ABRs
| Characteristics . | 10 IU/kg . | 40 IU/kg . | On demand . |
|---|---|---|---|
| All patients | |||
| N | 30 | 29 | 15 |
| Median (IQR) | 2.93 (0.99-6.02) | 1.04 (0.00-4.00) | 15.58 (9.56-26.47) |
| Estimated rate (95% CI)* | 4.56 (3.01-6.90) | 2.51 (1.42-4.43) | — |
| P value† | .40 | .01 | |
| Previous prophylaxis patients | |||
| N | 20 | 17 | 2 |
| Bleeding rate during the last 12 months before trial‡ | |||
| Median | 4.75 | 4.00 | 9.50 |
| Estimated rate | 5.13 | 7.49 | 9.50 |
| Bleeding rates during trial | |||
| Median | 2.99 | 1.93 | 25.69 |
| Estimated rate | 4.68 | 3.33 | 29.4 |
| Previous on-demand patients | |||
| N | 10 | 12 | 13 |
| Bleeding rate during the last 12 months before trial‡ | |||
| Median | 14.0 | 12.5 | 15.0 |
| Estimated rate | 17.9 | 21.2 | 22.7 |
| Bleeding rates during trial | |||
| Median | 2.06 | 0.52 | 13.0 |
| Estimated rate | 4.30 | 1.32 | 17.6 |
| All patients by type of bleed | |||
| Spontaneous bleeding episodes | |||
| Median (IQR) | 0.97 (0.00-4.01) | 0.00 (0.00-0.98) | 11.1 (7.16-15.8) |
| Estimated rate (95% CI)* | 3.14 (1.78-5.56) | 1.22 (0.48-3.10) | |
| Traumatic bleeding episodes | |||
| Median (IQR) | 0.98 (0.00-1.93) | 0.00 (0.00-2.04) | 1.73 (0.00-8.95) |
| Estimated rate (95% CI)* | 1.35 (0.81-2.24) | 1.29 (0.76-2.19) |
| Characteristics . | 10 IU/kg . | 40 IU/kg . | On demand . |
|---|---|---|---|
| All patients | |||
| N | 30 | 29 | 15 |
| Median (IQR) | 2.93 (0.99-6.02) | 1.04 (0.00-4.00) | 15.58 (9.56-26.47) |
| Estimated rate (95% CI)* | 4.56 (3.01-6.90) | 2.51 (1.42-4.43) | — |
| P value† | .40 | .01 | |
| Previous prophylaxis patients | |||
| N | 20 | 17 | 2 |
| Bleeding rate during the last 12 months before trial‡ | |||
| Median | 4.75 | 4.00 | 9.50 |
| Estimated rate | 5.13 | 7.49 | 9.50 |
| Bleeding rates during trial | |||
| Median | 2.99 | 1.93 | 25.69 |
| Estimated rate | 4.68 | 3.33 | 29.4 |
| Previous on-demand patients | |||
| N | 10 | 12 | 13 |
| Bleeding rate during the last 12 months before trial‡ | |||
| Median | 14.0 | 12.5 | 15.0 |
| Estimated rate | 17.9 | 21.2 | 22.7 |
| Bleeding rates during trial | |||
| Median | 2.06 | 0.52 | 13.0 |
| Estimated rate | 4.30 | 1.32 | 17.6 |
| All patients by type of bleed | |||
| Spontaneous bleeding episodes | |||
| Median (IQR) | 0.97 (0.00-4.01) | 0.00 (0.00-0.98) | 11.1 (7.16-15.8) |
| Estimated rate (95% CI)* | 3.14 (1.78-5.56) | 1.22 (0.48-3.10) | |
| Traumatic bleeding episodes | |||
| Median (IQR) | 0.98 (0.00-1.93) | 0.00 (0.00-2.04) | 1.73 (0.00-8.95) |
| Estimated rate (95% CI)* | 1.35 (0.81-2.24) | 1.29 (0.76-2.19) |
Estimated rates for prophylaxis patients are based on a Poisson regression model with dose as a factor, allowing for overdispersion and using treatment duration as an offset.
P values are from the 1-sided test of the null hypothesis that the estimated rate is at least 4.8, evaluated at the 2.5% level.
Bleeding rate during the last 12 months before trial on that particular treatment regimen.