosing cholangitis (PSC) is increasingly diagnosed in pediatric populations.
- Long-term prognosis for pediatric PSC remains largely uncertain.
- Many children with PSC present with concomitant inflammatory bowel disease (IBD).
Purpose of the Study:
- To determine the long-term outcome and survival of children diagnosed with PSC.
- To identify factors associated with survival in pediatric PSC.
- To evaluate the impact of medical therapy on long-term outcomes.
Main Methods:
- Longitudinal cohort study of 52 children with cholangiography-proven PSC over a 20-year period.
- Follow-up duration of up to 16.7 years.
- Cox regression analysis to identify predictors of survival.
Main Results:
- Median survival free of liver transplantation was 12.7 years, significantly shorter than the general pediatric population.
- Lower platelet count, splenomegaly, and older age were associated with decreased survival.
- Medical therapies, including ursodeoxycholic acid and immunosuppressants, provided transient clinical/biochemical benefits but did not impact long-term survival.
Conclusions:
- Primary sclerosing cholangitis (PSC) significantly impairs long-term survival in children.
- Current pharmacologic therapies offer limited long-term benefit for pediatric PSC.
- Further research is needed to improve outcomes for children with PSC.
Abstract:
Primary sclerosing cholangitis (PSC) is increasingly diagnosed in children and adolescents, but its long-term prognosis remains uncertain. The aim of this longitudinal, cohort study was to determine the long-term outcome of children with PSC. Fifty-two children with cholangiography-proven PSC (34 boys and 18 girls; mean age 13.8 +/- 4.2 years; range, 1.5-19.6 years) who were seen at our institution over a 20-year period were followed-up for up to 16.7 years. Two thirds presented with symptoms and/or signs of PSC and 81% had concomitant inflammatory bowel disease (IBD). Twenty-five percent had total alkaline phosphatase activity within the normal range for the age group, but all of them had elevated gamma-glutamyl transpeptidase levels. Autoimmune hepatitis overlapping with PSC was present in 35% of children. A positive but transient clinical and/or biochemical response occurred under therapy with ursodeoxycholic acid, alone or in combination with immunosuppressive medications. During follow-up, 11 children underwent liver transplantation for end-stage PSC and 1 child died. The median (50%) survival free of liver transplantation was 12.7 years. Compared with an age- and gender-matched U.S. population, survival was significantly shorter in children with PSC (P <.001). In a Cox regression model, lower platelet count, splenomegaly, and older age were associated with shorter survival. Presence of autoimmune hepatitis overlapping with PSC (P =.2) or medical therapy (P =.2) did not affect survival. In conclusion, PSC significantly decreases survival in this child population. Although pharmacologic therapy may improve symptoms and liver test results initially, it does not seem to impact the long-term outcome.