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Updated: Jun 26, 2026
Isolation of Neonatal Extrahepatic Cholangiocytes
Published on: June 5, 2014
A retrospective single-center review of primary sclerosing cholangitis in children
Tamir Miloh1, Ronen Arnon, Benjamin Shneider
1Department of Pediatrics, and Recanati/Miller Transplant Institute, Mount Sinai Hospital, New York, New York, USA.
Insights
Pediatric primary sclerosing cholangitis (PSC) often presents with inflammatory bowel disease or autoimmune overlap and advanced fibrosis. Ursodeoxycholic acid (UDCA) therapy improved liver enzymes, even in patients with autoimmune markers.
Area of Science:
- Pediatric Gastroenterology
- Hepatology
- Autoimmune Liver Diseases
Background:
- Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease with limited pediatric data.
- Characterized by bile duct inflammation and fibrosis.
Purpose of the Study:
- To characterize pediatric PSC patients.
- Evaluate disease presentation, comorbidities, and treatment outcomes.
Main Methods:
- Retrospective chart review of 47 pediatric PSC patients.
- Analysis of clinical presentation, imaging (MRCP), colonoscopy, and treatment response.
Main Results:
- Mean age at diagnosis: 11 years.
- 81% had symptoms prior to presentation; 59% had inflammatory bowel disease (IBD), 25% had autoimmune hepatitis overlap.
- 65% had advanced fibrosis; UDCA improved liver enzymes, including in those with autoimmune markers.
Conclusions:
- Pediatric PSC frequently involves IBD or autoimmune overlap and advanced fibrosis at diagnosis.
- UDCA is effective in normalizing liver enzymes, even in cases with positive autoimmune markers.
- Small-duct PSC showed lower liver enzyme levels.
Background & Aims:
Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease characterized by inflammation and progressive bile duct fibrosis. There are limited data on pediatric PSC.
Methods:
We performed a retrospective chart review of 47 pediatric patients with PSC.
Results:
The mean age at diagnosis was 11 +/- 4.9 years. Symptoms occurred before presentation in 81% of patients; inflammatory bowel disease was found in 59% and autoimmune hepatitis (overlap syndrome) in 25% of patients. Magnetic resonance cholangiography revealed both extrahepatic and intrahepatic, isolated intrahepatic, isolated extrahepatic, and no biliary involvement (small-duct PSC) in 40%, 14%, 10%, and 36%, respectively. Advanced fibrosis (stage >II) was present in 65%. Colonoscopy revealed pancolitis, rectal sparing, and normal findings in 24%, 24%, and 18%, respectively. All patients were treated with ursodeoxycholic acid (UDCA); 9 with overlap syndrome also received immunosuppressants. Fifteen patients without overlap syndrome had positive autoimmune markers and responded to UDCA monotherapy. Liver transplantation was performed in 9 patients (3 with overlap syndrome and 2 with small-duct PSC) at a median time of 7 years after diagnosis. The 10-year posttransplant survival rate was 89%.
Conclusions:
In one of the largest single-center studies of children with PSC, we found that most children with PSC had inflammatory bowel disease or autoimmune overlap and advanced fibrosis at diagnosis. Levels of alanine aminotransferase and gamma-glutamyl transferase were highest in patients with overlap syndrome and lowest in those with small-duct PSC. Levels of serum liver enzymes normalized after therapy with UDCA, including patients with positive autoimmune markers without histologic features of autoimmune hepatitis.