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

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.

Grant Number: 5K23HL163402-04
NIH Institute/Center: NIH
Principal Investigator: Catherine Auriemma