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Health-Related Quality of Life in Children and Young Adults with Marfan Syndrome

Handisides, Jill C ; Hollenbeck-Pringle, Danielle ; Uzark, Karen ; Trachtenberg, Felicia L ; Pemberton, Victoria L ; Atz, Teresa W ; Bradley, Timothy J ; Cappella, Elizabeth ; De Nobele, Sylvia and Groh, Georgeann Keh-Teng , et al. (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.

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author collaboration
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type
Contribution to journal
publication status
published
subject
in
Journal of Pediatrics
volume
204
pages
1 - 255
publisher
Academic Press
external identifiers
  • scopus:85053937143
  • pmid:30270167
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
2021-06-23 03:03:24
@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 &lt; .001) domains; mean psychosocial scores for adults (19-25 years) were greater than healthy norms (P &lt; .001). HRQOL across multiple domains correlated inversely with frequency of patient-reported symptoms (r = 0.30-0.38, P &lt; .0001). Those &lt;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 &lt; .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},
}