Noninvasive Ventilation in Children with Cystic Fibrosis

描述(申请人提供)：囊性纤维化(CF)是高加索人最常见的、缩短生命的遗传病。绝大多数患者死于心肺衰竭。尽管大多数患者现在都活到了成年早期，但三分之一的患者将在21岁生日之前死亡。无创正压通气(NIPPV)已被有效地用于治疗成人慢性肺病患者的呼吸衰竭。然而，目前还没有关于NIPPV用于延缓CF儿童慢性呼吸衰竭的发病或进展的对照前瞻性研究。我们假设早期无创正压通气干预可以改善囊性纤维化患者的日常功能和生活质量，并延缓呼吸衰竭的发生。具体地说，与对照组相比，接受NIPPV治疗的严重CF相关肺部疾病的儿童和青少年，我们预测：(1)呼吸功能改善，表现为肺功能下降速度减缓，运动耐力增强，呼吸肌耐力提高，清醒和睡眠时气体交换改善；(2)营养和代谢状况改善，体重增加，静息能量消耗减少；(3)生活质量改善，睡眠质量提高，肺部恶化次数减少。这项研究将首次使用前瞻性、随机、双盲设计来全面评估NIPPV治疗儿童CF患者的疗效。为了与RFA的使命保持一致，这项研究将使用多学科方法来改善慢性呼吸衰竭儿童的管理和提高生活质量。

DESCRIPTION (provided by applicant): Cystic fibrosis (CF) is the most common, life-shortening genetic disease in Caucasians. The vast majority of patients die from cardiorespiratory failure. Although most patients now live into early adulthood, a third will die before their 21st birthday. Noninvasive positive pressure ventilation (NIPPV) has been used effectively to treat respiratory failure in adults with chronic lung disease. However, there have been no controlled, prospective studies of the use of NIPPV in delaying the onset or progression of chronic respiratory failure in children with CF. We hypothesize that early intervention with noninvasive positive pressure ventilation improves daily function and quality of life, and delays the onset of respiratory failure, in patients with cystic fibrosis. Specifically, in children and adolescents with severe CF-related lung disease treated with NIPPV, compared to matched controls, we predict (1) An improvement in respiratory function, as evidenced by a slower rate of decline of pulmonary function tests, better exercise tolerance, improved ventilatory muscle endurance and improved gas exchange during wakefulness and sleep; (2) An improvement in nutritional and metabolic status, as evidenced by weight gain and a decreased resting energy expenditure; and (3) Improved quality of life, better quality of sleep and a decreased number of pulmonary exacerbations. This study will be the first to use a prospective, randomized, double-blinded design to comprehensively evaluate the effects of NIPPV in pediatric patients with CF. In keeping with the mission of the RFA, this study will use a multidisciplinary approach to improve management and enhance the quality of life in children with chronic respiratory failure.