title: Quality of Life and Its Determinants in a Multicenter Cohort of Children with Alagille Syndrome.
abstract: OBJECTIVE
  To assess health-related quality of life (HRQOL) in children with Alagille syndrome (ALGS) in comparison with healthy and other liver disease cohorts, and to identify determinants of HRQOL in patients with ALGS.
  
  
  METHODS
  Within the Childhood Liver Disease Research Network prospective study of cholestasis, Pediatric Quality of Life Inventory (PedsQL) questionnaires were administered to 70 children with ALGS, 95 children with alpha-1-antitrypsin deficiency (A1ATD), and 49 children with other causes of chronic intrahepatic cholestasis (IHC) aged 5-18 years. Parent proxy PedsQL scores were recorded for children aged 2-18 years (98 ALGS, 123 A1ATD, and 68 IHC).
  
  
  RESULTS
  Mean ages and total bilirubin (mg/dL) were ALGS 9.4 years; 4.4, A1ATD 9.5 years; 0.7, and IHC 10.3 years; 2.9. ALGS child PedsQL scores were lower than in healthy children and children with A1ATD (mean 73 vs 83; P = .001). Children with ALGS and IHC were similar, except in physical scores (73 vs 79; P = .05). Parents of children with ALGS perceived their children to have worse HRQOL than A1ATD (P ≤ .001) and marginally lower compared with IHC. Univariate analysis revealed ALGS child-reported scores were positively associated with better growth and inversely with total bilirubin. Growth failure, elevated international normalized ratio, and an intracardiac defect were predictive of poor parental scores (P ≤ .05). In multivariate analysis, only weight z-score remained significant for child- and parent-reported scores.
  
  
  CONCLUSIONS
  HRQOL is impaired in children with ALGS compared with healthy and children with A1ATD, similar to children with IHC and is associated with growth failure, which is a potentially treatable cause of impaired HRQOL.
authors.affiliation: The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada. Electronic address: binita.kamath@sickkids.ca.
authors.forename: Binita M
authors.initials: BM
authors.lastname: Kamath
authors.affiliation: University of Michigan, Ann Arbor, MI.
authors.forename: Zhen
authors.initials: Z
authors.lastname: Chen
authors.affiliation: Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, GA.
authors.forename: Rene
authors.initials: R
authors.lastname: Romero
authors.affiliation: University of Michigan, Ann Arbor, MI.
authors.forename: Emily M
authors.initials: EM
authors.lastname: Fredericks
authors.affiliation: Ann and Robert H. Lurie Children's Hospital and Northwestern University, Chicago, IL.
authors.forename: Estella M
authors.initials: EM
authors.lastname: Alonso
authors.affiliation: Mount Sinai Medical Center, New York City, NY.
authors.forename: Ronen
authors.initials: R
authors.lastname: Arnon
authors.affiliation: Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: James
authors.initials: J
authors.lastname: Heubi
authors.affiliation: Baylor College of Medicine and Texas Children's Hospital, Houston, TX.
authors.forename: Paula M
authors.initials: PM
authors.lastname: Hertel
authors.affiliation: Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, GA.
authors.forename: Saul J
authors.initials: SJ
authors.lastname: Karpen
authors.affiliation: The Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
authors.forename: Kathleen M
authors.initials: KM
authors.lastname: Loomes
authors.affiliation: Seattle Children's Hospital and the University of Washington School of Medicine, Seattle, WA.
authors.forename: Karen F
authors.initials: KF
authors.lastname: Murray
authors.affiliation: University of California, San Francisco, CA.
authors.forename: Philip
authors.initials: P
authors.lastname: Rosenthal
authors.affiliation: Johns Hopkins Medical Institutions, Baltimore, MD.
authors.forename: Kathleen B
authors.initials: KB
authors.lastname: Schwarz
authors.affiliation: Riley Hospital for Children, Indiana University, Indianapolis, IN.
authors.forename: Girish
authors.initials: G
authors.lastname: Subbarao
authors.affiliation: Saint Louis University School of Medicine, St. Louis, MO.
authors.forename: Jeffrey H
authors.initials: JH
authors.lastname: Teckman
authors.affiliation: Washington University, St. Louis, MO.
authors.forename: Yumirle P
authors.initials: YP
authors.lastname: Turmelle
authors.affiliation: Children's Hospital Los Angeles, Los Angeles, CA.
authors.forename: Kasper S
authors.initials: KS
authors.lastname: Wang
authors.affiliation: National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD.
authors.forename: Averell H
authors.initials: AH
authors.lastname: Sherker
authors.affiliation: University of Colorado School of Medicine and Children's Hospital Colorado, Aurora, CO.
authors.forename: Ronald J
authors.initials: RJ
authors.lastname: Sokol
authors.affiliation: University of Michigan, Ann Arbor, MI.
authors.forename: John C
authors.initials: JC
authors.lastname: Magee
mesh_terms: D000293:Adolescent; D016738:Alagille Syndrome / Q000150:complications* / Q000503:physiopathology / Q000523:psychology*; D016022:Case-Control Studies; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D004644:Emotions; D005260:Female; D006304:Health Status; D006801:Humans; D008297:Male; D011788:Quality of Life; D012919:Social Behavior; D011795:Surveys and Questionnaires; D019896:alpha 1-Antitrypsin Deficiency / Q000150:complications / Q000503:physiopathology / Q000523:psychology