A total of 986 BEs was treated with Nuwiq in a prospective, randomized, actively controlled, cross-over, open-label, Phase II study of 22 adult and adolescent PTPs with severe hemophilia A. In total, 416 (42.2%) were minor, 566 (57.4%) were moderate to major and 3 (0.3%) were major to life-threatening. The severity for one BE was unknown. The mean on-demand treatment dose was 32.3 IU/kg. The median (range) number of infusions required to stop a BE was 1.0 (range 1–13). The mean duration of treatment of BEs overall was 1.1 days. The overall efficacy of on-demand treatment was evaluated based on criteria including improvement of objective signs of bleeding, number of infusions required to control the bleeding, and the time until bleeding improvement. The proportion of BEs with successful treatment (rated as “good” or “excellent”) was 94.4% (931/986 BEs). The rate of BEs successfully treated with just 1 or 2 infusions was 96.8% (954/986 BEs).
Efficacy of Nuwiq® in the treatment of bleeding episodes, number of infusions used to treat BEs and doses per infusion in clinical study GENA-01
| Efficacy rating | GENA-01 (n = 22 subjects) |
|---|---|
| Number of BEs | 997 |
| Number of treated BEs | 986 |
| Any BE (N) | 986 |
| Excellent | 60.3% |
| Good | 34.1% |
| Moderate | 5.5% |
| None | – |
| Number of infusions Median (range) | 1.0 (1-13) |
| Dose per infusion (IU/kg) Mean ± SD (range) | 32.3 ± 10.6 (7-61) |
Efficacy rating data are percentages.
BE = bleeding episode; N = number of BEs.
Adapted from Nuwiq Product Monograph
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