Early Intervention in Pulmonary Exacerbation in CF

描述（由申请人提供）：囊性纤维化（CF）患者每天都有呼吸道症状，如咳嗽和呼吸困难。尽管在护理方面有了巨大的改善，但由于进行性呼吸衰竭，他们的生命大大缩短。CF病程以复发性肺恶化为特征，表现为症状恶化和肺功能下降。CF急性肺加重导致肺功能更快下降和增高。我们小组的初步数据表明，延迟就医与急性发作后肺功能恢复不足有关。本提案的主要目的是进行一项临床试验，以确定早期干预治疗青少年和成人CF急性肺加重的疗效。该研究将评估两种治疗CF肺加重的新方法的整合：1)使用家庭肺活量计来识别和触发加重的治疗；2)使用基于家庭的患者症状评估来识别和触发肺恶化的治疗，两者都纳入一个协议化的护理计划。这种新方法将与常规治疗进行比较，主要结局指标是用第一秒用力呼气量（FEV1）测量肺功能。我们假设，如果肺部恶化的早期治疗与多方面的干预，肺部疾病的进展将会减慢。这种方法将利用最先进的远程保健设备，改进对患有CF的青少年和成人的评估和护理。

DESCRIPTION (provided by applicant): Individuals with cystic fibrosis (CF) are plagued by daily respiratory symptoms such as cough and dyspnea. In spite of dramatic improvements in care, their lives are greatly shortened due to progressive respiratory failure. The course of CF is marked by recurrent pulmonary exacerbations characterized by worsened symptoms and declines in pulmonary function. CF acute pulmonary exacerbations result in more rapid decline in lung function and increased. Preliminary data from our groups suggest that delays in seeking care are associated with lack of recovery of lung function after an exacerbation. The primary goal of this proposal is to perform a clinical trial to determine the efficacy of early intervention in the treatment of adolescent and adult CF acute pulmonary exacerbation. The study will assess the integration of two novel approaches to the treatment of CF pulmonary exacerbation: 1) the use of home spirometers to identify and trigger treatment of an exacerbation; and 2) the use of home based assessment of patient symptoms to identify and trigger the treatment of a pulmonary exacerbation, both integrated into one protocolized care plan. This novel approach will be compared to usual care with the primary outcome measure being lung function as measured with forced expiratory volume in the first second (FEV1). We hypothesize that if pulmonary exacerbations are treated earlier with a multi-faceted intervention, the progression of lung disease will be slowed. This approach will utilize state of the art, telehealth equipment to improve the assessment and delivery of care to adolescents and adults with CF. PUBLIC HEALTH RELEVANCE: The primary goal of this proposal is to perform a clinical trial to determine the efficacy of early intervention in the treatment of adolescent and adult CF acute pulmonary exacerbation using remote monitoring with home spirometry and respiratory symptom assessment and protocolized management of CF exacerbation. This approach will utilize state of the art, telehealth equipment to improve the assessment and delivery of care to adolescents and adults with CF.