Response to treatment with eltrombopag in 12 MYH9-RD patients
| Primary endpoints . | . | . |
|---|---|---|
| Any response, % (95% CI) | 91.6 (61.5-99.7) | |
| Major response | 66.7 (34.9-90.1) | |
| Minor response | 25.0 (5.4-57.2) | |
| Platelet count, × 109/L | ||
| Mean baseline (SD) | 31.2 (14.8) | |
| Mean end therapy (SD) | 104.7 (56.7) | P = .002 |
| Mean difference (95%CI) | 73.5 (44.4-102.5) | |
| % of patients with bleeding diathesis (grade 1 or 2 of WHO bleeding score) | ||
| Baseline | 83.3 | |
| End therapy | 16.7 | P = .008 |
| Mean difference (95% CI) | 66.7 (31.7-100) |
| Primary endpoints . | . | . |
|---|---|---|
| Any response, % (95% CI) | 91.6 (61.5-99.7) | |
| Major response | 66.7 (34.9-90.1) | |
| Minor response | 25.0 (5.4-57.2) | |
| Platelet count, × 109/L | ||
| Mean baseline (SD) | 31.2 (14.8) | |
| Mean end therapy (SD) | 104.7 (56.7) | P = .002 |
| Mean difference (95%CI) | 73.5 (44.4-102.5) | |
| % of patients with bleeding diathesis (grade 1 or 2 of WHO bleeding score) | ||
| Baseline | 83.3 | |
| End therapy | 16.7 | P = .008 |
| Mean difference (95% CI) | 66.7 (31.7-100) |
CI indicates confidence interval; and SD, standard deviation.