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

CF-ePRO：电子自我报告和家长报告的囊性纤维化患者结果

基本信息

批准号：
8060275
负责人：
Margaret Rosenfeld
金额：
$ 30.64万
依托单位：
TALARIA, INC.
依托单位国家：
美国
项目类别：
财政年份：
2011
资助国家：
美国
起止时间：
2011-09-01 至 2013-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8060275
关键词：
11 year old 12 year old 6 year old Acute Adolescent Adult Affect Age Age-Years Algorithms Benchmarking Businesses Caring Cellular Phone Cessation of life Chronic Chronic Disease Chronic Obstructive Airway Disease Chronic lung disease Clinic Clinical Trials Cognitive Computers Coughing Cystic Fibrosis Data Databases Detection Development Disease Early Diagnosis Early identification Early treatment Electronic Mail Electronics Evaluation Focus Groups Frequencies Goals Health Health Personnel Health Status Healthy People 2010 Home environment Indium Individual Industry Institution Intention Internet Intervention Interview Laboratories Lead Life Life Expectancy Living Costs Lung Measurement Measures Methods Monitor Nurses Online Systems Outcome Parents Participant Patient Care Patient Outcomes Assessments Patients Pediatric Hospitals Personal Computers Pharmacologic Substance Phase Physicians Provider Psychometrics Quality of life Questionnaires Randomized Controlled Trials Reaction Readiness Recovery Regimen Reporting Research Research Personnel Self Management Severities Small Business Technology Transfer Research Social Workers Spirometry Symptoms System Technology Technology Transfer Testing Text Therapeutic Intervention Therapy Clinical Trials Universities Validation Washington Work age group aged base care seeking children with cystic fibrosis clinical care cost cystic fibrosis patients design diaries improved innovation innovative technologies instrument interest meetings multidisciplinary premature programs prototype respiratory response tool trend usability

项目摘要

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.

描述（由申请人提供）：“健康人2010”的首要目标是帮助所有年龄段的人延长预期寿命并提高生活质量。囊性纤维化 (CF) 是一种缩短寿命的慢性疾病，在美国影响约 30,000 人，患有慢性呼吸道症状。急性症状恶化时期（称为肺部病情加重）对 CF 患者的生存、生活质量和护理费用产生重大影响。目前，只有当个人寻求治疗时才会发现肺部病情加重。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加快康复速度、降低护理成本并改善预后。患者对呼吸系统健康状况的常规观察记录可以促进这一点。该小型企业技术转让 (STTR) 应用程序的目标是 1) 开发个性化电子疾病自我管理工具 CF-ePRO，使用现有的 CF 呼吸道症状日记记录 7-11 岁患者的呼吸道健康状况2) 为 0 至 6 岁的 CF 患者创建家长报告的体征仪器，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。该第一阶段项目的具体目标是 1) 制作 CF-ePRO 原型，以与任何具有网络访问权限的智能手机或台式电脑配合使用，2) 对 CF 患者进行形成性实验室和现场可用性测试，3) 进行与关键 CF 临床护理利益相关者一起形成焦点小组，4) 为 0-6 岁 CF 患者创建家长报告呼吸体征仪器，因为目前该年龄组尚无此类仪器，5) 评估概念验证和 II 期准备情况根据可量化的基准。在第二阶段，我们建议通过增加常规家庭肺活量测定和咳嗽频率和强度的客观监测来增强 CF-ePRO，并与传统护理相比进行 CF-ePRO 监测的随机对照试验。 STTR 计划的目的是促进创新型小型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，我们组建了一支由来自 Talaria, Inc.、西雅图儿童医院的多学科研究人员组成的团队和华盛顿大学。我们预计 CF-ePRO 将具有很高的市场价值，引起 CF 患者和护理中心以及规划治疗试验的学术和药物研究人员的极大兴趣。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。一种帮助囊性纤维化患者使用电脑或手机跟踪症状的系统可以帮助他们知道何时尽早寻求护理，从而延长寿命并提高生活质量。我们建议建立并测试这样一个系统。