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

描述（由申请人提供）：2010年健康人的第一个目标是帮助所有年龄段的个人增加预期寿命，提高生活质量。患有囊性纤维化（CF）的个体，这是一种缩短寿命的慢性疾病，在美国影响约30，000人，患有慢性呼吸道症状。在CF患者中，急性症状恶化期（称为肺部急性加重）对生存率、生活质量和护理费用有重大影响。目前，只有当一个人寻求治疗时，才能发现肺部恶化。来自CF和慢性阻塞性肺疾病的数据表明，早期识别和干预可以更快地恢复，降低护理成本并改善结局。常规患者记录的呼吸健康观察可以促进这一点。该小型企业技术转让（STTR）应用的目标是：1）开发个性化电子疾病自我管理工具CF-ePRO，用于使用现有CF呼吸症状日记对7-11岁和12岁至成年患者的呼吸健康进行患者记录观察，以及2）为0至6岁的CF患者创建父母报告体征工具，为所有CF患者提供适合年龄的CF-ePRO版本。CF-ePRO将设计为可定制的，用于临床护理，并作为临床试验终点。该I期项目的具体目标是：1）生产CF-ePRO原型，用于任何可访问Web的智能手机或台式PC，2）对CF患者进行形成性实验室和现场可用性测试，3）与关键CF临床护理利益相关者进行形成性焦点小组，4）为0-6岁的CF患者创建父母报告的呼吸体征仪器，因为目前不存在用于该年龄组的仪器，5）根据可量化的基准评估概念验证和第二阶段准备情况。在第二阶段，我们建议通过增加常规家庭肺功能测定和咳嗽频率和强度的客观监测来增强CF-ePRO，并进行一项CF-ePRO监测与传统护理相比的随机对照试验。为了与STTR计划的意图保持一致，以刺激创新的小企业和非营利研究机构之间的想法和技术的伙伴关系，以创造可商业化的创新技术，我们已经组建了一个多学科的调查团队，从塔拉里亚公司，西雅图儿童医院和华盛顿大学。我们预计，CF-ePRO将是高度适销对路，极大的兴趣，CF患者和护理中心，以及学术和制药研究人员计划治疗试验。 公共卫生相关性：囊性纤维化（CF）是一种慢性肺部疾病，影响美国约30，000人，造成痛苦和过早死亡。一个帮助CF患者使用电脑或手机跟踪他们症状的系统可以帮助他们知道何时更快地寻求护理，反过来，活得更长，生活质量更好。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.