Developing and Evaluating Quality-Weighted Hospital-Free Days as a Novel, Patient-Centered Outcome for Trials of Patients with Acute Respiratory Failure

项目摘要/摘要 候选人：凯瑟琳·奥里玛，医学博士，是一名肺部和重症监护内科医生兼科学家，对 了解和改善急性呼吸衰竭(ARF)患者的长期预后。晋级 在她走向研究独立的职业生涯中，她寻求指导性和经验性的培训来发展专业知识 共识方法、前瞻性研究设计、统计建模和模拟。 研究背景：ARF发病率的上升和死亡率的下降导致 幸存者面临严重的身体、认知和社会障碍，需要更多的医疗保健 利用率。推进ARF生存研究的一个关键障碍是缺乏一种结果衡量标准，即 以患者为中心，适合评估具有姑息性或恢复性意图的干预措施。医院- 免费天数(HFDs)，即在急性护理医院活着和在医院外度过的天数，在 对其他危重人群的研究。然而，HFD不考虑功能状态的减少或 生活质量；而HFDs在ARF患者中的双峰分布引发了关于 HFD对ARF的统计能力和结构效度。本研究建议设计并评估一种方法 使用患者报告的对功能、生活质量或照顾者负担的评估来对HFD进行质量加权。 具体目标：1)使用协商一致的方法制定最佳的质量权重HFD方法 利益相关者；2)评估ARF患者预期队列中的质量加权HFD；3)比较 在一个大的ARF队列中，有质量加权和没有质量加权的高分辨FDs的统计能力和结构效度。 研究计划：为了实现这些目标，奥里马博士将召集一个由ARF幸存者、家属组成的德尔菲小组 护理人员、临床医生和试验人员开发集成患者和护理人员报告的数据的最佳实践 纳入对HFD的评估。然后，她将把这种新方法应用于ARF患者的预期队列中，以 评估质量加权HFD的可接受性、响应性和可变性。最后，她将使用观察到的 在ARF患者中进行的一项大型临床试验中模拟质量加权HFD的Aim 2队列中的值 比较未加权HFD和质量加权HFD的统计能力和结构效度。 职业发展计划：与她的导师和顾问密切合作，奥里玛博士将发展 在将患者确定的多个核心价值转化为可衡量的核心价值所需的共识方法方面的专长 结果；2)获得设计、启动、监督和完成多站点、预期目标所需的技能 队列研究；以及3)在高级统计建模和模拟技术方面获得方法论专业知识。 环境：宾夕法尼亚大学为从事这项培训提供了理想的环境，拥有良好的 知名导师和她的家庭部门致力于奥列马博士的成功，以及几个 多学科研究中心，拥有长期培养成功的独立调查人员的记录。

Project Summary/Abstract Candidate: Catherine Auriemma, MD, is a pulmonary and critical care physician-scientist passionate about understanding and improving long-term outcomes of patients with acute respiratory failure (ARF). To advance her career towards research independence, she seeks didactic and experiential training to develop expertise in consensus methods, prospective study design, and statistical modeling and simulation. Research Context: Rising incidence and decreasing mortality of ARF has produced a growing population of survivors facing significant physical, cognitive, and social impairments requiring increased healthcare utilization. A crucial barrier to advancing ARF survivorship research is the lack of an outcome measure that is patient-centered and appropriate for evaluating interventions with either palliative or restorative intent. Hospital- free days (HFDs), the number of days spent alive and outside an acute care hospital, has become common in studies of other acutely ill populations. However, HFDs does not account for decrements in functional status or quality of life; and the bimodal distribution of HFDs among ARF patients raises questions regarding the statistical power and construct validity of HFDs for ARF. This study proposes to design and evaluate a method to quality-weight HFDs using patient-reported assessments of function, quality of life, or caregiver burden. Specific Aims: 1) Develop an optimal approach to quality-weight HFDs using consensus methods with key stakeholders; 2) Assess quality-weighted HFDs among a prospective cohort of ARF patients; 3) Compare statistical power and construct validity of HFDs with and without quality-weighting in a large ARF cohort. Research Plan: To accomplish these aims, Dr. Auriemma will convene a Delphi panel of ARF survivors, family caregivers, clinicians, and trialists to develop best practices for integrating patient- and caregiver-reported data into assessments of HFDs. She will then apply the novel method to a prospective cohort of ARF patients to assess acceptability, responsiveness, and variability of quality-weighted HFDs. Finally, she will use observed values in the Aim 2 cohort to simulate quality-weighted HFDs in a large clinical trial among patients with ARF to compare statistical power and construct validity of unweighted and quality-weighted HFDs. Career Development Plan: Working closely with her mentors and advisors, Dr. Auriemma will 1) develop expertise in consensus methods necessary to convert multiple patient-identified core values into measurable outcomes; 2) obtain the skills necessary to design, launch, oversee, and complete a multisite, prospective cohort study; and 3) gain methodologic expertise in advanced statistical modeling and simulation techniques. Environment: The University of Pennsylvania offers an ideal environment to pursue this training, with well- established mentors and her home department heavily dedicated to Dr. Auriemma’s success, and several multi-disciplinary research centers with long track records of producing successful, independent investigators.