Quantifying clinical change: discrepancies between patients’ and providers’ perspectives
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Interpreting the clinical significance of changes in patient-reported outcomes (PROs) is critically important. The most commonly used approach is to anchor mean changes on PRO scores against a global assessment of change. Whether the assessor of global change should be patients or their physicians is unknown. We compared patients’ and physicians’ assessments of change over time to examine which was more aligned with patients’ changes in PRO measures.
A total of 459 chronic heart failure patients aged >30 years were enrolled from 13 US centers. Data were obtained by medical record abstraction, physical assessments, and patient interviews at a baseline clinic visit and 6 weeks later. Health status was measured with the disease-specific Kansas City Cardiomyopathy Questionnaire (KCCQ), and both patients and physicians completed a validated 15-level global assessment of change, ranging from large deterioration to large improvement.
There was substantial variation between physicians/patients’ global assessment of clinical change (weighted kappa = 0.36, 95 % CI 0.28, 0.43). Overall, physician assessments were more strongly correlated with change on the KCCQ summary score than were patients’ assessments (physician R = 0.37, patient R = 0.29).
There was substantial variation between patients’ and physicians’ global assessment of 6-week change in heart failure status. Physician assessments of the importance of clinical changes were more strongly associated with changes in all domains of patient-reported health status, as assessed by the KCCQ, and may provide a more consistent method for defining the clinical importance of changes in patients’ health status.
KeywordsPatient-reported outcome measures Heart failure Kansas City Cardiomyopathy Questionnaire Clinical change
Compliance with ethical standards
Conflict of interest
Dr. Spertus is supported by grants from Gilead, Genentech, Lilly, Amorcyte, and EvaHeart, and has a copyright for the Kansas City Cardiomyopathy Questionnaire. Dr. Kutty is supported by a grant from the American Heart Association. All other authors report no conflict of interest.
All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Declaration of Helsinki and its later amendments or comparable ethical standards.
Informed consent was obtained from all individual participants included in the study.
- 3.Gliklich, R. E., Dreyer, N., Leavy, MB (Eds.). (2014). Use of patient-reported outcomes in registries. In I. Prepared by the Outcome DEcIDE Center Outcome Sciences, a Quintiles & c. u. C. N. TO7 (Eds.), Registries for evaluating patient outcomes: A user’s guide (3rd ed., Vol. 5). Rockville, MD: Agency for Healthcare Research and Quality (US).Google Scholar
- 4.Rumsfeld, J. S., Alexander, K. P., Goff, D. C, Jr, Graham, M. M., Ho, P. M., Masoudi, F. A., et al. (2013). Cardiovascular health: The importance of measuring patient-reported health status: A scientific statement from the American Heart Association. Circulation, 127(22), 2233–2249.CrossRefPubMedGoogle Scholar
- 5.Flynn, K. E., Lin, L., Ellis, S. J., Russell, S. D., Spertus, J. A., Whellan, D. J., et al. (2009). Outcomes, health policy, and managed care: Relationships between patient-reported outcome measures and clinical measures in outpatients with heart failure. American Heart Journal, 158(4 Suppl), S64–S71.CrossRefPubMedPubMedCentralGoogle Scholar
- 7.U.S. Department of Health and Human Services Food and Drug Administration (FDA). (2009). Guidance for industry patient-reported outcome measures: Use in medical product development to support labeling claims. http://www.fda.gov/downloads/Drugs/…/Guidances/UCM193282.pdf. Accessed May 15, 2015.
- 10.Bushmakin, A. G., Cappelleri, J. C., Taylor-Stokes, G., Sayers, J., Sadosky, A., Carroll, D., et al. (2011). Relationship between patient-reported disease severity and other clinical outcomes in osteoarthritis: A European perspective. Journal of Medical Economics, 14(4), 381–389.CrossRefPubMedGoogle Scholar
- 12.Basch, E., Iasonos, A., McDonough, T., Barz, A., Culkin, A., Kris, M. G., et al. (2006). Patient versus clinician symptom reporting using the National Cancer Institute Common Terminology Criteria for Adverse Events: Results of a questionnaire-based study. The Lancet Oncology, 7(11), 903–909.CrossRefPubMedGoogle Scholar
- 13.Spertus, J. (2014). Barriers to the use of patient-reported outcomes in clinical care. Circulation: Cardiovascular Quality and Outcomes, 7(1), 2–4.Google Scholar
- 15.Brozek, J. L., Guyatt, G. H., & Schunemann, H. J. (2006). How a well-grounded minimal important difference can enhance transparency of labelling claims and improve interpretation of a patient reported outcome measure. Health and Quality of Life Outcomes, 4, 69.CrossRefPubMedPubMedCentralGoogle Scholar
- 22.Spertus, J. A., Tooley, J., Jones, P., Poston, C., Mahoney, E., Deedwania, P., et al. (2002). Expanding the outcomes in clinical trials of heart failure: The quality of life and economic components of EPHESUS (EPlerenone’s neuroHormonal Efficacy and SUrvival Study). American Heart Journal, 143(4), 636–642.CrossRefPubMedGoogle Scholar
- 27.Hidding, A., van Santen, M., De Klerk, E., Gielen, X., Boers, M., Geenen, R., et al. (1994). Comparison between self-report measures and clinical observations of functional disability in ankylosing spondylitis, rheumatoid arthritis and fibromyalgia. Journal of Rheumatology, 21(5), 818–823.PubMedGoogle Scholar