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Objective: To assess health-related quality of life (HRQOL) in a large multicenter cohort of children and young adults with Marfan syndrome participating in the Pediatric Heart Network Marfan Trial. Study design: The Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales were administered to 321 subjects with Marfan syndrome (5-25 years). PedsQL scores were compared with healthy population norms. The impact of treatment arm (atenolol vs losartan), severity of clinical features, and number of patient-reported symptoms on HRQOL was assessed by general linear models. Results: Mean PedsQL scores in children (5-18 years) with Marfan syndrome were lower than healthy population norms for physical (P <= .003) and psychosocial (P < .001) domains; mean psychosocial scores for adults (19-25 years) were greater than healthy norms (P < .001). HRQOL across multiple domains correlated inversely with frequency of patient-reported symptoms (r = 0.30-0.38, P < .0001). Those <18 years of age with neurodevelopmental disorders (mainly learning disability, attention-deficit/hyperactivity disorder) had lower mean PedsQL scores (5.5-7.4 lower, P < .04). A multivariable model found age, sex, patient-reported symptoms, and neurodevelopmental disorder to be independent predictors of HRQOL. There were no differences in HRQOL scores by treatment arm, aortic root z score, number of skeletal features. or presence of ectopia lentis. Conclusions: Children and adolescents with Marian syndrome were at high risk for impaired HRQOL. Patient-reported symptoms and neurodevelopmental disorder. but not treatment arm or severity of Marian syndrome-related physical findings, were associated with lower HRQOL.
Keywords
CHRONIC PAIN, ATENOLOL, LOSARTAN, PEDSQL(TM)-4.0, ASSOCIATIONS, RELIABILITY, FATIGUE, TRIAL, MFS

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Chicago
Handisides, Jill C, Danielle Hollenbeck-Pringle, Karen Uzark, Felicia L Trachtenberg, Victoria L Pemberton, Teresa W Atz, Timothy J Bradley, et al. 2019. “Health-related Quality of Life in Children and Young Adults with Marfan Syndrome.” Journal of Pediatrics 204: 250–255.
APA
Handisides, J. C., Hollenbeck-Pringle, D., Uzark, K., Trachtenberg, F. L., Pemberton, V. L., Atz, T. W., Bradley, T. J., et al. (2019). Health-related quality of life in children and young adults with Marfan syndrome. JOURNAL OF PEDIATRICS, 204, 250–255.
Vancouver
1.
Handisides JC, Hollenbeck-Pringle D, Uzark K, Trachtenberg FL, Pemberton VL, Atz TW, et al. Health-related quality of life in children and young adults with Marfan syndrome. JOURNAL OF PEDIATRICS. 2019;204:250–5.
MLA
Handisides, Jill C et al. “Health-related Quality of Life in Children and Young Adults with Marfan Syndrome.” JOURNAL OF PEDIATRICS 204 (2019): 250–255. Print.
@article{8603982,
  abstract     = {Objective: To assess health-related quality of life (HRQOL) in a large multicenter cohort of children and young adults with Marfan syndrome participating in the Pediatric Heart Network Marfan Trial. 
Study design: The Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales were administered to 321 subjects with Marfan syndrome (5-25 years). PedsQL scores were compared with healthy population norms. The impact of treatment arm (atenolol vs losartan), severity of clinical features, and number of patient-reported symptoms on HRQOL was assessed by general linear models. 
Results: Mean PedsQL scores in children (5-18 years) with Marfan syndrome were lower than healthy population norms for physical (P {\textlangle}= .003) and psychosocial (P {\textlangle} .001) domains; mean psychosocial scores for adults (19-25 years) were greater than healthy norms (P {\textlangle} .001). HRQOL across multiple domains correlated inversely with frequency of patient-reported symptoms (r = 0.30-0.38, P {\textlangle} .0001). Those {\textlangle}18 years of age with neurodevelopmental disorders (mainly learning disability, attention-deficit/hyperactivity disorder) had lower mean PedsQL scores (5.5-7.4 lower, P {\textlangle} .04). A multivariable model found age, sex, patient-reported symptoms, and neurodevelopmental disorder to be independent predictors of HRQOL. There were no differences in HRQOL scores by treatment arm, aortic root z score, number of skeletal features. or presence of ectopia lentis. 
Conclusions: Children and adolescents with Marian syndrome were at high risk for impaired HRQOL. Patient-reported symptoms and neurodevelopmental disorder. but not treatment arm or severity of Marian syndrome-related physical findings, were associated with lower HRQOL.},
  author       = {Handisides, Jill C and Hollenbeck-Pringle, Danielle and Uzark, Karen and Trachtenberg, Felicia L and Pemberton, Victoria L and Atz, Teresa W and Bradley, Timothy J and Cappella, Elizabeth and De Nobele, Sylvia and Groh, Georgeann Keh-Teng and Hamstra, Michelle S and Korsin, Rosalind and Levine, Jami C and Lindauer, Bergen and Liou, Aimee and Mac Neal, Meghan K and Markham, Larry W and Morrison, Tonia and Mussatto, Kathleen A and Olson, Aaron K and Pierpont, Mary Ella M and Pyeritz, Reed E and Radojewski, Elizabeth A and Roman, Mary J and Xu, Mingfen and Lacro, Ronald V and Pediatric Heart Network Investigators, for the and Loeys, Bart and De Backer, Julie and Cobben, Jan Maarten and Sluysmans, Thierry and De Paepe, Anne},
  issn         = {0022-3476},
  journal      = {JOURNAL OF PEDIATRICS},
  language     = {eng},
  pages        = {250--255},
  title        = {Health-related quality of life in children and young adults with Marfan syndrome},
  url          = {http://dx.doi.org/10.1016/j.jpeds.2018.08.061},
  volume       = {204},
  year         = {2019},
}

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