Related Experiment Videos
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.
Hepatology (Baltimore, Md.)
|June 28, 2003
Summary
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.