ABRs of group 2 switching from on-demand treatment to once weekly prophylaxis with rIX-FP
| . | On-demand regimen (N = 19) . | Prophylaxis regimen (N = 19) . | Prophylaxis/on-demand . |
|---|---|---|---|
| Number of subjects with at least 1 spontaneous bleeding episode, n (%) | 19 (100.0) | 9 (47.4) | |
| AsBR* | |||
| Median (IQR) | 15.43 (7.98, 17.96) | 0.00 (0.00, 0.96) | |
| Estimated rate (95% CI)† | 13.62 (11.001, 16.868) | 0.55 (0.233, 1.322) | 0.04 (0.019, 0.089) |
| Reduction in AsBR with prophylaxis treatment (%) | |||
| Median (IQR) | 100.00 (90.53, 100.00) | ||
| P‡ | <.0001 | ||
| Total ABR* | |||
| Median (IQR) | 19.22 (16.70, 25.84) | 1.58 (0.00, 4.06) | |
| Estimated rate (95% CI)† | 20.09 (16.808, 24.003) | 2.22 (0.942, 5.243) | 0.11 (0.051, 0.238) |
| Reduction in total ABR with prophylaxis treatment (%) | |||
| Median (IQR) | 90.94 (81.19, 100.00) | ||
| P‡ | <.0001 |
| . | On-demand regimen (N = 19) . | Prophylaxis regimen (N = 19) . | Prophylaxis/on-demand . |
|---|---|---|---|
| Number of subjects with at least 1 spontaneous bleeding episode, n (%) | 19 (100.0) | 9 (47.4) | |
| AsBR* | |||
| Median (IQR) | 15.43 (7.98, 17.96) | 0.00 (0.00, 0.96) | |
| Estimated rate (95% CI)† | 13.62 (11.001, 16.868) | 0.55 (0.233, 1.322) | 0.04 (0.019, 0.089) |
| Reduction in AsBR with prophylaxis treatment (%) | |||
| Median (IQR) | 100.00 (90.53, 100.00) | ||
| P‡ | <.0001 | ||
| Total ABR* | |||
| Median (IQR) | 19.22 (16.70, 25.84) | 1.58 (0.00, 4.06) | |
| Estimated rate (95% CI)† | 20.09 (16.808, 24.003) | 2.22 (0.942, 5.243) | 0.11 (0.051, 0.238) |
| Reduction in total ABR with prophylaxis treatment (%) | |||
| Median (IQR) | 90.94 (81.19, 100.00) | ||
| P‡ | <.0001 |
The median rIX-FP dose during the once weekly prophylaxis regimen was 40.3 IU/kg (IQR, 37.6-50.6).
The ABR was defined as: 365.25 × (number of bleeding episodes)/(number of days in the observed treatment period of interest).
The estimated rate was calculated assuming a Poisson distribution.
P values are based on Wilcoxon Signed-rank test, with a hypothesis of H0: AsBR ratio (prophylaxis/on-demand) ≥0.50.