The Child-Centered Outcomes in Practice and Research (COPR) Center of Excellence: Strengthening the Clinical Validity Evidence Base for PROMIS Measures in Chronically Ill Children

以儿童为中心的实践与研究成果 (COPR) 卓越中心：加强慢性病儿童 PROMIS 措施的临床有效性证据基础

基本信息

批准号：
9077845
负责人：
CHRISTOPHER B FORREST
金额：
$ 417万
依托单位：
CHILDREN'S HOSP OF PHILADELPHIA
依托单位国家：
美国
项目类别：
财政年份：
2015
资助国家：
美国
起止时间：
2015-09-30 至 2021-08-31
项目状态：
已结题

项目摘要

DESCRIPTION (provided by applicant): Patient-reported outcome (PROs) instruments complement conventional clinical outcomes by examining from the perspective of patients how they feel, what they perceive they can do, which types of activities they believe they can participate in, and how satisfied they are with their health and lives. To advance the science of PRO assessment, the NIH launched in 2004 the Patient Reported Outcome Measurement Information System (PROMIS(r)). Although a key objective of PROMIS is to provide PROs for research and clinical practice across the life course, there is remarkably little known about the validity of PROMIS for children with chronic diseases or the degree to which PROMIS detects clinically meaningful changes in children's health. To address these scientific needs, we propose to create the Child-Centered Outcomes in Practice and Research (COPR) Center of Excellence, which will be devoted to rigorously examining and documenting PROMIS' clinical validity for children with chronic conditions. COPR comprises three research projects focusing on pediatric Crohn's disease, chronic kidney disease, and cancer. The projects are linked by common measures of children's Experienced Well-Being (a concept that subsumes both suffering and happiness), clinical validation methods, and infrastructure. Each project will assess the content validity, clinical meaningfulness of scores, responsiveness to clinical change, and longitudinal construct validity of the PROMIS Pediatric pain interference, fatigue, psychological stress experiences, and positive affect measures. The COPR approach will increase the measures' psychometric and clinical maturation according to PROMIS standards and provide evidence required by the FDA to qualify PRO instruments for clinical trials. The COPR team integrates: a PROMIS research group that developed 10 of the 20 extant PROMIS pediatric measures and helped lead the PROMIS initiative over the past 6 years; a PCORI-funded clinical data research network (PEDSnet) with deep expertise in electronic health record and PRO data management, quality assessment and reporting; a national network of pediatric gastroenterologists embarking on a PCORI-funded clinical trial for children with Crohn's disease; a national network of pediatric nephrologists who are conducting an NIH-funded cohort study on children with chronic kidney disease; and, one of the nation's largest pediatric oncology clinical centers located at St. Jude Children's Research Hospital. The COPR infrastructure will: facilitate coordination across its three research projects; standardize clinicl validation methods and informatics resources; synthesize results across the three projects; develop standards-based instrument dossiers to enhance clinical adoption of PROMIS and facilitate FDA qualification of PROMIS measures as clinical trial end-points; and, support collaboration with other PEPR research programs. COPR is optimized to successfully accomplish its three research projects, and its infrastructure can be readily scaled to support the informatics, statistical, and administrative needs of multiple other PEPR research initiatives.

描述（由适用提供）：通过从患者的感受，认为他们可以做的事情，他们认为他们可以参与的活动以及对健康和生活的满意程度来检查患者报告的结果（PRO）仪器补充常规临床结果。为了推进专业评估的科学，NIH于2004年启动，患者报告了结果测量信息系统（Promis（r））。尽管Promis的关键目标是在整个生活课程中为研究和临床实践提供专业人士，但对Promis对慢性疾病儿童的有效性鲜为人知，或Promis检测到儿童健康中临床意义上有意义的变化的程度。为了满足这些科学需求，我们建议在实践和研究中以儿童为中心的成果（COPR）卓越中心，这些结果将致力于严格检查和记录Promis的临床有效性。 COPR包括三个研究项目，重点是儿科克罗恩病，慢性肾脏疾病和癌症。这些项目与儿童经验丰富的福祉（既包含苦难和幸福的概念），临床验证方法和基础设施的共同度量联系在一起。每个项目都将评估成分的内容有效性，分数的临床意义，对临床变化的反应以及Promis小儿疼痛干扰的纵向构建有效性，疲劳，心理压力经历和积极的情感措施。 COPR方法将根据Promis标准增加测量值的心理测量和临床成熟，并提供FDA要求的证据，以使PRO工具合格进行临床试验。 COPR团队集成了：一个Promis研究小组，开发了20种广泛的Promis小儿测量中的10个，并在过去6年中帮助领导了Promis倡议； PCORI资助的临床数据研究网络（PEDSNET），具有电子健康记录和专业数据管理，质量评估和报告的深度专家；一家国家的小儿胃肠病学家网络，正在针对克罗恩病儿童进行PCORI资助的临床试验；一项针对NIH资助的慢性肾脏病儿童的小儿肾病学家的国家网络；而且，位于圣裘德儿童研究医院的美国最大的儿科肿瘤学临床中心之一。 COPR基础设施将：促进其三个研究项目的协调；标准化临床验证方法和信息资源；在这三个项目中合成结果；开发基于标准的仪器档案，以增强对Promis的临床采用，并促进FDA资格作为临床试验终点的Promis测量值；并且，支持与其他PEPR研究计划的合作。 COPR经过优化以成功完成其三个研究项目，并且可以轻松地扩展其基础架构以支持其他多项PEPR研究计划的信息，统计和行政需求。