Design of a large cross-sectional study to facilitate future clinical trials in children with the Fontan palliation

Lynn A. Sleeper, Page Anderson, Daphne T. Hsu, Lynn Mahony, Brian W. McCrindle, Stephen J. Roth, J. Phillip Saul, Richard V. Williams, Tal Geva, Steven D. Colan, Bernard J. Clark

Research output: Contribution to journalArticle

42 Citations (Scopus)

Abstract

Background: Clinical trials in children with congenital heart disease are often limited by the absence of the following: (1) a primary outcome that can be observed in a reasonable period; (2) information regarding health-related quality of life; (3) knowledge of the correlation between health status and ventricular function and exercise performance; (4) a sufficient number of children at a single institution to provide adequate statistical power; and (5) procedural and management differences between and within institutions. Methods: The NHLBI-funded Pediatric Heart Network designed a cross-sectional study of children aged 6 to 18 years, from 7 pediatric clinical centers, who had undergone a Fontan procedure as treatment for congenital heart disease. Health-related quality of life was measured by the Child Health Questionnaire and the Congenital Heart Adolescent and Teenager Questionnaire. Ventricular function was assessed by maximal exercise testing, echocardiography, cardiac magnetic resonance imaging, and B-type natriuretic peptide. The study was designed to detect a correlation of R ≥ 0.30 between health status scores and measures of ventricular function and performance in a subcohort with all study measures completed. Results: A total of 1078 children were screened by chart review; 644 (60%) were eligible. The consent rate was 85% and 546 children were enrolled. Acquisition of echocardiograms, B-type natriuretic peptide, and health status was ≥94%; completion rates were lower for maximal exercise testing (76%) and cardiac magnetic resonance imaging (41%). Conclusions: This large study provides unique information regarding the relationship between health status and clinical measures in post-Fontan patients that will facilitate the design of future randomized trials.

Original languageEnglish (US)
Pages (from-to)427-433
Number of pages7
JournalAmerican Heart Journal
Volume152
Issue number3
DOIs
StatePublished - Sep 2006

Fingerprint

Cross-Sectional Studies
Health Status
Clinical Trials
Ventricular Function
Brain Natriuretic Peptide
Exercise
Heart Diseases
Quality of Life
Magnetic Resonance Imaging
Pediatrics
National Heart, Lung, and Blood Institute (U.S.)
Fontan Procedure
Echocardiography
Surveys and Questionnaires
Therapeutics

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

Cite this

Design of a large cross-sectional study to facilitate future clinical trials in children with the Fontan palliation. / Sleeper, Lynn A.; Anderson, Page; T. Hsu, Daphne; Mahony, Lynn; McCrindle, Brian W.; Roth, Stephen J.; Saul, J. Phillip; Williams, Richard V.; Geva, Tal; Colan, Steven D.; Clark, Bernard J.

In: American Heart Journal, Vol. 152, No. 3, 09.2006, p. 427-433.

Research output: Contribution to journalArticle

Sleeper, LA, Anderson, P, T. Hsu, D, Mahony, L, McCrindle, BW, Roth, SJ, Saul, JP, Williams, RV, Geva, T, Colan, SD & Clark, BJ 2006, 'Design of a large cross-sectional study to facilitate future clinical trials in children with the Fontan palliation', American Heart Journal, vol. 152, no. 3, pp. 427-433. https://doi.org/10.1016/j.ahj.2006.02.009
Sleeper, Lynn A. ; Anderson, Page ; T. Hsu, Daphne ; Mahony, Lynn ; McCrindle, Brian W. ; Roth, Stephen J. ; Saul, J. Phillip ; Williams, Richard V. ; Geva, Tal ; Colan, Steven D. ; Clark, Bernard J. / Design of a large cross-sectional study to facilitate future clinical trials in children with the Fontan palliation. In: American Heart Journal. 2006 ; Vol. 152, No. 3. pp. 427-433.
@article{8060ab5ce4274ce39e788b6cd315fd88,
title = "Design of a large cross-sectional study to facilitate future clinical trials in children with the Fontan palliation",
abstract = "Background: Clinical trials in children with congenital heart disease are often limited by the absence of the following: (1) a primary outcome that can be observed in a reasonable period; (2) information regarding health-related quality of life; (3) knowledge of the correlation between health status and ventricular function and exercise performance; (4) a sufficient number of children at a single institution to provide adequate statistical power; and (5) procedural and management differences between and within institutions. Methods: The NHLBI-funded Pediatric Heart Network designed a cross-sectional study of children aged 6 to 18 years, from 7 pediatric clinical centers, who had undergone a Fontan procedure as treatment for congenital heart disease. Health-related quality of life was measured by the Child Health Questionnaire and the Congenital Heart Adolescent and Teenager Questionnaire. Ventricular function was assessed by maximal exercise testing, echocardiography, cardiac magnetic resonance imaging, and B-type natriuretic peptide. The study was designed to detect a correlation of R ≥ 0.30 between health status scores and measures of ventricular function and performance in a subcohort with all study measures completed. Results: A total of 1078 children were screened by chart review; 644 (60{\%}) were eligible. The consent rate was 85{\%} and 546 children were enrolled. Acquisition of echocardiograms, B-type natriuretic peptide, and health status was ≥94{\%}; completion rates were lower for maximal exercise testing (76{\%}) and cardiac magnetic resonance imaging (41{\%}). Conclusions: This large study provides unique information regarding the relationship between health status and clinical measures in post-Fontan patients that will facilitate the design of future randomized trials.",
author = "Sleeper, {Lynn A.} and Page Anderson and {T. Hsu}, Daphne and Lynn Mahony and McCrindle, {Brian W.} and Roth, {Stephen J.} and Saul, {J. Phillip} and Williams, {Richard V.} and Tal Geva and Colan, {Steven D.} and Clark, {Bernard J.}",
year = "2006",
month = "9",
doi = "10.1016/j.ahj.2006.02.009",
language = "English (US)",
volume = "152",
pages = "427--433",
journal = "American Heart Journal",
issn = "0002-8703",
publisher = "Mosby Inc.",
number = "3",

