Table 4:
Annualised bleeding rates of children receiving once-weekly prophylaxis with rIX-FP.
| <6 years (N=12) |
6–11 years (N=15) |
Total (N=27) |
|
|---|---|---|---|
| Number of patients with ≥1 spontaneous bleeding episode, n (%)* | 4 (33.3) | 9 (60.0) | 13 (48.1) |
| AsBR † | |||
| Median (IQR) | 0.00 (0.00, 0.00) | 0.78 (0.00, 1.99) | 0.00 (0.00, 0.91) |
| Estimated rate (95% CI) ‡ | 0.09 (0.01, 0.58) | 0.86 (0.49, 1.51) | 0.56 (0.32, 1.00) |
| Joint ABR † | |||
| Median (IQR) | 0.50 (0.00, 1.45) | 1.13 (0.00, 2.36) | 0.99 (0.00, 2.33) |
| Estimated rate (95% CI) ‡ | 1.18 (0.50, 2.82) | 1.60 (0.91, 2.84) | 1.44 (0.89, 2.33) |
| Total ABR † | |||
| Median (IQR) | 2.64 (2.00, 6.48) | 3.39 (0.76, 5.91) | 3.12 (0.91, 5.91) |
| Estimated rate (95% CI) ‡ | 4.09 (2.66, 6.28) | 3.44 (2.13, 5.54) | 3.69 (2.65, 5.13) |
| Time on study (days) | |||
| Median (IQR) | 356 (313.0, 373.5) | 456 (361.0, 492.0) | 382 (330.0, 469.0) |
ABR, annualised bleeding rate; AsBR, annualised spontaneous bleeding rate; CI, confidence interval; IQR, interquartile range. The median (IQR) rIX-FP dose during the once-weekly prophylaxis regimen was 47.2 IU/kg (40.6, 55.8).
* Number of patients with ≥1 spontaneous bleeding episode during the course of the study.
† 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.