Faculty Collaboration Database

Health-Related Quality of Life in Children and Young Adults with Marfan Syndrome. J Pediatr 2019 Jan;204:250-255.e1

Date

10/03/2018

Pubmed ID

30270167

Pubmed Central ID

PMC6800200

DOI

10.1016/j.jpeds.2018.08.061

Scopus ID

2-s2.0-85053937143 (requires institutional sign-in at Scopus site)   22 Citations

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 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.

Author List

Handisides JC, Hollenbeck-Pringle D, Uzark K, Trachtenberg FL, Pemberton VL, Atz TW, Bradley TJ, Cappella E, De Nobele S, Groh GK, Hamstra MS, Korsin R, Levine JC, Lindauer B, Liou A, Neal MKM, Markham LW, Morrison T, Mussatto KA, Olson AK, Pierpont MEM, Pyeritz RE, Radojewski EA, Roman MJ, Xu M, Lacro RV, Pediatric Heart Network Investigators

Author

Kathleen Mussatto Ph.D. Associate Professor in the School of Nursing department at Milwaukee School of Engineering

MESH terms used to index this publication - Major topics in bold

Adolescent
Adult
Antihypertensive Agents
Atenolol
Child
Child, Preschool
Female
Health Status Indicators
Humans
Losartan
Male
Marfan Syndrome
Patient Reported Outcome Measures
Quality of Life
Severity of Illness Index
Young Adult