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.

项目摘要/摘要 候选人：医学博士Catherine Auriemma是一种肺和重症监护的身体科学家 了解和改善急性呼吸衰竭（ARF）患者的长期结局。前进 她朝着研究独立性的职业生涯，寻求教学和专家培训，以发展专业知识 共识方法，前瞻性研究设计以及统计建模和模拟。 研究环境：ARF的不断增长和降低的ARF死亡率已导致越来越多的人口 面临重大身体，认知和社会障碍的幸存者需要增加医疗保健 利用率。推进ARF生存研究的关键障碍是缺乏结果指标 以患者为中心，适合评估姑息或恢复意图的干预措施。医院- 免费的日子（HFD）是活着的天数和急诊医院以外的天数，已经很普遍 对其他急性病人群的研究。但是，HFD不能说明功能状态的减少或 生活质量； ARF患者中HFD的双峰分布提出了有关 HFDS对ARF的统计能力和构造有效性。这项研究建议设计和评估一种方法 使用患者报告的功能，生活质量或护理人员伯恩（Burnen）进行质量重量的HFD。 具体目的：1）使用与密钥的共识方法开发出最佳的质量重量HFD 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，HFD的统计功率和构造有效性。 研究计划：为了实现这些目标，Auriemma博士将召集ARF冲浪者的Delphi小组 护理人员，临床医生和审判者开发了整合患者和护理人员报告的数据的最佳实践 进入HFD的评估。然后，她将将新方法应用于前瞻性ARF患者队列 评估质量加权HFD的可接受性，响应能力和可变性。最后，她将使用观察到 AIM 2队列中的值在ARF患者的大型临床试验中模拟质量加权HFD 比较未加权和质量加权的HFD的统计能力和构造有效性。 职业发展计划：与她的导师和顾问紧密合作，Auriemma博士将开发 将多个患者识别的核心值转换为可测量的共识方法的专业知识 结果； 2）获得设计，启动，监督和完成多个潜在的潜在技能所需的技能 队列研究； 3）获得高级统计建模和仿真技术方面的方法论专业知识。 环境：宾夕法尼亚大学为接受这项培训提供了理想的环境， 建立的导师和她的内政部大量致力于Auriemma博士的成功，还有一些 多学科研究中心拥有生产成功的独立研究人员的长期记录。

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.