UNLABELLED: There are limited data on the natural history of primary sclerosing cholangitis (PSC) in children. We aimed to describe the disease characteristics and long-term outcomes of pediatric PSC. We retrospectively collected all pediatric PSC cases from 36 participating institutions and conducted a survival analysis from the date of PSC diagnosis to dates of diagnosis of portal hypertensive or biliary complications, cholangiocarcinoma, liver transplantation, or death. We analyzed patients grouped by disease phenotype and laboratory studies at diagnosis to identify objective predictors of long-term outcome. We identified 781 patients, median age 12 years, with 4,277 person-years of follow-up; 33% with autoimmune hepatitis, 76% with inflammatory bowel disease, and 13% with small duct PSC. Portal hypertensive and biliary complications developed in 38% and 25%, respectively, after 10 years of disease. Once these complications developed, median survival with native liver was 2.8 and 3.5 years, respectively. Cholangiocarcinoma occurred in 1%. Overall event-free survival was 70% at 5 years and 53% at 10 years. Patient groups with the most elevated total bilirubin, gamma-glutamyltransferase, and aspartate aminotransferase-to-platelet ratio index at diagnosis had the worst outcomes. In multivariate analysis PSC-inflammatory bowel disease and small duct phenotypes were associated with favorable prognosis (hazard ratios 0.6, 95% confidence interval 0.5-0.9, and 0.7, 95% confidence interval 0.5-0.96, respectively). Age, gender, and autoimmune hepatitis overlap did not impact long-term outcome. CONCLUSION: PSC has a chronic, progressive course in children, and nearly half of patients develop an adverse liver outcome after 10 years of disease; elevations in bilirubin, gamma-glutamyltransferase, and aspartate aminotransferase-to-platelet ratio index at diagnosis can identify patients at highest risk; small duct PSC and PSC-inflammatory bowel disease are more favorable disease phenotypes. (Hepatology 2017;66:518-527).

Academic Medical Centre Amsterdam The Netherlands

Alder Hey Children's Hospital Liverpool UK

Children's Health Memorial Institute Warsaw Poland

Children's National Medical Center Washington DC

Columbia University College of Physicians and Surgeons New York NY

Emory University School of Medicine Atlanta GA

Lille University Hospital of Lille Lille France

Mayo Clinic Rochester MN

Medical College of Wisconsin Milwaukee WI

Memorial University St John's Newfoundland and Labrador Canada

Nemours Alfred 1 duPont Hospital For Children Wilmington DE

Northwest Pediatric Gastroenterology LLC Portland OR

Palacky University Olomouc Czech Republic

Prince Salman North West Armed Forces Hospital Tabuk Saudi Arabia

Sapienza University of Rome Rome Italy

Shaare Zedek Medical Center Jerusalem Israel

State University of New York Buffalo Buffalo NY

Teikyo University School of Medicine Tokyo Japan

Texas Children's Hospital Houston TX and Phoenix Children's Hospital Phoenix AZ

The Dana Dwek Children's Hospital The Tel Aviv Medical Center Tel Aviv University Tel Aviv Israel

University College Dublin Dublin Ireland

University of Alberta Edmonton Alberta Canada

University of Athens Athens Greece

University of California San Francisco San Francisco CA and Texas Children's Hospital Houston TX

University of Colorado School of Medicine Aurora CO

University of Helsinki Helsinki Finland

University of Liverpool Liverpool and University of Manchester Manchester UK

University of Ljubljana Ljubljana Slovenia

University of Manitoba Winnipeg Manitoba Canada

University of Naples Federico 2 Naples Italy

University of Pittsburgh Medical Center Pittsburgh PA

University of Rochester Medical Center Rochester NY

University of Toronto Toronto Ontario Canada

University of Ulsan Seoul South Korea

University of Utah Salt Lake City UT

Yale University School of Medicine New Haven CT

Hepatology. 2017 Aug;66(2):315-317. doi: 10.1002/hep.29208. PubMed

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