Table 4.
Summary of bleeding episodes and hemostatic response to rIX-FP
| Prophylaxis (group 1) | On-demand (group 2) | Overall | ||
|---|---|---|---|---|
| On-demand regimen | Prophylaxis regimen | |||
| Number of patients | 40 | 23 | 19 | 63 |
| Number of patients with bleeding episodes requiring treatment | 30 | 23 | 11 | 53 |
| Number of bleeding episodes requiring treatment | 101 | 220 | 37 | 358 |
| Type of bleeding episode, n (%) | ||||
| Spontaneous | 48 (47.5) | 145 (65.9) | 11 (29.7) | 204 (57.0) |
| Traumatic | 48 (47.5) | 67 (30.5) | 25 (67.6) | 140 (39.1) |
| Postsurgery | 0 | 0 | 0 | 0 |
| Unknown | 5 (5.0) | 8 (3.6) | 1 (2.7) | 14 (3.9) |
| Injections to treat a bleeding episode, n (%) | ||||
| 1 Injection | 93 (92.1) | 208 (94.5) | 34 (91.9) | 335 (93.6) |
| 2 Injections | 8 (7.9) | 9 (4.1) | 1 (2.7) | 18 (5.0) |
| >2 Injections | 0 | 3 (1.4) | 2 (5.4) | 5 (1.4) |
| 1 or 2 Injections | 101 (100.0) | 217 (98.6) | 35 (94.6) | 353 (98.6) |
| Probability of success* | NC | 98.6 | 94.6 | 98.6 |
| 95% CI for probability of success | NC | (94.3, 99.7) | (76.9, 98.9) | (96.2, 99.5) |
| Prophylaxis (group 1) | On-demand (group 2) | Overall (N = 63) | ||
|---|---|---|---|---|
| Number of bleeding episodes requiring treatment | 101 | 257 | 358 | |
| Hemostatic response,† n (%) | ||||
| Excellent | 72 (71.3) | 225 (87.5) | 297 (83.0) | |
| Good | 21 (20.8) | 19 (7.4) | 40 (11.2) | |
| Moderate | 3 (3.0) | 6 (2.3) | 9 (2.5) | |
| Poor/no response | 0 | 1 (0.4) | 1 (0.3) | |
| Missing | 5 (5.0) | 6 (2.3) | 11 (3.1) | |
A second dose of rIX-FP was administered at least 24 h after the first injection, if needed, to achieve hemostatsis, as determined by the judgment of the patient and/ or provider.
NC, not calculated.
Probability of success is derived from a repeated measures model (expressed as percentages) and is defined as the probability of achieving hemostasis with 1 or 2 infusions.
Hemostatic response was evaluated by the physician. Definitions of hemostatic response: excellent, pain relief and/or unequivocal improvement in objective signs of bleeding at ∼24 h after the first infusion and no additional infusions required in order to achieve hemostasis; good, definite pain relief and/or improvement in signs of bleeding at ∼24 h after the first infusion but required a second infusion in order to achieve hemostasis; moderate, probable or slight beneficial effect at ∼24 h after the first infusion and required >2 infusions to achieve hemostasis; poor/no response, no improvement or worsened at ∼24 h after the first infusion and additional hemostatic intervention required with other FIX product or plasma to achieve hemostasis.