Health-Related Quality of Life in Children and Young Adults with Marfan Syndrome
(2019) In Journal of Pediatrics 204. p.1-255- 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 ≤ .003) and... (More)
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 Marfan syndrome were at high risk for impaired HRQOL. Patient-reported symptoms and neurodevelopmental disorder, but not treatment arm or severity of Marfan syndrome-related physical findings, were associated with lower HRQOL.
(Less)
- author
- contributor
- Weismann, Constance
LU
- author collaboration
- publishing date
- 2019-01
- type
- Contribution to journal
- publication status
- published
- subject
- in
- Journal of Pediatrics
- volume
- 204
- pages
- 1 - 255
- publisher
- Academic Press
- external identifiers
-
- pmid:30270167
- scopus:85053937143
- ISSN
- 1097-6833
- DOI
- 10.1016/j.jpeds.2018.08.061
- language
- English
- LU publication?
- no
- id
- f582b2f4-6cb2-4ec7-be57-fbc82c8d9bb4
- date added to LUP
- 2019-01-25 14:35:54
- date last changed
- 2024-05-28 02:24:43
@article{f582b2f4-6cb2-4ec7-be57-fbc82c8d9bb4,
abstract = {{<p>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.</p><p>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.</p><p>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.</p><p>CONCLUSIONS: Children and adolescents with Marfan syndrome were at high risk for impaired HRQOL. Patient-reported symptoms and neurodevelopmental disorder, but not treatment arm or severity of Marfan syndrome-related physical findings, were associated with lower HRQOL.</p>}},
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 Neal, Meghan K Mac 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}},
issn = {{1097-6833}},
language = {{eng}},
pages = {{1--255}},
publisher = {{Academic Press}},
series = {{Journal of Pediatrics}},
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}},
doi = {{10.1016/j.jpeds.2018.08.061}},
volume = {{204}},
year = {{2019}},
}