Table 6.
HCMSQ responder definition estimates from anchor- and distribution-based approaches using MAVERICK-HCM and EXPLORER-HCM trial data sets
| Domain | Anchor-based approach | Distribution-based approach | ||
|---|---|---|---|---|
| PGIC improved week 16 (N = 10) | PGIS improved week 16 (N = 6) | ½ SD | 1 SEM | |
| MAVERICK-HCM trial data set | ||||
| Cardiovascular symptoms | 0.39 | – | 0.51 | 0.51 |
| Tiredness | 0.83 | 0.91 | 0.34 | 0.30 |
| Shortness of breath | 2.01 | 2.35 | 1.08 | 0.93 |
| Total score | 1.46 | 1.59 | 0.62 | 0.60 |
| EXPLORER-HCM trial data set | ||||
| Cardiovascular symptoms | – | – | 0.87 | 0.58 |
| Tiredness | – | – | 0.37 | 0.31 |
| Shortness of breath | – | – | 1.45 | 1.15 |
| Total score | – | – | 0.89 | 0.60 |
HCM hypertrophic cardiomyopathy, HCMSQ Hypertrophic Cardiomyopathy Symptom Questionnaire, PGIC Patient Global Impression of Change, PGIS Patient Global Impression of Severity, SD standard deviation, SEM standard error of the mean
For EXPLORER-HCM, only distribution-based approaches (i.e., ½ SD and 1 SEM) were conducted given that only baseline data were used