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

Pediatric Heart Network Investigators; New England Research Institutes

doi:10.1016/j.jpeds.2018.08.061

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

Pediatric Heart Network Investigators, New England Research Institutes

Pediatric Genetics & Metabolism

Research output: Contribution to journal › Article › peer-review

25 Scopus 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.

Original language	English (US)
Pages (from-to)	250-255.e1
Journal	Journal of Pediatrics
Volume	204
DOIs	https://doi.org/10.1016/j.jpeds.2018.08.061
State	Published - Jan 2019

Bibliographical note

Funding Information:
Supported by grants from the Marfan Foundation and the Pediatric Heart Network Nursing Research Program, by U01 grants from the National Heart, Lung, and Blood Institute (HL068269, HL068270, HL068279, HL068281, HL068285, HL068292, HL068290, HL068288, and HL085057) and by the Food and Drug Administration Office of Orphan Products Development. The contents of this paper are solely the responsibility of the authors and do not necessarily represent the official views of the National Institutes of Health or the National Heart, Lung, and Blood Institute. The authors declare no conflicts of interest.

Publisher Copyright:
© 2018 Elsevier Inc.

Keywords

neurodevelopmental disorders
patient-reported symptoms
Antihypertensive Agents/therapeutic use
Severity of Illness Index
Health Status Indicators
Humans
Child, Preschool
Male
Marfan Syndrome/complications
Young Adult
Adolescent
Quality of Life
Adult
Female
Losartan/therapeutic use
Child
Atenolol/therapeutic use
Patient Reported Outcome Measures

PubMed: MeSH publication types

Research Support, U.S. Gov't, P.H.S.
Randomized Controlled Trial
Multicenter Study
Journal Article
Research Support, N.I.H., Extramural

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10.1016/j.jpeds.2018.08.061

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@article{cf1a218eb2e4435fb830305054b8089b,

title = "Health-Related Quality of Life in Children and Young Adults with Marfan Syndrome",

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.",

keywords = "neurodevelopmental disorders, patient-reported symptoms, Antihypertensive Agents/therapeutic use, Severity of Illness Index, Health Status Indicators, Humans, Child, Preschool, Male, Marfan Syndrome/complications, Young Adult, Adolescent, Quality of Life, Adult, Female, Losartan/therapeutic use, Child, Atenolol/therapeutic use, Patient Reported Outcome Measures",

author = "{Pediatric Heart Network Investigators} and {New England Research Institutes} and Handisides, {Jill C.} and Danielle Hollenbeck-Pringle and Karen Uzark and Trachtenberg, {Felicia L.} and Pemberton, {Victoria L.} and Atz, {Teresa W.} and Bradley, {Timothy J.} and Elizabeth Cappella and {De Nobele}, Sylvia and Groh, {Georgeann Keh Teng} and Hamstra, {Michelle S.} and Rosalind Korsin and Levine, {Jami C.} and Bergen Lindauer and Aimee Liou and Neal, {Meghan K.Mac} and Markham, {Larry W.} and Tonia Morrison and Mussatto, {Kathleen A.} and Olson, {Aaron K.} and Pyeritz, {Reed E.} and Radojewski, {Elizabeth A.} and Roman, {Mary J.} and Pierpont, {Mary Ella M} and Lacro, {Ronald V.} and Gail Pearson and Mario Stylianou and Lynn Mahony and Lynn Sleeper and Sharon Tennstedt and Steven Colan and Gloria Klein and Lin Guey and Lisa Wruck and Thomas Travison and Shan Chen and Eric Gerstenberger and Tanya Olesker and Teitel, {David F.} and Jane Newburger and Martha King and Carolyn Dunbar-Masterson and Andrea Posa and Quincy Nang and Cara Hass and Daphne Hsu and Wyman Lai and William Hellenbrand and Beth Printz",

