Portal de Periódicos da CAPES

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

2018; Elsevier BV; Volume: 204; Linguagem: Inglês

10.1016/j.jpeds.2018.08.061

1097-6833

Jill Handisides, Danielle Hollenbeck-Pringle, Karen Uzark, Felicia Trachtenberg, Victoria L. Pemberton, Teresa W. Atz, Timothy J. Bradley, Elizabeth Cappella, Sylvia De Nobele, Georgeann Groh, Michelle Hamstra, Rosalind Korsin, Jami C. Levine, Bergen Lindauer, Aimée Liou, Meghan K. Mac Neal, Larry W. Markham, Tonia Morrison, Kathleen Mussatto, Aaron K. Olson, Mary Ella Pierpont, Reed E. Pyeritz, Elizabeth Radojewski, Mary J. Roman, Mingfen Xu, Ronald V. Lacro, Gail D. Pearson, Mario Stylianou, Lynn Mahony, Lynn A. Sleeper, Sharon L. Tennstedt, Steven D. Colan, Gloria L. Klein, Lin T. Guey, Lisa Wruck, Thomas G. Travison, Shan Chen, Eric Gerstenberger, Tanya Olesker, David F. Teitel, Jane W. Newburger, Martha King, Carolyn Dunbar‐Masterson, Andrea Posa, Quincy Nang, Cara Hass, Daphne T. Hsu, Wyman W. Lai, William E. Hellenbrand, Beth F. Printz, Richard B. Devereux, Greysi Sherwood, Victoria L. Vetter, Stephen M. Paridon, Marie M. Gleason, Nicole Mirarchi, Sandra DiLullo, Agbenu Ejembi, Ruth Morgan, D. Woodrow Benson, William L. Border, James F. Cnota, Haleh Heydarian, Jeanne James, Kathryn Hogan, Lois Bogenschutz, Mary Pat Benham, Teresa Barnard, Page A.W. Anderson, Jennifer S. Li, Stephanie Burns Wechsler, Amanda L. Cook, Charles Sang, Wesley Covitz, Lori Jo Sutton, Kari Crawford, Summer Roberts, Deborah Palmer, J. Philip Saul, Andrew M. Atz, Geoffrey A. Forbus, Patricia Infinger, Aparna Choudhury, LuAnn Minich, Richard D. Williams, Angela Yetman, Marian E. Shearrow, Michelle Robinson, June Porter, Brian W. McCrindle, Jennifer Russell, Jack M. Colman, Svetlana Khaikin, Nancy Slater, Harry C. Dietz, William Ravekes, Mary F. Rykiel, Elisabeth Sparks, Gretchen MacCarrick, Jennifer Leadroot, Charles E. Canter, Angela M. Sharkey, Alan C. Braverman, Cheryl A. Rainey, John L. Jefferies, Timothy C. Slesnick, Hugo R. Martinez, Andres Menesses, Tunu Tenende, David Liang, Elisabeth Merkel, Bart Loeys, Julie De Backer, Jan Maarten Cobben, Thierry Sluysmans, Anne De Paepe, Bruce D. Gelb, Shubhika Srivastava, Tejani Mendiz-Ramdeen, Constance G. Weismann, Emily Lawrence, Stephanie Chin, Helen Ko, Jen Le Yau, Steven A. Webber, Stacey Drant, Jane Luce, Kevin Stiegler, Cheryl Kinnard, C. Stewart, Sue Sommers, Carol Madison, Luciana Young, Megan Domenico, Kathryn Waitzman, Carla Lozano, Charles C. Baker, Erin E. Zielinski, Heidi Vander Velden, Alison Overman, Mark Lewin, Amy Payne, David L. Rimoin, Mitchel Pariani, Robert J. Siegel, Asim Rafique, Paul Grossfeld, Arlene Smith, Terri McLees-Palinkas, Steven D. Colan, Elif Seda Selamet Tierney, Shari Trevey, Marga Rivera, Michael Artman, Erle H. Austin, H. Scott Baldwin, Daniel Bernstein, Timothy F. Feltes, Julie Johnson, Thomas S. Klitzner, Jeffrey Krischer, G. Paul Matherne, Kenneth Zahka, John D. Kugler, David J. Driscoll, Mark Galantowicz, Sally Hunsberger, Thomas J. Knight, Holly L. Taylor,

Cardiovascular Issues in Pregnancy

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. 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. 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. 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. 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.