The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children

• Type of Material: Article
• Authors: Mark R Deneau, University of Utah; Cara Mack, Univ Colorado; Emily R Perito, University of California San Francisco; Amanda Ricciuto, University of Toronto; Pamela L Valentino, Yale University; Mansi Amin, Phoenix Children’s Hospital; Achiya Z Amir, Tel Aviv University; Madeleine Aumar, University of Lille; Marcus Auth, Alder Hey Children’s Hospital; Annemarie Broderick, Children’s Health Ireland at Crumlin; Matthew DiGuglielmo, Nemours Alfred I. duPont Hospital for Children; Laura G Draijer, University of Amsterdam; Eleonora Druve Tavares Fagundes, Federal University of Minas Gerais, Belo Horizonte; Wael El-Matary, University of Manitoba; Federica Ferrari, Sapienza University Rome; Katryn N Furuya, University of Wisconsin-Madison; Nitika Gupta, Emory University; Jessica T Hochberg, University of Miami; Matjaz Homan, University of Ljubljana; Simon Horslen, University of Washington; Raffaele Iorio, University of Naples Federico II; Kyle M Jensen, University of Utah; Maureen M Jonas, Boston Children’s Hospital; Binita M Kamath, University of Toronto; Nanda Kerkar, University of Rochester; Kyung Mo Kim, University of Ulsan; Kaija-Leena Kolho, University of Helsinki Hospital; Bart GP Koot, University of Amsterdam; Trevor J Laborda, University of Utah; Christine K Lee, Boston Children’s Hospital; Kathleen M Loomes, Childrens Hospital of Philadelphia; Mercedes Martinez, Columbia University; Alexander Miethke, Cincinnati Children’s Hospital Medical Center; Tamir Miloh, University of Miami; Douglas Mogul, Johns Hopkins University; Saeed Mohammad, Lurie Children’s Hospital; Parvathi Mohan, Children’s National Medical Center; Stacy Moroz, University of Southern California; Nadia Ovchinsky, Albert Einstein College of Medicine; Sirish Palle, Oklahoma University; Alexandra Papadopoulou, University of Athens; Girish Rao, Indiana University; Alexandre Rodrigues Ferreira, Federal University of Minas Gerais, Belo Horizonte; Pushpa Sathya, Memorial University, St. John’s, Newfoundland; Kathleen B Schwarz, Johns Hopkins University; Uzma Shah, Harvard Medical School; Eyal Shteyer, Shaare Zedek Medical Center; Ruchi Singh, Cincinnati Children’s Hospital Medical Center; Vratislav Smolka, Palacky University; Nisreen Soufi, University of Southern California; Atsushi Tanaka, Teikyo University; Raghu Varier, Northwest Pediat Gastroenterol LLC; Bernadette Vitola, Medical College of Wisconsin; Marek Woynarowski, UJK Kielce; Melissa Zerofsky, University of California San Francisco; Andréanne Zizzo, Western University, London; Stephen L Guthery, University of Utah
• Language: English
• Date: 2020-12-19
• Publisher: WILEY
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2020 by the American Association for the Study of Liver Diseases.
• Final Published Version (URL): http://dx.doi.org/10.1002/hep.31393
• Title of Journal or Parent Work: HEPATOLOGY
• Volume: 73
• Issue: 3
• Start Page: 1074
• End Page: 1087
• Keywords: Life Sciences & Biomedicine; MODEL; AUTOIMMUNE HEPATITIS; CHOLANGIOCARCINOMA; Gastroenterology & Hepatology; NATURAL-HISTORY; Science & Technology; SURVIVAL; VALIDATION; RISK

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.