Objective
To incorporate condition-specific concerns into generic patient-reported outcome measures
Study Design
| Design Element |
Description |
| Design |
Survey development and testing
|
| Data Sources and Data Sets |
Patient advisors and expert panel, 61 focus group participants with heart failure, 68 focus group participants with knee osteoarthritis, 10 cognitive interview participants with heart failure, 10 cognitive interview participants with knee osteoarthritis, 786 field-test survey respondents with heart failure, 911 field-test survey respondents with knee osteoarthritis
|
| Analytic Approach |
Psychometric analysis to evaluate survey reliability, validity, and sensitivity to change
|
| Outcomes |
Patient-reported outcome measures of heart failure and knee osteoarthritis
|
Researchers developed two disease-specific surveys, one for heart failure and the other for knee osteoarthritis, using Patient-Reported Outcomes Measurement Information System (PROMIS®) methods and recommendations from the International Society for Pharmacoeconomics and Outcomes Research’s Task Force. First, researchers conducted focus groups with 61 patients with heart failure and 68 patients with knee osteoarthritis to identify their priority outcome domains. Next, researchers drafted new survey items to address gaps between the priority domains and domains in existing PROMIS item banks. Researchers refined the new items based on the results of cognitive interviews with 10 patients with each condition and added the items to the generic measures.
Researchers field tested the new measures to evaluate reliability, validity, and sensitivity to change. To assess reliability and validity, the sample included 600 patients per condition from an online panel. The average ages of heart failure and knee osteoarthritis patients were 54 and 52, respectively. Among heart failure patients, 45% were female, 67% were white, 19% were African American, and 14% were other races. Among knee osteoarthritis patients, 60% were female, 70% were white, 18% were African American, and 12% were other races. Three to seven days after the initial surveys, researchers asked 100 patients per condition in this sample to retake the surveys to evaluate test-retest reliability.
To assess sensitivity to change, the sample included 311 patients with knee osteoarthritis and 186 patients with heart failure from nine clinics. The average ages of heart failure and knee osteoarthritis patients were 56 and 65, respectively. Among heart failure patients, 42% were female, 55% were white, 39% were African American, and 6% were other races. Among knee osteoarthritis patients, 68% were female, 87% were white, 8% were African American, and 5% were other races. Patients in this sample completed a follow-up survey three months after baseline.
Patient advisors and clinicians who had experience with these conditions provided input throughout the study.
Results
Results suggested that both measures had good reliability, validity, and sensitivity to change.
Heart failure. The final measure included 86 items across 18 domains.
- Reliability and validity. Internal consistency reliability of the domains, measured by Cronbach’s alpha, ranged from 0.52 to 0.94. Test-retest reliability of the domains, measured by Pearson’s r, ranged from 0.81 to 0.98. All domains had correlations greater than 0.6 with PROMIS Global Health Physical and Mental Health summary scores, indicating acceptable concurrent validity.
- Sensitivity to change. For 8 of 18 domains, patients who reported experiencing meaningful positive change in the domain had better domain-change scores than patients who reported negative or no change (p< 0.05).
Knee osteoarthritis. The final measure included 76 items across 14 domains. The new domains were independence, life satisfaction, and symptoms.
- Reliability and validity. Internal consistency reliability of the domains, as measured by Cronbach’s alpha, ranged from 0.67 to 0.95. Test-retest reliability of the domains, as measured by Pearson’s r, ranged from 0.88 to 0.98. All domains had correlations greater than 0.6 with PROMIS Global Health Physical and Mental Health summary scores, indicating acceptable concurrent validity.
- Sensitivity to change. Patients who reported experiencing meaningful positive change in the domain had better domain-change scores than patients who reported negative or no change (p< 0.05).
Limitations
Focus group participants did not represent the educational, racial, or ethnic backgrounds of all people in the United States, which may limit the generalizability of the priority domains identified for the measures. Also, the full final instruments may be too long for ongoing data collection in clinical settings.
Conclusions and Relevance
Using two high-impact health conditions, researchers created disease-specific patient-reported outcome measures. These measures added items about patient concerns to existing generic patient-reported outcome measures.
Future Research Needs
Researchers could use this study’s methodology to adapt generic patient-reported outcome measures for other specific conditions. Additional studies could also test the knee osteoarthritis and heart failure measures developed for this study with patients of different educational, racial, or ethnic backgrounds and create short forms of the measures for use in clinical settings.
