Details of bleeding episodes and hemostatic response
| Details . | 10 IU/kg . | 40 IU/kg . | On demand . | All . |
|---|---|---|---|---|
| Number of patients | 30 | 29 | 15 | 74 |
| Number of patients with bleeding episodes | 25 | 16 | 14 | 55 |
| Number of bleeding episodes | 132 | 70 | 143 | 345 |
| Type of bleeding episode, n (%) | ||||
| Spontaneous | 91 (68.9) | 34 (48.6) | 102 (71.3) | 227 (65.8) |
| Traumatic | 39 (29.5) | 36 (51.4) | 41 (28.7) | 116 (33.6) |
| After minor surgery | 1 (0.8) | — | — | 1 (0.3) |
| Other | 1 (0.8) | — | — | 1 (0.3) |
| Injections needed to treat bleeding episode (from start to stop of bleeding episode), n (%) | ||||
| 1 injection | 111 (84.1) | 69 (98.6) | 120 (83.9) | 300 (87.0) |
| 2 injections | 16 (12.1) | — | 20 (14.0) | 36 (10.4) |
| 3 injections | 2 (1.5) | 1 (1.4) | — | 3 (0.9) |
| ≥4 injections | 3 (2.3) | — | 3 (2.1) | 6 (1.7) |
| Hemostatic response, n (%)* | ||||
| Excellent | 41 (31.1) | 35 (50.0) | 43 (30.1) | 119 (34.5) |
| Good | 72 (54.5) | 32 (45.7) | 92 (64.3) | 196 (56.8) |
| Moderate | 13 (9.8) | 2 (2.9) | 7 (4.9) | 22 (6.4) |
| Poor | 4 (3.0) | — | — | 4 (1.2) |
| Missing | 2 (1.5) | 1 (1.4) | 1 (0.7) | 4 (1.2) |
| Successful hemostatic responses, n (%)†,¶ | 113 (86.9) | 67 (97.1) | 135 (95.1) | 315 (92.4) |
| Failure hemostatic responses, n (%)‡,¶ | 17 (13.1) | 2 (2.9) | 7 (4.9) | 26 (7.6) |
| Details . | 10 IU/kg . | 40 IU/kg . | On demand . | All . |
|---|---|---|---|---|
| Number of patients | 30 | 29 | 15 | 74 |
| Number of patients with bleeding episodes | 25 | 16 | 14 | 55 |
| Number of bleeding episodes | 132 | 70 | 143 | 345 |
| Type of bleeding episode, n (%) | ||||
| Spontaneous | 91 (68.9) | 34 (48.6) | 102 (71.3) | 227 (65.8) |
| Traumatic | 39 (29.5) | 36 (51.4) | 41 (28.7) | 116 (33.6) |
| After minor surgery | 1 (0.8) | — | — | 1 (0.3) |
| Other | 1 (0.8) | — | — | 1 (0.3) |
| Injections needed to treat bleeding episode (from start to stop of bleeding episode), n (%) | ||||
| 1 injection | 111 (84.1) | 69 (98.6) | 120 (83.9) | 300 (87.0) |
| 2 injections | 16 (12.1) | — | 20 (14.0) | 36 (10.4) |
| 3 injections | 2 (1.5) | 1 (1.4) | — | 3 (0.9) |
| ≥4 injections | 3 (2.3) | — | 3 (2.1) | 6 (1.7) |
| Hemostatic response, n (%)* | ||||
| Excellent | 41 (31.1) | 35 (50.0) | 43 (30.1) | 119 (34.5) |
| Good | 72 (54.5) | 32 (45.7) | 92 (64.3) | 196 (56.8) |
| Moderate | 13 (9.8) | 2 (2.9) | 7 (4.9) | 22 (6.4) |
| Poor | 4 (3.0) | — | — | 4 (1.2) |
| Missing | 2 (1.5) | 1 (1.4) | 1 (0.7) | 4 (1.2) |
| Successful hemostatic responses, n (%)†,¶ | 113 (86.9) | 67 (97.1) | 135 (95.1) | 315 (92.4) |
| Failure hemostatic responses, n (%)‡,¶ | 17 (13.1) | 2 (2.9) | 7 (4.9) | 26 (7.6) |
Definitions of hemostatic response: excellent = abrupt pain relief and/or clear improvement in objective signs of bleeding within 8 hours after a single injection; good = noticeable pain relief and/or improvement in signs of bleeding within 8 hours after a single injection; moderate = probable or slight beneficial effect within the first 8 hours after the first injection but requiring more than a single injection within 8 hours; poor = either no improvement or worsening of symptoms within 8 hours after 2 injections.
A successful hemostatic response was defined as an excellent or good hemostatic response. Note that this observed rate is slightly different from the model-based estimate presented in Results, which was 92.2%.
A failure hemostatic response was defined as a moderate or poor hemostatic response.
Bleeding episodes with a missing hemostatic response were not included in the calculation of successful and failure hemostatic responses.