Primary sclerosing cholangitis in children

A long-term follow-up study

Ariel E. Feldstein, Jean Perrault, Mounif El-Youssif, Keith Lindor, Deborah K. Freese, Paul Angulo

Research output: Contribution to journalArticle

164 Citations (Scopus)

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 γ-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.

Original languageEnglish (US)
Pages (from-to)210-217
Number of pages8
JournalHepatology
Volume38
Issue number1
DOIs
StatePublished - Jul 1 2003
Externally publishedYes

Fingerprint

Sclerosing Cholangitis
Survival
Autoimmune Hepatitis
Liver Transplantation
Ursodeoxycholic Acid
Cholangiography
gamma-Glutamyltransferase
Splenomegaly
Immunosuppressive Agents
Platelet Count
Inflammatory Bowel Diseases
Proportional Hazards Models
Population
Signs and Symptoms
Alkaline Phosphatase
Longitudinal Studies
Reference Values
Cohort Studies
Therapeutics
Age Groups

ASJC Scopus subject areas

  • Hepatology

Cite this

Feldstein, A. E., Perrault, J., El-Youssif, M., Lindor, K., Freese, D. K., & Angulo, P. (2003). Primary sclerosing cholangitis in children: A long-term follow-up study. Hepatology, 38(1), 210-217. https://doi.org/10.1053/jhep.2003.50289

Primary sclerosing cholangitis in children : A long-term follow-up study. / Feldstein, Ariel E.; Perrault, Jean; El-Youssif, Mounif; Lindor, Keith; Freese, Deborah K.; Angulo, Paul.

In: Hepatology, Vol. 38, No. 1, 01.07.2003, p. 210-217.

Research output: Contribution to journalArticle

Feldstein, AE, Perrault, J, El-Youssif, M, Lindor, K, Freese, DK & Angulo, P 2003, 'Primary sclerosing cholangitis in children: A long-term follow-up study', Hepatology, vol. 38, no. 1, pp. 210-217. https://doi.org/10.1053/jhep.2003.50289
Feldstein AE, Perrault J, El-Youssif M, Lindor K, Freese DK, Angulo P. Primary sclerosing cholangitis in children: A long-term follow-up study. Hepatology. 2003 Jul 1;38(1):210-217. https://doi.org/10.1053/jhep.2003.50289
Feldstein, Ariel E. ; Perrault, Jean ; El-Youssif, Mounif ; Lindor, Keith ; Freese, Deborah K. ; Angulo, Paul. / Primary sclerosing cholangitis in children : A long-term follow-up study. In: Hepatology. 2003 ; Vol. 38, No. 1. pp. 210-217.
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