Department of Pediatrics, Division of Pediatric Cardiology, Stanford University, School of Medicine, Palo Alto, CA, USA.
Department of Medicine, Stanford University, School of Medicine, Palo Alto, CA, USA.
Am Heart J. 2023 May;259:68-78. doi: 10.1016/j.ahj.2023.02.006. Epub 2023 Feb 14.
In this manuscript, we describe the design and rationale of a randomized controlled trial in pediatric Fontan patients to test the hypothesis that a live-video-supervised exercise (aerobic+resistance) intervention will improve cardiac and physical capacity; muscle mass, strength, and function; and endothelial function. Survival of children with single ventricles beyond the neonatal period has increased dramatically with the staged Fontan palliation. Yet, long-term morbidity remains high. By age 40, 50% of Fontan patients will have died or undergone heart transplantation. Factors that contribute to onset and progression of heart failure in Fontan patients remain incompletely understood. However, it is established that Fontan patients have poor exercise capacity which is associated with a greater risk of morbidity and mortality. Furthermore, decreased muscle mass, abnormal muscle function, and endothelial dysfunction in this patient population is known to contribute to disease progression. In adult patients with 2 ventricles and heart failure, reduced exercise capacity, muscle mass, and muscle strength are powerful predictors of poor outcomes, and exercise interventions can not only improve exercise capacity and muscle mass, but also reverse endothelial dysfunction. Despite these known benefits of exercise, pediatric Fontan patients do not exercise routinely due to their chronic condition, perceived restrictions to exercise, and parental overprotection. Limited exercise interventions in children with congenital heart disease have demonstrated that exercise is safe and effective; however, these studies have been conducted in small, heterogeneous groups, and most had few Fontan patients. Critically, adherence is a major limitation in pediatric exercise interventions delivered on-site, with adherence rates as low as 10%, due to distance from site, transportation difficulties, and missed school or workdays. To overcome these challenges, we utilize live-video conferencing to deliver the supervised exercise sessions. Our multidisciplinary team of experts will assess the effectiveness of a live-video-supervised exercise intervention, rigorously designed to maximize adherence, and improve key and novel measures of health in pediatric Fontan patients associated with poor long-term outcomes. Our ultimate goal is the translation of this model to clinical application as an "exercise prescription" to intervene early in pediatric Fontan patients and decrease long-term morbidity and mortality.
在本手稿中，我们描述了一项针对儿科 Fontan 患者的随机对照试验的设计和原理，以检验以下假设：即现场视频监督下的运动（有氧运动+阻力运动）干预将改善心脏和身体能力；肌肉质量、力量和功能；以及内皮功能。随着分期 Fontan 姑息疗法的发展，患有单心室的儿童在新生儿期后的存活率大大提高。然而，长期发病率仍然很高。到 40 岁时，50%的 Fontan 患者将会死亡或接受心脏移植。导致 Fontan 患者心力衰竭发作和进展的因素仍不完全清楚。然而，已经确定 Fontan 患者的运动能力较差，这与发病率和死亡率增加有关。此外，该患者群体的肌肉质量减少、肌肉功能异常和内皮功能障碍已知会导致疾病进展。在患有 2 个心室和心力衰竭的成年患者中，运动能力、肌肉质量和肌肉力量的降低是预后不良的有力预测因素，运动干预不仅可以提高运动能力和肌肉质量，还可以逆转内皮功能障碍。尽管运动有这些已知的益处，但由于患儿的慢性疾病、对运动的认知限制和父母的过度保护，儿科 Fontan 患者通常不运动。在患有先天性心脏病的儿童中进行的有限运动干预表明，运动是安全有效的；然而，这些研究是在小而不同质的群体中进行的，而且大多数 Fontan 患者人数较少。关键的是，由于距离治疗场所远、交通困难以及错过上学或工作日，现场提供的儿科运动干预的依从性是一个主要的限制因素，依从率低至 10%。为了克服这些挑战，我们利用现场视频会议来提供监督下的运动课程。我们的多学科专家团队将评估现场视频监督下的运动干预的有效性，该干预方案经过精心设计，最大限度地提高依从性，并改善与不良长期预后相关的儿科 Fontan 患者的关键和新颖的健康指标。我们的最终目标是将这种模式转化为临床应用，作为一种“运动处方”，以便早期干预儿科 Fontan 患者，降低长期发病率和死亡率。
