Faculty Collaboration Database

Assessing the Validity of Adult-derived Prognostic Models for Primary Sclerosing Cholangitis Outcomes in Children. J Pediatr Gastroenterol Nutr 2020 Jan;70(1):e12-e17

Date

10/28/2019

Pubmed ID

31651664

DOI

10.1097/MPG.0000000000002522

Scopus ID

2-s2.0-85077298792 (requires institutional sign-in at Scopus site)   2 Citations

Abstract

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.

Author List

Deneau MR, Valentino PL, Mack C, Alqoaer K, Amin M, Amir AZ, Aumar M, Auth M, Broderick A, DiGuglielmo M, Draijer LG, El-Matary W, Ferrari F, Furuya KN, Gottrand F, Gupta N, Homan M, Jensen MK, Kamath BM, Kim KM, Kolho KL, Koot B, Iorio R, Martinez M, Miloh T, Mohan P, Palle S, Papadopoulou A, Ricciuto A, Saubermann L, Sathya P, Shteyer E, Smolka V, Tanaka A, Varier R, Venkat V, Vitola B, Woynarowski M, Guthery S

Authors

Cara Lynn Mack MD Chief, Professor in the Pediatrics department at Medical College of Wisconsin
Bernadette Vitola MD, MPH Associate Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Child
Cholangitis, Sclerosing
Female
Gastroenterology
Hepatitis, Autoimmune
Humans
Kaplan-Meier Estimate
Liver Function Tests
Male
Models, Statistical
Pediatrics
Predictive Value of Tests
Prognosis
Reproducibility of Results
Risk Assessment