Assessing the Validity of Adult-derived Prognostic Models for Primary Sclerosing Cholangitis Outcomes in Children

Mark R. Deneau*, Pamela L. Valentino, Cara Mack, Khaled Alqoaer, Mansi Amin, Achiya Z. Amir, Madeleine Aumar, Marcus Auth, Annemarie Broderick, Matthew Diguglielmo, Laura G. Draijer, Wael El-Matary, Federica Ferrari, Katryn N. Furuya, Frederic Gottrand, Nitika Gupta, Matjaz Homan, M. K. Jensen, Binita M. Kamath, Kyung Mo KimKaija Leena Kolho, Bart Koot, Raffaele Iorio, Mercedes Martinez, Tamir Miloh, Parvathi Mohan, Sirish Palle, Alexandra Papadopoulou, Amanda Ricciuto, Lawrence Saubermann, Pushpa Sathya, Eyal Shteyer, Vratislav Smolka, Atsushi Tanaka, Raghu Varier, Veena Venkat, Bernadette Vitola, Marek Woynarowski, Stephen Guthery

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

2 Scopus citations


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.

Original languageEnglish
Pages (from-to)E12-E17
JournalJournal of Pediatric Gastroenterology and Nutrition
Issue number1
StatePublished - 1 Jan 2020


FundersFunder number
Primary Children's Hospital Foundation
Primary Children’s Hospital Foundation
National Institutes of Health8UL1TR000105
National Institutes of Health
National Center for Research ResourcesUL1RR025764
National Center for Research Resources
Gilead Sciences
National Center for Advancing Translational SciencesKL2TR001065
National Center for Advancing Translational Sciences
PSC Partners Seeking a Cure


    • natural history
    • pediatric
    • primary sclerosing cholangitis
    • prognosis
    • risk stratification


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