Mark R Deneau1, Cara Mack2, Emily R Perito3, Amanda Ricciuto4, Pamela L Valentino5, Mansi Amin6, Achiya Z Amir7, Madeleine Aumar8, Marcus Auth9, Annemarie Broderick10, Matthew DiGuglielmo11, Laura G Draijer12, Eleonora Druve Tavares Fagundes13, Wael El-Matary14, Federica Ferrari15, Katryn N Furuya16, Nitika Gupta17, Jessica T Hochberg18, Matjaz Homan19, Simon Horslen20, Raffaele Iorio21, M Kyle Jensen1, Maureen M Jonas22, Binita M Kamath4, Nanda Kerkar23, Kyung Mo Kim24, Kaija-Leena Kolho25, Bart G P Koot12, Trevor J Laborda1, Christine K Lee22, Kathleen M Loomes26, Mercedes Martinez27, Alexander Miethke28, Tamir Miloh18, Douglas Mogul29, Saeed Mohammad30, Parvathi Mohan31, Stacy Moroz32, Nadia Ovchinsky33, Sirish Palle34, Alexandra Papadopoulou35, Girish Rao36, Alexandre Rodrigues Ferreira13, Pushpa Sathya37, Kathleen B Schwarz29,38, Uzma Shah39, Eyal Shteyer40, Ruchi Singh28, Vratislav Smolka41, Nisreen Soufi32, Atsushi Tanaka42, Raghu Varier43, Bernadette Vitola44, Marek Woynarowski45, Melissa Zerofsky46, Andréanne Zizzo47, Stephen L Guthery1. 1. University of Utah and Intermountain Primary Children's Hospital, Salt Lake City, UT. 2. University of Colorado School of Medicine, Aurora, CO. 3. University of California, San Francisco, San Francisco, CA. 4. University of Toronto, Toronto, Ontario, Canada. 5. Yale University School of Medicine, New Haven, CT. 6. Phoenix Children's Hospital, Phoenix, AZ. 7. The Dana-Dwek Children's Hospital, The Tel-Aviv Medical Center, Tel-Aviv University, Tel Aviv, Israel. 8. University of Lille, CHU Lille, Lille, France. 9. Alder Hey Children's Hospital, Liverpool, United Kingdom. 10. Children's Health Ireland at Crumlin & University College Dublin, Dublin, Ireland. 11. Nemours Alfred I. duPont Hospital for Children, Wilmington, DE. 12. Amsterdam University Medical Center, Amsterdam, The Netherlands. 13. Federal University of Minas Gerais, Belo Horizonte, Brazil. 14. University of Manitoba, Winnipeg, Manitoba, Canada. 15. Sapienza University of Rome, Rome, Italy. 16. University of Wisconsin-Madison School of Medicine and Public Health, Madison, WI. 17. Emory University School of Medicine, Atlanta, GA. 18. University of Miami, Miami, FL. 19. University of Ljubljana, Ljubljana, Slovenia. 20. University of Washington, Seattle, WA. 21. University of Naples Federico II, Naples, Italy. 22. Boston Children's Hospital and Harvard Medical School, Boston, MA. 23. University of Rochester Medical Center, Rochester, NY. 24. University of Ulsan, Seoul, South Korea. 25. University of Helsinki Hospital and Tampere University, Helsinki, Finland. 26. Children's Hospital of Philadelphia, Philadelphia, PA. 27. Columbia University, New York, NY. 28. Cincinnati Children's Hospital Medical Center, Cincinnati, OH. 29. Johns Hopkins University, Baltimore, MD. 30. Lurie Children's Hospital, Chicago, IL. 31. Children's National Medical Center, Washington, DC. 32. University of Southern California, Los Angeles, CA. 33. Children's Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY. 34. Oklahoma University, Oklahoma City, OK. 35. First Department of Pediatrics, University of Athens, Children's Hospital Agia Sofia, Athens, Greece. 36. Indiana University, Indianapolis, IN. 37. Memorial University, St. John's, Newfoundland, Canada. 38. University of California San Diego, San Diego, CA. 39. Massachusetts General Hospital, Harvard Medical School, Boston, MA. 40. Shaare Zedek Medical Center, Jerusalem, Israel. 41. Palacky University, Olomouc, Czech Republic. 42. Teikyo University School of Medicine, Tokyo, Japan. 43. Northwest Pediatric Gastroenterology LLC, Portland, OR. 44. Medical College of Wisconsin, Milwaukee, WI. 45. Faculty of Medicine and Health Sciences, UJK Kielce, Kielce, Poland. 46. University of California San Francisco, San Francisco, CA. 47. London Health Sciences Center, Western University, London, Ontario, Canada.
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.
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.
Authors: Jens J W Tischendorf; Hartmut Hecker; Martin Krüger; Michael P Manns; Peter N Meier Journal: Am J Gastroenterol Date: 2006-10-13 Impact factor: 10.864
Authors: Annika Bergquist; Anders Ekbom; Rolf Olsson; Dan Kornfeldt; Lars Lööf; Ake Danielsson; Rolf Hultcrantz; Stefan Lindgren; Hanne Prytz; Hanna Sandberg-Gertzén; Sven Almer; Fredrik Granath; Ulrika Broomé Journal: J Hepatol Date: 2002-03 Impact factor: 25.083
Authors: Cynthia W Buness; Kevin M Johnson; Ahmad Hassan Ali; Leina Alrabadi; Keith D Lindor; Tamir Miloh; Kenneth L Cox Journal: Clin J Gastroenterol Date: 2020-11-24
Authors: Mark R Deneau; Cara Mack; Douglas Mogul; Emily R Perito; Pamela L Valentino; Achiya Z Amir; Matthew DiGuglielmo; Laura G Draijer; Wael El-Matary; Katryn N Furuya; Nitika Gupta; Jessica T Hochberg; Simon Horslen; M Kyle Jensen; Maureen M Jonas; Nanda Kerkar; Bart G P Koot; Trevor J Laborda; Christine K Lee; Kathleen M Loomes; Mercedes Martinez; Alexander Miethke; Tamir Miloh; Saeed Mohammad; Nadia Ovchinsky; Girish Rao; Amanda Ricciuto; Pushpa Sathya; Kathleen B Schwarz; Uzma Shah; Ruchi Singh; Bernadette Vitola; Andréanne Zizzo; Stephen L Guthery Journal: Hepatology Date: 2021-03 Impact factor: 17.425
Authors: Angelo Di Giorgio; Anna Tulone; Emanuele Nicastro; Lorenzo Norsa; Aurelio Sonzogni; Lorenzo D'Antiga Journal: World J Hepatol Date: 2021-12-27