Quality of Life and Its Determinants in a Multicenter Cohort of Children with Alagille Syndrome

摘要: Objectives To assess health-related quality of life (HRQOL) in children with Alagille syndrome (ALGS) comparison healthy and other liver disease cohorts, to identify determinants HRQOL patients ALGS. Study design Within the Childhood Liver Disease Research Network prospective study cholestasis, Pediatric Quality Life Inventory (PedsQL) questionnaires were administered 70 ALGS, 95 alpha-1-antitrypsin deficiency (A1ATD), 49 causes chronic intrahepatic cholestasis (IHC) aged 5-18 years. Parent proxy PedsQL scores recorded for 2-18 years (98 123 A1ATD, 68 IHC). Results Mean ages total bilirubin (mg/dL) ALGS 9.4 years; 4.4, A1ATD 9.5 years; 0.7, IHC 10.3 years; 2.9. child lower than (mean 73 vs 83; P  = .001). Children similar, except physical (73 79;  = .05). Parents perceived their have worse (  ≤ .001) marginally compared IHC. Univariate analysis revealed child-reported positively associated better growth inversely bilirubin. Growth failure, elevated international normalized ratio, an intracardiac defect predictive poor parental  ≤ .05). In multivariate analysis, only weight z-score remained significant child- parent-reported scores. Conclusions is impaired similar which a potentially treatable cause HRQOL.