Q&A: Clinical outcomes in CVD treatment ‘may not reflect what patients truly value’
There has been much discussion about patient-centered outcomes in CVD therapy and treatment, but little consensus exists as to their definition and value.
In a study published in JAMA Network Open, researchers, along with patients, caregivers and clinicians, worked together to define five patient-defined adverse CV and non-CV events (PACE) that may form a meaningful composite outcome from a patient perspective for use in clinical practice and future CV trials.
“Because patients with advanced cardiovascular disease are likely to be elderly with substantial comorbidities, they are more often concerned with how treatment might impact their ability to lead an independent life with less impairment and outside of chronic care institutions,” Louise Sun, MD, SM, FRCPC, FAHA, cardiac anesthesiologist, clinician scientist and director of big data and health bioinformatics research at the University of Ottawa Heart Institute and associate professor of anesthesiology and epidemiology and tier II clinical research chair in big data and cardiovascular outcomes at the University of Ottawa, and colleagues wrote.
For this pan-Canadian, consensus-based, qualitative study, researchers collaborated with 11 clinicians and 24 patients with CVD and caregivers (88% patients; 54% men) to participate in several rounds of questionnaires designed to narrow the isolate PACE to five individual outcomes.
The clinicians included three cardiac surgeons, one cardiac anesthesiologist, one cardiac surgical intensivist, one interventional and three noninterventional cardiologists, one clinical health psychologist and one registered nurse specializing in the care of cardiac patients. Patients were included based on the diagnosis of CVD treated with medical therapy and/or cardiac surgery and/or catheter-based procedure, left ventricular assist device implantation or heart transplantation.
Healio spoke with Sun about the composite PACE outcome and what it may mean for clinical practice and research.
Question: What was the motivation to pursue this research?
Sun: CV research has traditionally been focused on “tombstone” outcomes such as death and major adverse CV events. To date, little attention has been paid to outcomes that are most relevant from the patient’s perspective. Many so-called patient-centered metrics are defined by clinicians alone and may not reflect what patients truly value. We previously derived and validated “disability-free survival” as a patient-defined outcome after cardiac surgery. We in this paper refined the disability outcome by supplementing patients’ perspectives with those of caregivers and clinicians, and recoined this “PACE.”
Q: Could you briefly explain the study methodology?
Sun: We followed a modified Delphi approach, through iterative questionnaires followed by an in-person meeting. This was done through a national panel of patients with lived experience, their caregivers and a multidisciplinary team of clinicians. Together, the panel narrowed down the definition of PACE into a five-item list.
Q: What is the primary takeaway from these results?
Sun: PACE is a five-item composite outcome, consisting of: severe stroke necessitating hospitalization for 14 days or more or inpatient rehabilitation, ventilator dependence, new-onset or worsening HF, nursing home admission and new-onset dialysis.
Q: Why are these results significant?
Sun: PACE is versatile with administrative databases as well as prospectively collected data. It can readily be applied as a patient-centered outcome in interventional clinical trials and large epidemiological studies to ensure management of CVD is founded on outcomes that are important and relevant to patients, caregivers and clinicians.
Q: Is there any further research needed or planned to validate PACE?
Sun: Studies are currently underway to describe the epidemiology of PACE in the general and medical/surgical populations, as well as to derive and validate personalized PACE risk prediction indices in a variety of clinical settings.
For more information:
Louise Sun, MD, SM, FRCPC, FAHA, can be reached at firstname.lastname@example.org.