CF-ePRO: Electronic Self- and Parent-Reported Patient Outcomes in Cystic Fibrosis

描述(申请人提供)：2010年健康人的第一个目标是帮助所有年龄段的人延长预期寿命和改善他们的生活质量。囊性纤维化(CF)是一种在美国影响约30,000人的缩短寿命的慢性疾病，患者患有慢性呼吸道症状。急性症状恶化的时期，称为肺恶化，对CF患者的生存、生活质量和护理费用有重大影响。目前，只有在个人寻求治疗时才能确定肺部恶化。来自慢性阻塞性肺疾病和慢性阻塞性肺疾病的数据表明，早期识别和干预会导致更快的恢复，降低护理成本，并改善结果。常规的患者记录的呼吸健康观察可以促进这一点。这一小型企业技术转让(STTR)应用程序的目标是：1)开发个性化的电子疾病自我管理工具CF-ePRO，使用7-11岁和12岁至成人的患者现有的CF-ePRO呼吸症状日记，记录患者的呼吸健康状况；2)为0-6岁的CF患者创建父母报告的体征工具，使所有CF患者能够使用适合年龄的CF-ePRO版本。Cf-ePRO将设计为可定制用于临床护理，并作为临床试验终点。此第一阶段项目的具体目标是：1)生产一个可与任何智能手机或可访问网络的台式PC配合使用的CF-ePRO原型；2)对CF患者进行形成性的实验室和现场可用性测试；3)与关键的CF临床护理利益相关者一起开展形成焦点小组；4)为0-6岁的CF患者创建家长报告呼吸体征仪器，因为这个年龄段目前还没有这种仪器；以及5)根据可量化的基准评估概念验证和第二阶段的就绪性。在第二阶段，我们建议通过增加常规的家庭肺活量测定和对咳嗽频率和强度的客观监测来增强CF-ePRO，并进行一项与传统护理相比的CF-ePRO监测的随机对照试验。根据科技创新研究计划旨在促进创新小企业和非营利性研究机构之间的创意和技术合作以创造可商业化的创新技术的意图，我们组建了一个由来自Talaria，Inc.、西雅图儿童医院和华盛顿大学的多学科研究人员组成的团队。我们预计，CF-ePRO将非常畅销，将引起CF患者和护理中心以及计划进行治疗试验的学术和制药研究人员的极大兴趣。 与公共卫生相关：囊性纤维化(CF)是一种慢性肺部疾病，在美国约有30,000人受到影响，导致痛苦和过早死亡。一种帮助CF患者使用电脑或手机跟踪症状的系统，可以帮助他们更早地知道何时寻求治疗，进而延长寿命，提高生活质量。我们建议建立和测试这样一个系统。

DESCRIPTION (provided by applicant): The first goal of Healthy People 2010 is to help individuals of all ages increase life expectancy and improve their quality of life. Individuals with cystic fibrosis (CF), a life-shortening chronic disease affecting ~30,000 individuals in the U.S., suffer from chronic respiratory symptoms. Periods of acute symptom worsening, known as pulmonary exacerbations, have a major impact on survival, quality of life, and cost of care among CF patients. Currently, pulmonary exacerbations are identified only when an individual seeks care. Data from CF and chronic obstructive pulmonary disease suggest that earlier identification and intervention lead to more rapid recovery, reduced cost of care, and improved outcomes. Routine patient-recorded observations of respiratory health could facilitate this. The objectives of this small business technology transfer (STTR) application are 1) to develop a personalized electronic disease self- management tool, CF-ePRO, for patient-recorded observations of respiratory health using existing CF respiratory symptom diaries for patients aged 7-11 years and 12 years to adulthood, and 2) to create a parent-reported sign instrument for CF patients 0 to 6 years of age, enabling age-appropriate versions of the CF-ePRO for all CF patients. CF-ePRO will be designed to be customizable for use in clinical care and to serve as a clinical trial endpoint. The specific aims of this Phase I project are to 1) produce a prototype of CF-ePRO to work with any smartphone or desktop PC with web access, 2) conduct formative lab- and field-based usability testing with CF patients, 3) conduct formative focus groups with key CF clinical care stakeholders, 4) create a parent-report respiratory sign instrument for CF patients 0-6 years old as none currently exists for this age group, and 5) evaluate proof-of-concept and Phase II readiness according to quantifiable benchmarks. In Phase II, we propose to enhance the CF-ePRO by adding routine home spirometry and objective monitoring of cough frequency and intensity and to conduct a randomized controlled trial of CF-ePRO monitoring compared to conventional care. In keeping with the intention of the STTR program to stimulate a partnership of ideas and technologies between innovative small businesses and non-profit research institutions to create commercializable innovative technologies, we have assembled a multidisciplinary team of investigators from Talaria, Inc., Seattle Children's Hospital, and the University of Washington. We anticipate that CF-ePRO will be highly marketable, of great interest to CF patients and care centers as well as to academic and pharmaceutical researchers planning therapeutic trials. PUBLIC HEALTH RELEVANCE: Cystic fibrosis (CF) is a chronic lung disease that affects about 30,000 people in the U.S., causing suffering and premature death. A system to help CF patients to track their symptoms, using computers or cell phones, could help them know when to seek care sooner and, in turn, live longer with a better quality of life. We propose to build and test such a system.