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

Autor: Deneau MR; University of Utah and Intermountain Primary Children's HospitalSalt Lake CityUT., Mack C; University of Colorado School of MedicineAuroraCO., Perito ER; University of California, San FranciscoSan FranciscoCA., Ricciuto A; University of TorontoTorontoOntarioCanada., Valentino PL; Yale University School of MedicineNew HavenCT., Amin M; Phoenix Children's HospitalPhoenixAZ., Amir AZ; The Dana-Dwek Children's HospitalThe Tel-Aviv Medical CenterTel-Aviv UniversityTel AvivIsrael., Aumar M; University of LilleCHU LilleLilleFrance., Auth M; Alder Hey Children's HospitalLiverpoolUnited Kingdom., Broderick A; Children's Health Ireland at Crumlin & University College DublinDublinIreland., DiGuglielmo M; Nemours Alfred I. duPont Hospital for ChildrenWilmingtonDE., Draijer LG; Amsterdam University Medical CenterAmsterdamThe Netherlands., Tavares Fagundes ED; Federal University of Minas GeraisBelo HorizonteBrazil., El-Matary W; University of ManitobaWinnipegManitobaCanada., Ferrari F; Sapienza University of RomeRomeItaly., Furuya KN; University of Wisconsin-Madison School of Medicine and Public HealthMadisonWI., Gupta N; Emory University School of MedicineAtlantaGA., Hochberg JT; University of MiamiMiamiFL., Homan M; University of LjubljanaLjubljanaSlovenia., Horslen S; University of WashingtonSeattleWA., Iorio R; University of Naples Federico IINaplesItaly., Jensen MK; University of Utah and Intermountain Primary Children's HospitalSalt Lake CityUT., Jonas MM; Boston Children's Hospital and Harvard Medical SchoolBostonMA., Kamath BM; University of TorontoTorontoOntarioCanada., Kerkar N; University of Rochester Medical CenterRochesterNY., Kim KM; University of UlsanSeoulSouth Korea., Kolho KL; University of Helsinki Hospital and Tampere UniversityHelsinkiFinland., Koot BGP; Amsterdam University Medical CenterAmsterdamThe Netherlands., Laborda TJ; University of Utah and Intermountain Primary Children's HospitalSalt Lake CityUT., Lee CK; Boston Children's Hospital and Harvard Medical SchoolBostonMA., Loomes KM; Children's Hospital of PhiladelphiaPhiladelphiaPA., Martinez M; Columbia UniversityNew YorkNY., Miethke A; Cincinnati Children's Hospital Medical CenterCincinnatiOH., Miloh T; University of MiamiMiamiFL., Mogul D; Johns Hopkins UniversityBaltimoreMD., Mohammad S; Lurie Children's HospitalChicagoIL., Mohan P; Children's National Medical CenterWashingtonDC., Moroz S; University of Southern CaliforniaLos AngelesCA., Ovchinsky N; Children's Hospital at MontefioreAlbert Einstein College of MedicineBronxNY., Palle S; Oklahoma UniversityOklahoma CityOK., Papadopoulou A; First Department of PediatricsUniversity of AthensChildren's Hospital Agia SofiaAthensGreece., Rao G; Indiana UniversityIndianapolisIN., Rodrigues Ferreira A; Federal University of Minas GeraisBelo HorizonteBrazil., Sathya P; Memorial UniversitySt. John'sNewfoundlandCanada., Schwarz KB; Johns Hopkins UniversityBaltimoreMD.; University of California San DiegoSan DiegoCA., Shah U; Massachusetts General Hospital, Harvard Medical SchoolBostonMA., Shteyer E; Shaare Zedek Medical CenterJerusalemIsrael., Singh R; Cincinnati Children's Hospital Medical CenterCincinnatiOH., Smolka V; Palacky UniversityOlomoucCzech Republic., Soufi N; University of Southern CaliforniaLos AngelesCA., Tanaka A; Teikyo University School of MedicineTokyoJapan., Varier R; Northwest Pediatric Gastroenterology LLCPortlandOR., Vitola B; Medical College of WisconsinMilwaukeeWI., Woynarowski M; Faculty of Medicine and Health SciencesUJK KielceKielcePoland., Zerofsky M; University of California San FranciscoSan FranciscoCA., Zizzo A; London Health Sciences CenterWestern UniversityLondonOntarioCanada., Guthery SL; University of Utah and Intermountain Primary Children's HospitalSalt Lake CityUT.
Jazyk: angličtina
Zdroj: Hepatology (Baltimore, Md.) [Hepatology] 2021 Mar; Vol. 73 (3), pp. 1074-1087. Date of Electronic Publication: 2020 Dec 19.
DOI: 10.1002/hep.31393
Abstrakt: 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.)
Databáze: MEDLINE
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