Table 5:
Summary of mild and moderate bleeding episodes and haemostatic response to rIX-FP.
| <6 years | 6–11 years | Overall | |
|---|---|---|---|
| Number of patients | 12 | 15 | 27 |
| Number of patients with bleeding episodes requiring treatment | 11 | 12 | 23 |
| Number of bleeding episodes requiring treatment | 45 | 61 | 106 |
| Type of bleeding episode, n (%) | |||
| Spontaneous | 1 (2.2) | 15 (24.6) | 16 (15.1) |
| Traumatic | 38 (84.4) | 35 (57.4) | 73 (68.9) |
| Post-surgery | 0 | 1 (1.6) | 1 (0.9) |
| Unknown | 6 (13.3) | 10 (16.4) | 16 (15.1) |
| Injections to treat a bleeding episode, n (%) | |||
| 1 injection | 40 (88.9) | 54 (88.5) | 94 (88.7) |
| 2 injections | 5 (11.1) | 4 (6.6) | 9 (8.5) |
| >2 injections | 0 | 3 (4.9) | 3 (2.8) |
| 1 or 2 injections | 45 (100.0) | 58 (95.1) | 103 (97.2) |
| Probability of success*, (95% CI) | NC | 95.1 (86.7, 98.3) | 97.2 (92.0, 99.0) |
| Number of minor/ moderate bleeding episodes requiring treatment | 43 | 61 | 104 |
| Haemostatic response †, n (%) | |||
| Excellent | 36 (83.7) | 42 (68.9) | 78 (75.0) |
| Good | 6 (14.0) | 16 (26.2) | 22 (21.2) |
| Moderate | 0 | 1 (1.6) | 1 (1.0) |
| Poor/No Response | 0 | 0 | 0 |
| Missing | 1 (2.3) | 2 (3.3) | 3 (2.9) |
CI, confidence interval; NC, not calculated.
* Probability of success is derived from a repeated measures model (expressed as percentages) and is defined as the probability of achieving haemostasis with 1 or 2 injections.
† Haemostatic response was evaluated by the physician. Definitions of haemostatic response for mild to moderate bleeding episodes: Excellent–pain relief and/or unequivocal improvement in objective signs of bleeding at approximately 24 hours after the first infusion and no additional infusions required in order to achieve haemostasis; Good–definite pain relief and/or improvement in signs of bleeding at approximately 24 hours after the first infusion, but required a second infusion in order to achieve haemostasis; Moderate–probable or slight beneficial effect at approximately 24 hours after the first infusion, and required more than 2 injections to achieve haemostasis; Poor/no response–no improvement or worsened at approximately 24 hours after the first infusion and additional haemostatic intervention required with other FIX product or plasma to achieve haemostasis.