title: Wilson's disease in children: 37-year experience and revised King's score for liver transplantation.
abstract: Wilson's disease (WD) is a rare liver-based disorder of copper metabolism. Prognostic criteria described by our group in 1986 to predict death without transplantation have not been universally validated. The clinical features of 88 children were reviewed, retrospectively in 74 and prospectively in 14. Data from the retrospectively recruited patients that died or survived on long-term chelation were used to evaluate the validity of our old scoring system and to devise a new prognostic index, then assessed in the 14 prospectively recruited patients. Using the old scoring system, 5 children scoring > or = 7, the cutoff value for death without transplantation, survived, whereas 4 scoring < or = 7 died (sensitivity 87% and specificity 90%). A new index based on serum bilirubin, international normalized ratio, aspartate aminotransferase (AST), and white cell count (WCC) at presentation identified a cutoff score of 11 for death and proved to be 93% sensitive and 98% specific, with a positive predictive value of 88%. When the new index was evaluated prospectively in 14 patients, it predicted the need for transplantation in only the 4 who required it, although 1 child with a score of 11 survived on medical treatment. In conclusion, the new Wilson Index is more sensitive and specific in predicting mortality without transplantation than the old scoring system, but needs to be validated in a larger number of patients.
authors.affiliation: Paediatric Liver Service, King's College Hospital, London, UK. anil.dhawan@kcl.ac.uk
authors.forename: Anil
authors.initials: A
authors.lastname: Dhawan
authors.forename: Rachel M
authors.initials: RM
authors.lastname: Taylor
authors.forename: Paul
authors.initials: P
authors.lastname: Cheeseman
authors.forename: Pamela
authors.initials: P
authors.lastname: De Silva
authors.forename: Leah
authors.initials: L
authors.lastname: Katsiyiannakis
authors.forename: Giorgina
authors.initials: G
authors.lastname: Mieli-Vergani
mesh_terms: D000293:Adolescent; D001219:Aspartate Aminotransferases / Q000097:blood; D001663:Bilirubin / Q000097:blood; D015913:Chelation Therapy; D002648:Child; D002675:Child, Preschool; D003300:Copper; D006305:Health Status Indicators; D006527:Hepatolenticular Degeneration / Q000175:diagnosis / Q000401:mortality / Q000601:surgery* / Q000628:therapy; D006801:Humans; D019934:International Normalized Ratio; D016031:Liver Transplantation; D011379:Prognosis; D011446:Prospective Studies; D012372:ROC Curve; D012189:Retrospective Studies; D012680:Sensitivity and Specificity
chemical_list: D003300:Copper; D001219:Aspartate Aminotransferases; D001663:Bilirubin