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

制定和评估质量加权的非住院日作为急性呼吸衰竭患者试验的一种新颖的、以患者为中心的结果

• 批准号: 10676728
• 负责人: Catherine Louise Auriemma
• 金额: $ 19.24万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-08-05 至 2027-07-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10676728
• 关键词: Acute; Acute respiratory failure; Admission activity; Adult; American; Career Mobility; Caregiver Burden; Caregiver research; Caregivers; Clinical Trials; Cognitive; Cohort Studies; Consensus; Critical Care; Critical Illness; Data; Data Reporting; Dedications; Development Plans; Electronic Health Record; Enrollment; Environment; Family; Family Caregiver; Family member; Financial Support; Frequencies; Future; Goals; Health; Home; Hospital Mortality; Hospitals; Impaired health; Impairment; Incidence; Institution; Intensive Care Units; Interdisciplinary Study; International; Intervention; K-Series Research Career Programs; Knowledge; Lead; Lung; Measures; Mentors; Methodology; Methods; Modality; National Heart, Lung, and Blood Institute; Outcome; Outcome Measure; Patient Outcomes Assessments; Patient-Focused Outcomes; Patients; Pennsylvania; Physicians; Population; Positioning Attribute; Property; Prospective Studies; Prospective cohort; Prospective, cohort study; Psychometrics; Public Health; Quality of Life Assessment; Quality of life; Records; Reporting; Research; Research Design; Research Personnel; Sample Size; Scholarship; Science; Scientist; Site; Statistical Models; Structure; Surveys; Survivors; Techniques; Training; Translating; Universities; Weight; Work; acute care; career development; cohort; design; digital; evidence base; experience; follow-up; functional status; health care service utilization; health goals; hospital care; hospital readmission; improved; innovation; instrument; models and simulation; mortality; novel; palliative; patient engagement; patient oriented; prospective; psychosocial; randomized trial; recruit; response; skills; social; success; survivorship

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.

项目概要/摘要 候选人：Catherine Auriemma，医学博士，是一位肺科和重症监护医师兼科学家，热衷于 了解并改善急性呼吸衰竭（ARF）患者的长期预后。 她的职业生涯致力于研究独立，她寻求教学和体验式培训来发展专业知识 共识方法、前瞻性研究设计以及统计建模和模拟。 研究背景：ARF 发病率上升和死亡率下降导致 ARF 人口不断增长 幸存者面临严重的身体、认知和社交障碍，需要加强医疗保健 推进 ARF 生存研究的一个关键障碍是缺乏结果衡量标准。 以患者为中心，适合评估姑息性或恢复性干预措施。 空闲天数（HFD），即在急症护理医院外活着的天数，在 然而，HFD 并不能解释功能状态或功能下降的情况。 生活质量；ARF 患者中 HFD 的双峰分布引发了有关 本研究提出设计和评估 ARF 的 HFD 的统计功效和构造有效性。 使用患者报告的功能、生活质量或护理人员负担的评估来评估质量权重 HFD。 具体目标： 1) 使用关键的共识方法开发质量权重 HFD 的最佳方法 利益相关者；2) 评估 ARF 患者前瞻性队列中的质量加权 HFD；3) 比较 在大型 ARF 队列中，有或没有质量加权的 HFD 的统计功效和结构有效性。 研究计划：为了实现这些目标，Auriemma 博士将召集由 ARF 幸存者、家人组成的德尔福小组 护理人员、牧师和试验人员最好地开发整合患者和护理人员报告数据的实践 然后，她将把这种新方法应用于 ARF 患者的前瞻性队列中，以评估 HFD 的情况。 最后，她将使用观察到的质量加权 HFD 来评估可接受性、响应性和可变性。 Aim 2 队列中的值用于模拟 ARF 患者的大型临床试验中的质量加权 HFD 比较未加权和质量加权 HFD 的统计功效和结构有效性。 职业发展计划：Auriemma 博士与她的导师和顾问密切合作，将 1) 发展 将多个患者确定的核心价值观转化为可衡量的共识方法方面的专业知识 成果；2) 获得设计、启动、监督和完成多站点、前瞻性项目所需的技能。 队列研究；3) 获得先进统计建模和模拟技术的方法学专业知识。 环境：宾夕法尼亚大学提供了进行这种培训的理想环境，拥有良好的 已建立的导师和她的家庭部门大力致力于 Auriemma 博士的成功，还有几位 多学科研究中心，长期培养成功的独立研究人员。

项目成果

"It's that invisible illness": Patient and clinician perspectives on outcomes in pulmonary arterial hypertension treatment.

• DOI: 10.1002/pul2.12236
• 发表时间: 2023-04
• 期刊: PULMONARY CIRCULATION
• 影响因子: 2.6
• 作者: Auriemma, Catherine L. L.;Minhas, Jasleen;Blue, Randi;Lapatra, Tess;Kawut, Steven M. M.;Courtright, Katherine R. R.
• 通讯作者: Courtright, Katherine R. R.