Table 2.
Efficacy end points for prophylaxis and episodic treatment groups
| Arm 1: individualized prophylaxis (n = 117) | Arm 2: weekly prophylaxis (n = 23) | Arm 3: episodic treatment (n = 23) | |
|---|---|---|---|
| ABR, negative binomial model (95% CI) | 2.9 (2.3-3.7) | 8.9 (5.5-14.5) | 37.3 (24.0-57.7) |
| % Reduction vs arm 3 (P)* | 92 (<.001) | 76 (<.001) | — |
| ABR by type and location of bleeds, median (IQR) | |||
| Overall | 1.6 (0.0, 4.7) | 3.6 (1.9, 8.4) | 33.6 (21.1, 48.7) |
| Spontaneous | 0.0 (0.0, 2.0) | 1.9 (0.0, 4.8) | 20.2 (12.2, 36.8) |
| Traumatic | 0.0 (0.0, 1.8) | 1.7 (0.0, 3.3) | 9.3 (1.7, 11.9) |
| Joint | |||
| Spontaneous | 0.0 (0.0, 1.7) | 0.0 (0.0, 3.8) | 18.6 (7.6, 29.6) |
| Traumatic | 0.0 (0.0, 1.2) | 0.0 (0.0, 2.0) | 3.9 (0.0, 8.6) |
| Muscle | |||
| Spontaneous | 0.0 (0.0, 0.0) | 0.0 (0.0, 0.0) | 5.1 (1.8, 6.8) |
| Traumatic | 0.0 (0.0, 0.0) | 0.0 (0.0, 0.0) | 0.0 (0.0, 2.0) |
| Subjects with no bleeding episodes, n (%) | 53 (45.3) | 4 (17.4) | 0 (0) |
| Weekly dose, IU/kg, median (min, max) | |||
| Overall | 77.9 (54.0, 141.5) | 65.6 (59.4, 70.7) | — |
| Weekly dose, IU/kg, mean (SD) | |||
| Overall | 85.4 (19.3) | 65.8 (2.9) | — |
| Dosing interval during last 3 mo on study,† n (%) | |||
| 3 or more d | 111 (99.1) | NA | NA |
| 4 or more d | 39 (34.8) | NA | NA |
| 5 d | 33 (29.5) | NA | NA |
—, none; SD, standard deviation; NA, not applicable.
IQR, 25th and 75th percentiles.
*
Reduction in ABR compared with arm 3, calculated using negative binomial model.
†
In subjects on study for ≥6 months; n = 112 in arm 1, n = 16 in arm 2.