}

TY - JOUR

T1 - Design of a large cross-sectional study to facilitate future clinical trials in children with the Fontan palliation

AU - Sleeper, Lynn A.

AU - Anderson, Page

AU - T. Hsu, Daphne

AU - Mahony, Lynn

AU - McCrindle, Brian W.

AU - Roth, Stephen J.

AU - Saul, J. Phillip

AU - Williams, Richard V.

AU - Geva, Tal

AU - Colan, Steven D.

AU - Clark, Bernard J.

PY - 2006/9

Y1 - 2006/9

N2 - Background: Clinical trials in children with congenital heart disease are often limited by the absence of the following: (1) a primary outcome that can be observed in a reasonable period; (2) information regarding health-related quality of life; (3) knowledge of the correlation between health status and ventricular function and exercise performance; (4) a sufficient number of children at a single institution to provide adequate statistical power; and (5) procedural and management differences between and within institutions. Methods: The NHLBI-funded Pediatric Heart Network designed a cross-sectional study of children aged 6 to 18 years, from 7 pediatric clinical centers, who had undergone a Fontan procedure as treatment for congenital heart disease. Health-related quality of life was measured by the Child Health Questionnaire and the Congenital Heart Adolescent and Teenager Questionnaire. Ventricular function was assessed by maximal exercise testing, echocardiography, cardiac magnetic resonance imaging, and B-type natriuretic peptide. The study was designed to detect a correlation of R ≥ 0.30 between health status scores and measures of ventricular function and performance in a subcohort with all study measures completed. Results: A total of 1078 children were screened by chart review; 644 (60%) were eligible. The consent rate was 85% and 546 children were enrolled. Acquisition of echocardiograms, B-type natriuretic peptide, and health status was ≥94%; completion rates were lower for maximal exercise testing (76%) and cardiac magnetic resonance imaging (41%). Conclusions: This large study provides unique information regarding the relationship between health status and clinical measures in post-Fontan patients that will facilitate the design of future randomized trials.

AB - Background: Clinical trials in children with congenital heart disease are often limited by the absence of the following: (1) a primary outcome that can be observed in a reasonable period; (2) information regarding health-related quality of life; (3) knowledge of the correlation between health status and ventricular function and exercise performance; (4) a sufficient number of children at a single institution to provide adequate statistical power; and (5) procedural and management differences between and within institutions. Methods: The NHLBI-funded Pediatric Heart Network designed a cross-sectional study of children aged 6 to 18 years, from 7 pediatric clinical centers, who had undergone a Fontan procedure as treatment for congenital heart disease. Health-related quality of life was measured by the Child Health Questionnaire and the Congenital Heart Adolescent and Teenager Questionnaire. Ventricular function was assessed by maximal exercise testing, echocardiography, cardiac magnetic resonance imaging, and B-type natriuretic peptide. The study was designed to detect a correlation of R ≥ 0.30 between health status scores and measures of ventricular function and performance in a subcohort with all study measures completed. Results: A total of 1078 children were screened by chart review; 644 (60%) were eligible. The consent rate was 85% and 546 children were enrolled. Acquisition of echocardiograms, B-type natriuretic peptide, and health status was ≥94%; completion rates were lower for maximal exercise testing (76%) and cardiac magnetic resonance imaging (41%). Conclusions: This large study provides unique information regarding the relationship between health status and clinical measures in post-Fontan patients that will facilitate the design of future randomized trials.

UR - http://www.scopus.com/inward/record.url?scp=33747177409&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=33747177409&partnerID=8YFLogxK

U2 - 10.1016/j.ahj.2006.02.009

DO - 10.1016/j.ahj.2006.02.009

M3 - Article

VL - 152

SP - 427

EP - 433

JO - American Heart Journal

JF - American Heart Journal

SN - 0002-8703

IS - 3

ER -