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: 1) a primary outcome that can be observed in a reasonable period of time; 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.

A Cross-Sectional Study of Exercise Performance During the First 2 Decades of Life After the Fontan Operation

“…Of 831, 644 (77%) were fully eligible to participate in the complete Fontan protocol, on the basis of their ability to be contacted and perform the necessary testing at participating centers. Details regarding the Pediatric Heart Network and the methods of the Fontan cross-sectional study have been reported previously (12). A total of 546 (85% of 644) children were enrolled; of these, 411 (75% of 546) underwent exercise testing.…”

“…Diastolic function was classified according to 2 systems: restrictive pattern present or absent, and diastolic dysfunction grade (20). Studies were evaluated at a core laboratory as described in the design article (12). Statistical analysis.…”

“…After consent, prospective maximal exercise testing was performed with a ramp protocol on an electronically braked cycle ergometer. Equipment was calibrated to manufacturers' specifications, and testing was performed with standard protocols previously used in children, adolescents, and subjects with Fontan physiology (7,(12)(13)(14). Resting pulmonary measurements.…”

Functional health status in children and adolescents after Fontan: comparison of generic and disease-specific assessments

“…2 Written informed consent or assent was obtained from all participants as approved by the institutional review committees at each of the seven North American institutions. Patients aged 6–18 years at enrolment and who underwent a Fontan procedure 6 months or earlier were included.…”

“…2 This study enroled 546 Fontan patients aged 6–18 years and included assessment of patient characteristics and medical history, the functional health status, and standardised assessment in terms of cardiopulmonary exercise testing, echocardiography, cardiac magnetic resonance imaging, and measurement of brain natriuretic peptide levels.…”