TY - JOUR
T1 - Quality of Life and Its Determinants in a Multicenter Cohort of Children with Alagille Syndrome
AU - Kamath, Binita M.
AU - Chen, Zhen
AU - Romero, Rene
AU - Fredericks, Emily M.
AU - Alonso, Estella M.
AU - Arnon, Ronen
AU - Heubi, James
AU - Hertel, Paula M.
AU - Karpen, Saul J.
AU - Loomes, Kathleen M.
AU - Murray, Karen F.
AU - Rosenthal, Philip
AU - Schwarz, Kathleen B.
AU - Subbarao, Girish
AU - Teckman, Jeffrey H.
AU - Turmelle, Yumirle P.
AU - Wang, Kasper S.
AU - Sherker, Averell H.
AU - Sokol, Ronald J.
AU - Magee, John C.
N1 - Funding Information:
Supported by the National Institute of Diabetes, Digestive and Kidney Diseases ( DK 62470 [Children's Healthcare of Atlanta, Atlanta, GA], DK 62503 [Johns Hopkins School of Medicine, Baltimore, MD], DK 62436 [Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL], DK 62497 [Cincinnati Children's Hospital Medical Center, Cincinnati, OH], U54DK078377 and DK 62453 [Children's Hospital Colorado, Aurora, CO], DK 62470 [Texas Children's Hospital, Houston, TX], DK 84536 [Riley Hospital for Children, Indianapolis, IN], DK 84538 [Children's Hospital Los Angeles, Los Angeles, CA], DK 62452 [Washington University School of Medicine, St. Louis, MO], DK 62456 [University of Michigan at Ann Arbor, Ann Arbor, MI], DK 62445 [Mount Sinai School of Medicine, New York City, NY], DK 62481 [The Children's Hospital of Philadelphia, Philadelphia, PA], DK 62466 [Children's Hospital of Pittsburgh, Pittsburgh, PA], DK 62500 [UCSF Children's Hospital, San Francisco, CA], DK 84575 [Seattle Children's Hospital, Seattle, WA]), National Center for Advancing Translational Sciences (Clinical and Translational Science Awards)/ National Institutes of Health ( UL1 TR000154 [Children's Hospital Colorado, Aurora, CO], UL1 TR000004 [UCSF Children's Hospital, San Francisco, CA], UL 1TR000424 [Johns Hopkins School of Medicine, Baltimore, MD], UL 1TR000150 [Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL], UL1 TR0000775 [Cincinnati Children's Hospital Medical Center, Cincinnati, OH], UL 1TR000130 [Children's Hospital Los Angeles, Los Angeles, CA], UL1 TR000448 [Washington University School of Medicine, St. Louis, MO], UL1 TR000067 [Mount Sinai School of Medicine, New York City, NY], UL1 TR000003 [The Children's Hospital of Philadelphia, Philadelphia, PA], UL1 TR000005 [Children's Hospital of Pittsburgh, Pittsburgh, PA]), the Alpha-1 Foundation , and the Alagille Syndrome Alliance .
Publisher Copyright:
© 2015 Elsevier Inc.
PY - 2015/8/1
Y1 - 2015/8/1
N2 - Objectives 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. Study design 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.
AB - Objectives 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. Study design 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.
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U2 - 10.1016/j.jpeds.2015.04.077
DO - 10.1016/j.jpeds.2015.04.077
M3 - Article
C2 - 26059338
AN - SCOPUS:84937725853
SN - 0022-3476
VL - 167
SP - 390-396.e3
JO - Journal of Pediatrics
JF - Journal of Pediatrics
IS - 2
ER -