Table 2.
Optimal posology results—Adolescents group (12–17 years); 5% target trough level with 10% control trough level.
| Parameter | rIX-FP | rFIX |
rFIXFc WP |
rFIXFc IP |
rFIXFc* WP and IP |
N9-GP |
|---|---|---|---|---|---|---|
|
Steady-state FIX trough levels, IU/dL Median (IQR) |
7.7 (6.0–9.0) |
2.6 (1.5–4.2) |
4.4 (3.4–5.0) |
3.9 (2.9–5.0) |
4.2 (3.3–5.0) |
5.1 (5.1–5.2) |
| Patients below target, % | 1.2 | 83.0 | 63.8 | 75.3 | 67.3 | 0.0 |
|
Dose (IU/kg per week) Median (IQR) |
35.0 (25.0–35.0) |
182.0 (182.0–182.0) |
88.0 (79.4–88.0) |
87.5 (87.5–87.5) |
87.5 (84.5–88.0) |
12.5 (10.0–15.5) |
| Patients among dose-intervals, %† | ||||||
| ≤ 7 days | 47.4 | 100 | 100.0 | 0.0 | 68.8 | 100.0 |
| 8–10 days | 16.7 | 0.0 | 0.0 | 98.6 | 30.9 | 0.0 |
| 11–14 days | 35.9 | 0.0 | 0.0 | 1.4 | 0.4 | 0.0 |
| 15–21 days | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 |
|
Ratio between weekly dose (IU/kg) FIX product and rIX-FP Median (IQR) |
- |
5.2 (5.2–7.3) |
2.5 (2.4–3.3) |
2.5 (2.5–3.5) |
2.5 (2.5–3.5) |
0.4 (0.3–0.5) |
*Patients were randomly assigned to individualized interval prophylaxis from a Bernoulli distribution with a probability 0.315, based on data from clinical trial 998HB102 (NCT01027364). In the simulations underlying the results of this table, 68.8% of the simulated patients were assigned to receive weekly prophylaxis and 31.3% to individualized interval prophylaxis. †Percentages may not sum to 100 due to rounding.
FIX, factor IX; IP, individualized interval prophylaxis; IQR, interquartile range; IU, international units; WP, weekly prophylaxis.