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 > or = 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 > or = 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.
authors.affiliation: New England Research Institutes, Watertown, MA 02472, USA. lsleeper@neriscience.com
authors.forename: Lynn A
authors.initials: LA
authors.lastname: Sleeper
authors.forename: Page
authors.initials: P
authors.lastname: Anderson
authors.forename: Daphne T
authors.initials: DT
authors.lastname: Hsu
authors.forename: Lynn
authors.initials: L
authors.lastname: Mahony
authors.forename: Brian W
authors.initials: BW
authors.lastname: McCrindle
authors.forename: Stephen J
authors.initials: SJ
authors.lastname: Roth
authors.forename: J Phillip
authors.initials: JP
authors.lastname: Saul
authors.forename: Richard V
authors.initials: RV
authors.lastname: Williams
authors.forename: Tal
authors.initials: T
authors.lastname: Geva
authors.forename: Steven D
authors.initials: SD
authors.lastname: Colan
authors.forename: Bernard J
authors.initials: BJ
authors.lastname: Clark
mesh_terms: D000293:Adolescent; D002648:Child; D002986:Clinical Trials as Topic / Q000379:methods* / Q000639:trends; D003430:Cross-Sectional Studies; D005260:Female; D018729:Fontan Procedure / Q000706:statistics & numerical data* / Q000639:trends; D006330:Heart Defects, Congenital / Q000453:epidemiology* / Q000601:surgery; D006801:Humans; D008279:Magnetic Resonance Imaging / Q000379:methods / Q000639:trends; D008297:Male; D015337:Multicenter Studies as Topic / Q000379:methods / Q000639:trends; D010166:Palliative Care / Q000379:methods* / Q000639:trends; D011788:Quality of Life; D016032:Randomized Controlled Trials as Topic / Q000379:methods / Q000639:trends; D011795:Surveys and Questionnaires