BACKGROUND: Natural history models for primary sclerosing cholangitis (PSC) are derived from adult patient data, but have never been validated in children. It is unclear how accurate such models are for children with PSC. METHODS: We utilized the pediatric PSC consortium database to assess the Revised Mayo Clinic, Amsterdam-Oxford, and Boberg models. We calculated the risk stratum and predicted survival for each patient within each model using patient data at PSC diagnosis, and compared it with observed survival. We evaluated model fit using the c-statistic. RESULTS: Model fit was good at 1 year (c-statistics 0.93, 0.87, 0.82) and fair at 10 years (0.78, 0.75, 0.69) in the Mayo, Boberg, and Amsterdam-Oxford models, respectively. The Mayo model correctly classified most children as low risk, whereas the Amsterdam-Oxford model incorrectly classified most as high risk. All of the models underestimated survival of patients classified as high risk. Albumin, bilirubin, AST, and platelets were most associated with outcomes. Autoimmune hepatitis was more prevalent in higher risk groups, and over-weighting of AST in these patients accounted for the observed versus predicted survival discrepancy. CONCLUSIONS: All 3 models offered good short-term discrimination of outcomes but only fair long-term discrimination. None of the models account for the high prevalence of features of autoimmune hepatitis overlap in children and the associated elevated aminotransferases. A pediatric-specific model is needed. AST, bilirubin, albumin, and platelets will be important predictors, but must be weighted to account for the unique features of PSC in children.

Academic Medical Centre Amsterdam The Netherlands

Alder Hey Children's Hospital Liverpool United Kingdom

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

King Salman Armed Forces Hospital Tabuk Saudi Arabia

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

Phoenix Children's Hospital Phoenix AZ

Sapienza University of Rome Rome Italy

Shaare Zedek Medical Center Jerusalem Israel

Teikyo University School of Medicine Tokyo Japan

Texas Children's Hospital Houston TX

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

University College Dublin Dublin Ireland

University of Athens Athens Greece

University of Colorado School of Medicine Aurora CO

University of Helsinki Helsinki Finland

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

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