Outcomes, health policy, and managed care: Relationships between patient-reported outcome measures and clinical measures in outpatients with heart failure

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Background

Patient-reported outcomes are increasingly used to assess the efficacy of new treatments. Understanding relationships between these and clinical measures can facilitate their interpretation. We examined associations between patient-reported measures of health-related quality of life and clinical indicators of disease severity in a large, heterogeneous sample of patients with heart failure.

Methods

Patient-reported measures, including the Kansas City Cardiomyopathy Questionnaire (KCCQ) and the EuroQol Visual Analog Scale (VAS), and clinical measures, including peak Vo2, 6-minute walk distance, and New York Heart Association (NYHA) class, were assessed at baseline in 2331 patients with heart failure. We used general linear models to regress patient-reported measures on each clinical measure. Final models included for significant sociodemographic variables and 2-way interactions.

Results

The KCCQ was correlated with peak Vo2 (r = .21) and 6-minute walk distance (r = .27). The VAS was correlated with peak Vo2 (r = .09) and 6-minute walk distance (r = .11). Using the KCCQ as the response variable, a 1-SD difference in peak Vo2 (4.7 mL/kg/min) was associated with a 2.86-point difference in the VAS (95% CI, 1.98-3.74) and a 4.75-point difference in the KCCQ (95% CI, 3.78-5.72). A 1-SD difference in 6-minute walk distance (105 m) was associated with a 2.78-point difference in the VAS (95% CI, 1.92-3.64) and a 5.92-point difference in the KCCQ (95% CI, 4.98-6.87); NYHA class III was associated with an 8.26-point lower VAS (95% CI, 6.59-9.93) and a 12.73-point lower KCCQ (95% CI, 10.92-14.53) than NYHA class II.

Conclusions

These data may inform deliberations about how to best measure benefits of heart failure interventions, and they generally support the practice of considering a 5-point difference on the KCCQ and a 3-point difference on the VAS to be clinically meaningful.

Section snippets

Methods

HF-ACTION was a multicenter, randomized controlled trial designed to test the long-term safety and efficacy of aerobic exercise training versus usual care in patients with left ventricular dysfunction and New York Heart Association (NYHA) class II to IV heart failure. 6 Baseline assessments included health-related quality of life and health status measures and prerandomization cardiopulmonary exercise testing to determine aerobic capacity. Enrollment criteria included left ventricular ejection

Results

HF-ACTION randomly assigned 2331 patients, of whom 2330 (99.9%) completed the KCCQ and 2274 (98%) completed the VAS. Participant characteristics are shown in Table I . Table II shows the distributions of patient-reported and clinical outcome measures. Mean peak Vo2 was 15 mL/kg per minute, mean 6-minute walk distance was 365 m, and nearly two thirds of the patients were NYHA class II. The mean KCCQ score was 66, and the mean VAS score was 66.

Discussion

We used baseline data from HF-ACTION to characterize relationships among important patient-reported health-related quality-of-life and clinical measures and to examine whether the relationships differed among subgroups. As expected, the patient-reported outcome measures (ie, VAS and KCCQ) were moderately correlated with each other, and the heart failure–specific KCCQ had stronger correlations with exercise capacity than the more general VAS. The KCCQ physical limitation subscale, which was

Conclusions

Differences in patient-reported outcome measures (including the KCCQ, its subscales, and the VAS) are associated with differences in clinical measures (including peak Vo2, 6-minute walk distance, and NYHA class). The VAS, a general patient-reported measure, had smaller correlations with exercise capacity than the disease-specific KCCQ. With a few exceptions, these relationships are consistent across different groups of patients with heart failure. While the small size of the correlations

Disclosures

Dr Ellis reports receiving grants from GE Medical. Dr Spertus owns the copyright to the Kansas City Cardiomyopathy Questionnaire. Dr Whellan reports receiving grants or funding from GE Medical and the National Institutes of Health. Dr Piña reports receiving grants or funding from the National Institutes of Health; receiving personal income for consulting from the Food and Drug Administration; and receiving honoraria from AstraZeneca, Innovia, Merck, Novartis, Sanofi-Aventis, and Solvay. Dr

Acknowledgements

We thank Susan Kay Roll, RN, University of Cincinnati, for her role as an author on the abstract and Damon M. Seils, MA, Duke University, for assistance with manuscript preparation. Ms Roll and Mr Seils did not receive compensation for their assistance apart from their employment at institutions where the study was conducted.

References (24)

  • LutherS.A. et al.

    The relationship between B-type natriuretic peptide and health status in patients with heart failure

    J Card Fail

    (2005)
  • WeinbergerM. et al.

    Does increased access to primary care reduce hospital readmissions? Veterans Affairs Cooperative Study Group on Primary Care and Hospital Readmission

    N Engl J Med

    (1996)
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    This research was supported by National Institutes of Health grants: 5U01HL063747, 5U01HL068973, 5U01HL066501, 5U01HL066482, 5U01HL064250, 5U01HL066494, 5U01HL064257, 5U01HL066497, 5U01HL068980, 5U01HL064265, 5U01HL066491, 5U01HL064264, 5U01HL066461, R37AG18915, and P60AG10484.

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