A retrospective single-center review of primary sclerosing cholangitis in children.
OBJECTIVE
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.
Miloh
Tamir
T
Department of Pediatrics, and Recanati/Miller Transplant Institute, Mount Sinai Hospital, New York, New York, USA.
Arnon
Ronen
R
Shneider
Benjamin
B
Suchy
Frederick
F
Kerkar
Nanda
N
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D002756:Cholagogues and Choleretics / Q000627:therapeutic use; D002758:Cholangiography; D015209:Cholangitis, Sclerosing / Q000150:complications / Q000175:diagnosis* / Q000473:pathology* / Q000628:therapy; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D016031:Liver Transplantation; D008297:Male; D012189:Retrospective Studies; D016896:Treatment Outcome; D014580:Ursodeoxycholic Acid / Q000627:therapeutic use; D055815:Young Adult
D002756:Cholagogues and Choleretics; D007166:Immunosuppressive Agents; D014580:Ursodeoxycholic Acid