Sucrose-Formulated Recombinant Factor VIII Dosing Flexibility in Prophylaxis Regimens: Experience from Postmarketing Surveillance Studies
Table 1
Demographic and dosing characteristics.
Prophylaxis 1-2x/wk ()
Prophylaxis ≥3x/wk ()
Total ()
Age, y
Mean
23
21
22
Median (range)
20 (0–63)
15 (1–71)
17 (0–71)
18 y, (%)
28 (44)
43 (57)
71 (51)
Race, (%)
White
52 (83)
61 (80)
113 (81)
Asian
5 (8)
4 (5)
9 (7)
Others
2 (3)
3 (4)
5 (4)
Missing
4 (6)
8 (11)
12 (9)
Target joint present, (%)
17 (27)
33 (43)
50 (36)
Prophylaxis observation period, d
Mean ± SD
573 ± 220
609 ± 207
593 ± 213
Median (range)
695 (151–826)
731 (140–839)
726 (140–839)
Number of excluded nonprophylaxis days/patient
Mean ± SD
41 ± 108
34 ± 96
37 ± 101
Median (range)
0 (0–516)
0 (0–506)
0 (0–516)
Number of all injections/wk/patient
Mean ± SD
1.8 ± 0.5
3.1 ± 0.5
2.5 ± 0.8
Median (range)
1.9 (1.0–3.2)
3.0 (2.0–4.8)
2.6 (1.0–4.8)
Number of prophylaxis injections/wk/patient
Mean ± SD
1.6 ± 0.4
2.8 ± 0.4
2.3 ± 0.7
Median (range)
1.6 (0.9–2.2)
2.8 (1.5–3.8)
2.3 (0.9–3.8)
Time between prophylaxis injections,† d
Mean ± SD
4.4 ± 1.4
2.2 ± 0.4
3.2 ± 1.5
Median (range)
4.0 (3.0–7.0)
2.0 (2.0–3.0)
3.0 (2.0–7.0)
Prophylaxis dose/wk, IU/kg
Mean ± SD
44.1 ± 26.8
73.5 ± 33.9
60.2 ± 34.1
Median (range)
33.5 (11.4–101.9)
71.5 (17.1–166.5)
56.2 (11.4–166.5)
Prophylaxis dose/injection, IU/kg
Mean ± SD
27.0 ± 13.5
26.2 ± 11.2
26.6 ± 12.2
Median (range)
26.6 (6.3–56.4)
27.0 (6.5–54.2)
26.9 (6.3–56.4)
Prophylaxis dose/y, IU/kg
Mean ± SD
2300.1 ± 1396.5
3834.3 ± 1768.9
3139.0 ± 1778.8
Median (range)
1750.3 (594.8–5318.1)
3732.8 (890.7–8687.3)
2930.6 (594.8–8687.3)
Interruptions in prophylaxis treatment, defined as periods of ≥28 days without any prophylaxis injection, were excluded from the main analysis. †Median time per patient between 2 prophylaxis infusions was analyzed.
