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

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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.