Efficacy analyses for participants younger than 12 years of age treated with emicizumab 3 mg/kg every 2 weeks (group B) and 6 mg/kg every 4 weeks (group C)
| . | Group B: emicizumab every 2 wk, n = 10 . | Group C: emicizumab every 4 wk, n = 10 . | ||
|---|---|---|---|---|
| Bleed result . | 95% CI . | Bleed result . | 95% CI . | |
| Efficacy period | ||||
| Median duration (range), wk | 21.3 (18.6-24.1) | NA | 19.9 [8.9-24.1] | NA |
| Bleeding events treated with BPA | ||||
| Annualized rate of bleeding events | 0.2 | 0.03-1.72* | 2.2 | 0.69-6.81* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA | 0.0 (0.00-3.26) | NA |
| 0 bleeds, n (%) | 9 (90.0) | 55.5-99.7 | 6 (60.0) | 26.2-87.8 |
| 0-3 bleeds, n (%) | 10 (100) | 69.2-100 | 10 (100) | 69.2-100 |
| All bleeding events, regardless of treatment with BPA | ||||
| Annualized rate of bleeding events | 1.5 | 0.62-3.40* | 3.8 | 1.42-10.11* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-2.81) | NA | 1.6 (0.00-4.84) | NA |
| 0 bleeds, n (%) | 6 (60.0) | 26.2-87.8 | 5 (50.0) | 18.7-81.3 |
| 0-3 bleeds, n (%) | 10 (100) | 69.2-100 | 9 (90.0) | 55.5-99.7 |
| Treated events of spontaneous bleeding | ||||
| Annualized rate of bleeding events | Not estimable | Not estimable* | 0.8 | 0.05-12.00* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA | 0.0 (0.00-0.00) | NA |
| 0 bleeds, n (%) | 10 (100) | 69.2-100 | 9 (90.0) | 55.5-99.7 |
| 0-3 bleeds, n (%) | 10 (100) | 69.2-100 | 10 (100) | 69.2-100 |
| Treated events of joint bleeding | ||||
| Annualized rate of bleeding events | 0.2 | 0.03-1.72* | 1.7 | 0.60-4.89* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA | 0.0 (0.00-3.26) | NA |
| 0 bleeds, n (%) | 9 (90.0) | 55.5-99.7 | 6 (60.0) | 26.2-87.8 |
| 0-3 bleeds, n (%) | 10 (100) | 69.2-100 | 10 (100) | 69.2-100 |
| Treated events of target joint bleeding | ||||
| Annualized rate of bleeding events | 0.2 | 0.03-1.72* | 0.5 | 0.05-5.88* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA | 0.0 (0.00-0.00) | NA |
| 0 bleeds, n (%) | 9 (90.0) | 55.5-99.7 | 9 (90.0) | 55.5-99.7 |
| 0-3 bleeds, n (%) | 10 (100) | 69.2-100 | 10 (100) | 69.2-100 |
| . | Group B: emicizumab every 2 wk, n = 10 . | Group C: emicizumab every 4 wk, n = 10 . | ||
|---|---|---|---|---|
| Bleed result . | 95% CI . | Bleed result . | 95% CI . | |
| Efficacy period | ||||
| Median duration (range), wk | 21.3 (18.6-24.1) | NA | 19.9 [8.9-24.1] | NA |
| Bleeding events treated with BPA | ||||
| Annualized rate of bleeding events | 0.2 | 0.03-1.72* | 2.2 | 0.69-6.81* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA | 0.0 (0.00-3.26) | NA |
| 0 bleeds, n (%) | 9 (90.0) | 55.5-99.7 | 6 (60.0) | 26.2-87.8 |
| 0-3 bleeds, n (%) | 10 (100) | 69.2-100 | 10 (100) | 69.2-100 |
| All bleeding events, regardless of treatment with BPA | ||||
| Annualized rate of bleeding events | 1.5 | 0.62-3.40* | 3.8 | 1.42-10.11* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-2.81) | NA | 1.6 (0.00-4.84) | NA |
| 0 bleeds, n (%) | 6 (60.0) | 26.2-87.8 | 5 (50.0) | 18.7-81.3 |
| 0-3 bleeds, n (%) | 10 (100) | 69.2-100 | 9 (90.0) | 55.5-99.7 |
| Treated events of spontaneous bleeding | ||||
| Annualized rate of bleeding events | Not estimable | Not estimable* | 0.8 | 0.05-12.00* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA | 0.0 (0.00-0.00) | NA |
| 0 bleeds, n (%) | 10 (100) | 69.2-100 | 9 (90.0) | 55.5-99.7 |
| 0-3 bleeds, n (%) | 10 (100) | 69.2-100 | 10 (100) | 69.2-100 |
| Treated events of joint bleeding | ||||
| Annualized rate of bleeding events | 0.2 | 0.03-1.72* | 1.7 | 0.60-4.89* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA | 0.0 (0.00-3.26) | NA |
| 0 bleeds, n (%) | 9 (90.0) | 55.5-99.7 | 6 (60.0) | 26.2-87.8 |
| 0-3 bleeds, n (%) | 10 (100) | 69.2-100 | 10 (100) | 69.2-100 |
| Treated events of target joint bleeding | ||||
| Annualized rate of bleeding events | 0.2 | 0.03-1.72* | 0.5 | 0.05-5.88* |
| Median annualized rate of bleeding events (IQR) | 0.0 (0.00-0.00) | NA | 0.0 (0.00-0.00) | NA |
| 0 bleeds, n (%) | 9 (90.0) | 55.5-99.7 | 9 (90.0) | 55.5-99.7 |
| 0-3 bleeds, n (%) | 10 (100) | 69.2-100 | 10 (100) | 69.2-100 |
A loading dose of 3 mg/kg emicizumab was given for 4 weeks followed by the maintenance dose listed.
See Tables 1 and 2 for expansion of abbreviations.
Model-based annualized rates were estimated using negative binomial regression.