@article{3102751,
    title = "The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children",
    author = "Deneau, M.R. and Mack, C. and Perito, E.R. and Ricciuto, A. and Valentino, P.L. and Amin, M. and Amir, A.Z. and Aumar, M. and Auth, M. and Broderick, A. and DiGuglielmo, M. and Draijer, L.G. and Tavares Fagundes, E.D. and El-Matary, W. and Ferrari, F. and Furuya, K.N. and Gupta, N. and Hochberg, J.T. and Homan, M. and Horslen, S. and Iorio, R. and Jensen, M.K. and Jonas, M.M. and Kamath, B.M. and Kerkar, N. and Kim, K.M. and Kolho, K.-L. and Koot, B.G.P. and Laborda, T.J. and Lee, C.K. and Loomes, K.M. and Martinez, M. and Miethke, A. and Miloh, T. and Mogul, D. and Mohammad, S. and Mohan, P. and Moroz, S. and Ovchinsky, N. and Palle, S. and Papadopoulou, A. and Rao, G. and Rodrigues Ferreira, A. and Sathya, P. and Schwarz, K.B. and Shah, U. and Shteyer, E. and Singh, R. and Smolka, V. and Soufi, N. and Tanaka, A. and Varier, R. and Vitola, B. and Woynarowski, M. and Zerofsky, M. and Zizzo, A. and Guthery, S.L.",
    journal = "WORLD JOURNAL OF HEPATOLOGY",
    year = "2021",
    volume = "73",
    number = "3",
    pages = "1074-1087",
    publisher = "John Wiley and Sons Inc",
    doi = "10.1002/hep.31393",
    keywords = "albumin;  bilirubin;  gamma glutamyltransferase;  bilirubin;  gamma glutamyltransferase;  serum albumin, adolescent;  Article;  child;  cholangiography;  cohort analysis;  comparative study;  controlled study;  death;  digestive system disease assessment;  female;  follow up;  human;  human tissue;  liver biopsy;  liver fibrosis;  liver stiffness;  liver transplantation;  low risk patient;  major clinical study;  male;  Model For End Stage Liver Disease Score;  phenotype;  platelet count;  prediction;  primary sclerosing cholangitis;  priority journal;  prognosis;  retrospective study;  Sclerosing Cholangitis Outcomes Pediatrics Index;  transient elastography;  biopsy;  blood;  disease exacerbation;  mortality;  pathology;  prognosis;  risk factor;  sclerosing cholangitis, Adolescent;  Bilirubin;  Biopsy;  Child;  Cholangiography;  Cholangitis, Sclerosing;  Disease Progression;  Female;  gamma-Glutamyltransferase;  Humans;  Liver Transplantation;  Male;  Platelet Count;  Prognosis;  Retrospective Studies;  Risk Factors;  Serum Albumin",
    abstract = "Background and Aims: Disease progression in children with primary sclerosing cholangitis (PSC) is variable. Prognostic and risk-stratification tools exist for adult-onset PSC, but not for children. We aimed to create a tool that accounts for the biochemical and phenotypic features and early disease stage of pediatric PSC. Approach and Results: We used retrospective data from the Pediatric PSC Consortium. The training cohort contained 1,012 patients from 40 centers. We generated a multivariate risk index (Sclerosing Cholangitis Outcomes in Pediatrics [SCOPE] index) that contained total bilirubin, albumin, platelet count, gamma glutamyltransferase, and cholangiography to predict a primary outcome of liver transplantation or death (TD) and a broader secondary outcome that included portal hypertensive, biliary, and cancer complications termed hepatobiliary complications (HBCs). The model stratified patients as low, medium, or high risk based on progression to TD at rates of <1%, 3%, and 9% annually and to HBCs at rates of 2%, 6%, and 13% annually, respectively (P < 0.001). C-statistics to discriminate outcomes at 1 and 5 years were 0.95 and 0.82 for TD and 0.80 and 0.76 for HBCs, respectively. Baseline hepatic fibrosis stage was worse with increasing risk score, with extensive fibrosis in 8% of the lowest versus 100% with the highest risk index (P < 0.001). The model was validated in 240 children from 11 additional centers and performed well. Conclusions: The SCOPE index is a pediatric-specific prognostic tool for PSC. It uses routinely obtained, objective data to predict a complicated clinical course. It correlates strongly with biopsy-proven liver fibrosis. SCOPE can be used with families for shared decision making on clinical care based on a patient’s individual risk, and to account for variable disease progression when designing future clinical trials. © 2020 by the American Association for the Study of Liver Diseases."
}