Noninvasive Ventilation in Children with Cystic Fibrosis

描述（由申请人提供）：囊性纤维化（CF）是高加索人中最常见的缩短寿命的遗传性疾病。绝大多数患者死于心肺功能衰竭。虽然大多数患者现在都能活到成年早期，但三分之一的患者将在21岁生日前死亡。无创正压通气（NIPPV）已被有效地用于治疗慢性肺病成人呼吸衰竭。然而，目前还没有关于使用NIPPV延缓CF儿童慢性呼吸衰竭发作或进展的对照、前瞻性研究。我们假设，早期无创正压通气干预可改善囊性纤维化患者的日常功能和生活质量，并延迟呼吸衰竭的发生。具体而言，在接受NIPPV治疗的重度CF相关肺病儿童和青少年中，与匹配的对照组相比，我们预测（1）呼吸功能改善，表现为肺功能测试下降速度较慢，运动耐量更好，呼吸肌耐力改善，清醒和睡眠期间气体交换改善;（2）营养和代谢状态改善，如体重增加和静息能量消耗减少所证明的;和（3）生活质量改善，睡眠质量更好，肺部恶化次数减少。本研究将首次采用前瞻性、随机、双盲设计，全面评价无创正压通气（NIPPV）治疗儿童CF患者的疗效。为了与RFA的使命保持一致，本研究将采用多学科方法来改善慢性呼吸衰竭儿童的管理并提高其生活质量。

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.