note = "Funding Information: Supported by grants from the Marfan Foundation and the Pediatric Heart Network Nursing Research Program, by U01 grants from the National Heart, Lung, and Blood Institute (HL068269, HL068270, HL068279, HL068281, HL068285, HL068292, HL068290, HL068288, and HL085057) and by the Food and Drug Administration Office of Orphan Products Development. The contents of this paper are solely the responsibility of the authors and do not necessarily represent the official views of the National Institutes of Health or the National Heart, Lung, and Blood Institute. The authors declare no conflicts of interest. Publisher Copyright: {\textcopyright} 2018 Elsevier Inc.",

year = "2019",

month = jan,

doi = "10.1016/j.jpeds.2018.08.061",

language = "English (US)",

volume = "204",

pages = "250--255.e1",

journal = "Journal of Pediatrics",

issn = "0022-3476",

publisher = "Elsevier Inc.",

}

TY - JOUR

T1 - Health-Related Quality of Life in Children and Young Adults with Marfan Syndrome

AU - Pediatric Heart Network Investigators

AU - New England Research Institutes

AU - Handisides, Jill C.

AU - Hollenbeck-Pringle, Danielle

AU - Uzark, Karen

AU - Trachtenberg, Felicia L.

AU - Pemberton, Victoria L.

AU - Atz, Teresa W.

AU - Bradley, Timothy J.

AU - Cappella, Elizabeth

AU - De Nobele, Sylvia

AU - Groh, Georgeann Keh Teng

AU - Hamstra, Michelle S.

AU - Korsin, Rosalind

AU - Levine, Jami C.

AU - Lindauer, Bergen

AU - Liou, Aimee

AU - Neal, Meghan K.Mac

AU - Markham, Larry W.

AU - Morrison, Tonia

AU - Mussatto, Kathleen A.

AU - Olson, Aaron K.

AU - Pyeritz, Reed E.

AU - Radojewski, Elizabeth A.

AU - Roman, Mary J.

AU - Pierpont, Mary Ella M

AU - Lacro, Ronald V.

AU - Pearson, Gail

AU - Stylianou, Mario

AU - Mahony, Lynn

AU - Sleeper, Lynn

AU - Tennstedt, Sharon

AU - Colan, Steven

AU - Klein, Gloria

AU - Guey, Lin

AU - Wruck, Lisa

AU - Travison, Thomas

AU - Chen, Shan

AU - Gerstenberger, Eric

AU - Olesker, Tanya

AU - Teitel, David F.

AU - Newburger, Jane

AU - King, Martha

AU - Dunbar-Masterson, Carolyn

AU - Posa, Andrea

AU - Nang, Quincy

AU - Hass, Cara

AU - Hsu, Daphne

AU - Lai, Wyman

AU - Hellenbrand, William

AU - Printz, Beth

N1 - Funding Information: Supported by grants from the Marfan Foundation and the Pediatric Heart Network Nursing Research Program, by U01 grants from the National Heart, Lung, and Blood Institute (HL068269, HL068270, HL068279, HL068281, HL068285, HL068292, HL068290, HL068288, and HL085057) and by the Food and Drug Administration Office of Orphan Products Development. The contents of this paper are solely the responsibility of the authors and do not necessarily represent the official views of the National Institutes of Health or the National Heart, Lung, and Blood Institute. The authors declare no conflicts of interest. Publisher Copyright: © 2018 Elsevier Inc.

PY - 2019/1

Y1 - 2019/1

N2 - 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.

AB - 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.

KW - neurodevelopmental disorders

KW - patient-reported symptoms

KW - Antihypertensive Agents/therapeutic use

KW - Severity of Illness Index

KW - Health Status Indicators

KW - Humans

KW - Child, Preschool

KW - Male

KW - Marfan Syndrome/complications

KW - Young Adult

KW - Adolescent

KW - Quality of Life

KW - Adult

KW - Female

KW - Losartan/therapeutic use

KW - Child

KW - Atenolol/therapeutic use

KW - Patient Reported Outcome Measures

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DO - 10.1016/j.jpeds.2018.08.061

M3 - Article

C2 - 30270167

AN - SCOPUS:85053937143

SN - 0022-3476

VL - 204

SP - 250-255.e1

JO - Journal of Pediatrics

JF - Journal of Pediatrics

ER -

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

Abstract

Bibliographical note

Keywords

PubMed: MeSH publication types

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