Table 2.
ABR among participants younger than 12 years of age treated with emicizumab 1.5 mg/kg per week (group A)
| Group A: emicizumab once weekly, n = 65 | ||
|---|---|---|
| Bleed result | 95% CI | |
| Efficacy period | ||
| Median duration (range), wk | 57.6 (17.9-92.6) | NA |
| Bleeding events treated with BPA | ||
| Annualized rate of bleeding events | 0.3 | 0.17-0.50* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA |
| 0 bleeds, n (%) | 50 (76.9) | 64.8-86.5 |
| 0-3 bleeds, n (%) | 65 (100) | 94.5-100 |
| All bleeding events, regardless of treatment with BPA | ||
| Annualized rate of bleeding events | 3.2 | 1.94-5.22* |
| Median annualized rate of bleeding events (IQR) | 0.6 (0.00-2.92) | NA |
| 0 bleeds, n (%) | 32 (49.2) | 36.6-61.9 |
| 0-3 bleeds, n (%) | 47 (72.3) | 59.8-82.7 |
| Treated events of spontaneous bleeding | ||
| Annualized rate of bleeding events | 0.0 | 0.01-0.10* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA |
| 0 bleeds, n (%) | 63 (96.9) | 89.3-99.6 |
| 0-3 bleeds, n (%) | 65 (100) | 94.5-100 |
| Treated events of joint bleeding | ||
| Annualized rate of bleeding events | 0.2 | 0.08-0.29* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA |
| 0 bleeds, n (%) | 55 (84.6) | 73.5-92.4 |
| 0-3 bleeds, n (%) | 65 (100) | 94.5-100 |
| Treated events of target joint bleeding | ||
| Annualized rate of bleeding events | Not estimable | Not estimable* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA |
| 0 bleeds, n (%) | 62 (95.4) | 87.1-99.0 |
| 0-3 bleeds, n (%) | 65 (100) | 94.5-100 |
A loading dose of 3 mg/kg emicizumab was given for 4 weeks followed by 1.5 mg/kg per week.
NA, not applicable. See Table 1 for expansion of other abbreviations.
*
Model-based annualized rates were estimated using negative binomial regression.