Table 1 Outcome measures
Outcome measure | Scale | Type | Measure | Method of analysis |
|---|---|---|---|---|
Feasibility outcomes | ||||
 Enrollment rate per year | Ratio | Continuous | Number of participants enrolled per year | Feasibility threshold: 24 patients per year |
 Refusal rate | Ratio | Continuous | Number of eligible patients refusing to participate | Feasibility threshold: < 40% |
 Retention rate | Ratio | Continuous | Number of participants completing study | Feasibility threshold: > 80% |
 Compliance rate | Ratio | Continuous | Number of participants taking study drug appropriately (≥ 80% study drug) | Feasibility threshold: > 90% |
Clinical outcomes | ||||
 Imaging changes | Ratio | Continuous | Change in global longitudinal strain on speckle tracking echocardiography and ejection fraction | ANCOVA |
 Biomarker changes | Ratio | Continuous | Change in NT-proBNP | ANCOVA |
 Functional changes | Ratio | Categorical | Changes in NYHA class | McNemar’s test |
 Quality of life changes | Ratio | Continuous | Changes on the Kansas City Cardiomyopathy Questionnaire | Descriptive |
 All-cause mortality | Ratio | Categorical | Difference in mortality between groups | McNemar’s test |
 Heart failure hospitalizations | Ratio | Categorical | Proportion of hospitalizations due to heart failure | McNemar’s test |
 Heart failure-related emergency room visits | Ratio | Categorical | Proportion of ER visits due to heart failure | McNemar’s test |