Design of a large cross-sectional study to facilitate future clinical trials in children with the Fontan palliation
Design of a large cross-sectional study to facilitate future clinical trials in children with the Fontan palliation
复制标题
DOI：
10.1016/j.ahj.2006.02.009
复制
发表时间：
2006-09-01
影响因子：
4.8
通讯作者：
Clark, Bernard J.
中科院分区：
文献类型：
--
作者：
Sleeper, Lynn A.;Anderson, Page;Clark, Bernard J.
背景先天性心脏病儿童的临床试验通常因缺乏以下内容而受到限制：（1）在合理的时间内可以观察到的主要结局;（2）与健康相关的生活质量信息;（3）健康状况与心室功能和运动能力之间的相关性知识;（4）一个机构有足够的儿童人数，以提供足够的统计能力;和（5）机构之间和机构内部的程序和管理差异。来自7个儿科临床中心的6 - 18岁儿童的横断面研究，这些儿童接受了Fontan手术作为先天性心脏病的治疗。健康相关的生活质量通过儿童健康问卷和先天性心脏病青少年问卷进行测量。通过最大运动试验、超声心动图、心脏磁共振成像和B型利钠肽评估心室功能。该研究的目的是检测R >= 0.30之间的相关性的健康状况评分和措施的心室功能和性能在一个子队列与所有的研究measurement.Results共筛选了1078名儿童的图表审查，644（60%）是合格的。同意率为85%，546名儿童入组。超声心动图、B型利钠肽和健康状况的采集率>= 94%;最大运动试验（76%）和心脏磁共振成像（41%）的完成率较低。结论这项大型研究提供了关于Fontan术后患者健康状况和临床措施之间关系的独特信息，这将有助于未来随机试验的设计。
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.