Jill C Handisides1, Danielle Hollenbeck-Pringle2, Karen Uzark3, Felicia L Trachtenberg2, Victoria L Pemberton4, Teresa W Atz5, Timothy J Bradley6, Elizabeth Cappella7, Sylvia De Nobele8, Georgeann Keh-Teng Groh9, Michelle S Hamstra10, Rosalind Korsin11, Jami C Levine1, Bergen Lindauer12, Aimee Liou13, Meghan K Mac Neal14, Larry W Markham15, Tonia Morrison16, Kathleen A Mussatto17, Aaron K Olson18, Mary Ella M Pierpont19, Reed E Pyeritz20, Elizabeth A Radojewski6, Mary J Roman21, Mingfen Xu22, Ronald V Lacro23. 1. Boston Children's Hospital, Harvard Medical School, Boston, MA. 2. New England Research Institutes Inc., Watertown, MA. 3. C. S. Mott Children's Hospital, University of Michigan, Ann Arbor, MI. 4. National Heart, Lung, and Blood Institute, Bethesda, MD. 5. Medical University of South Carolina, Charleston, SC. 6. The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada. 7. Ann and Robert H. Lurie Children's Hospital, Chicago, IL. 8. Ghent University Hospital, Ghent, Belgium. 9. Washington University School of Medicine, St. Louis, MO. 10. Cincinnati Children's Hospital Medical Center, Cincinnati, OH. 11. Children's Hospital of New York, New York, NY. 12. Primary Children's Hospital, University of Utah, Salt Lake City, UT. 13. Texas Children's Hospital, Houston, TX. 14. Icahn School of Medicine at Mount Sinai, New York, NY. 15. The Monroe Carell Jr. Children's Hospital at Vanderbilt, Nashville, TN. 16. Children's Hospital of Philadelphia, Philadelphia, PA. 17. Children's Hospital of Wisconsin, Milwaukee, WI. 18. Seattle Children's Hospital, Seattle, WA. 19. Children's Hospital and Clinic of Minnesota, St. Paul, MN. 20. The Perlman School of Medicine, University of Pennsylvania, Philadelphia, PA. 21. Weill Cornell Medical Center, New York, NY. 22. Duke University School of Medicine, Durham, NC. 23. Boston Children's Hospital, Harvard Medical School, Boston, MA. Electronic address: ron.lacro@cardio.chboston.org.
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.
RCT Entities:
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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