ABR and hemostatic success of patients in pathfinder6
| . | Pre-prophylaxis period∗ . | Prophylaxis period∗ . | All patients (total observation period) . |
|---|---|---|---|
| No. of patients | 54 | 65 | 80 |
| No. of patients with bleeds, N (%) | 46 (85.2) | 54 (83.1) | 73 (91.3) |
| No. of bleeds | 159 | 266 | 425 |
| Mean treatment period (years) | 0.470 | 2.168 | 2.079 |
| Estimated mean ABR (95% CI)† | 6.62 (5.34; 8.21) | 2.37 (1.79; 3.15) | 3.34 (2.64; 4.22) |
| Median ABR (IQR) | 6.63 (2.61; 13.04) | 1.42 (0.76; 3.13) | 2.14 (1.14; 5.71) |
| Bleeds treated with ≤2 injections, % | 87.2 | 91.2 | 89.7 |
| Success rate for treatment of bleeds, % (95% CI)‡ | 82.7 (73.1; 89.4) | 90.5 (84.9; 94.1) | 86. 5 (81.0; 90.6) |
| . | Pre-prophylaxis period∗ . | Prophylaxis period∗ . | All patients (total observation period) . |
|---|---|---|---|
| No. of patients | 54 | 65 | 80 |
| No. of patients with bleeds, N (%) | 46 (85.2) | 54 (83.1) | 73 (91.3) |
| No. of bleeds | 159 | 266 | 425 |
| Mean treatment period (years) | 0.470 | 2.168 | 2.079 |
| Estimated mean ABR (95% CI)† | 6.62 (5.34; 8.21) | 2.37 (1.79; 3.15) | 3.34 (2.64; 4.22) |
| Median ABR (IQR) | 6.63 (2.61; 13.04) | 1.42 (0.76; 3.13) | 2.14 (1.14; 5.71) |
| Bleeds treated with ≤2 injections, % | 87.2 | 91.2 | 89.7 |
| Success rate for treatment of bleeds, % (95% CI)‡ | 82.7 (73.1; 89.4) | 90.5 (84.9; 94.1) | 86. 5 (81.0; 90.6) |
Patients who switched from pre-prophylaxis to prophylaxis are represented in both columns. ABR analysis includes bleeds treated only with bypassing products; these patients were omitted for hemostatic success calculations. ITI period was excluded.
Based on a negative binomial model with the log of treatment duration as an offset.
Rate estimate and 95% confidence interval (CI) obtained from a binomial model, with subject random effects; missing is included as failure.