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

描述（由申请人提供）：2010年健康人计划的第一个目标是帮助各个年龄段的人延长预期寿命，提高生活质量。囊性纤维化（CF）是一种缩短寿命的慢性疾病，在美国约有3万人患有此病，他们患有慢性呼吸道症状。急性症状恶化的时期，被称为肺恶化，对CF患者的生存、生活质量和护理成本有重大影响。目前，只有当个人寻求治疗时才能确定肺部恶化。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果。对病人呼吸健康的常规观察记录可以促进这一点。这项小型企业技术转移（STTR）应用的目标是：1)开发一种个性化的电子疾病自我管理工具CF- epro，用于使用现有的7-11岁和12岁至成年的CF呼吸症状日记记录患者的呼吸健康情况；2)为0 - 6岁的CF患者创建一种家长报告的体征工具，为所有CF患者提供适合年龄的CF- epro版本。CF-ePRO将被设计为可定制用于临床护理，并作为临床试验终点。这个第一阶段项目的具体目标是：1)制作CF- epro的原型，以便与任何具有网络访问功能的智能手机或台式电脑一起工作；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进行形成性的焦点小组；4)为0-6岁的CF患者创建家长报告的呼吸体征仪器，因为目前尚无针对该年龄组的仪器。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II期，我们建议通过增加常规的家庭肺量测定和咳嗽频率和强度的客观监测来增强CF-ePRO，并进行CF-ePRO监测与常规护理的随机对照试验。为了与STTR计划的意图保持一致，即促进创新小企业和非营利研究机构之间的思想和技术合作，以创造可商业化的创新技术，我们已经组建了一个由塔拉里亚公司、西雅图儿童医院和华盛顿大学组成的多学科研究小组。我们预计CF- epro将具有很高的市场价值，CF患者和护理中心以及计划治疗试验的学术和制药研究人员将对此非常感兴趣。

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.