Table 2.
Efficacy of on-demand treatment with nonacog beta pegol in previously treated patients with haemophilia B in phase 3 trials
| Treatment | No. of ptsa | Pts with bleeds (%)b | No. of bleeds | Type/location of bleeds (%) | Bleeds requiring 1, 2, 3, ≥ 4 injections to treat (%) | Successful HSRc (%) |
||
|---|---|---|---|---|---|---|---|---|
| Spontaneous | Traumatic | Joint | ||||||
| Paradigm 2 [8] | ||||||||
| PRX 10 IU/kg qwd | 30 | 83.3 | 132 | 68.9 | 29.5 | 81.1 | 84.1, 12.1, 1.5, 2.3 | 86.9 |
| PRX 40 IU/kg qwd | 29 | 55.2 | 70 | 48.6 | 51.4 | 78.3 | 98.6, 0, 1.4, 0 | 97.1 |
| On-demand | 15 | 93.3 | 143 | 71.3 | 28.7 | 76.4 | 83.9, 14.0, 0, 2.1 | 95.1 |
| Total | 74 | 74.3 | 345 | 65.8 | 33.6 | 78.5 | 87.0, 10.4, 0.9, 1.7 | 92.4e |
| Paradigm 4 [15] | ||||||||
| PRX 10 IU/kg qw | 21 | 66.7 | 35 | 71.4 | 28.6 | 74.3 | 82.9, 14.3, 2.9, 0 | 97.1 |
| PRX 40 IU/kg qw | 52 | 59.6 | 98 | 36.7 | 57.1 | 73.5 | 84.7, 10.2, 4.1, 1.0 | 94.8 |
| PRX 80 IU/kg q2w | 2 | 50.0 | 1 | 0 | 100.0 | 100.0 | 100.0, 0, 0, 0 | 100.0 |
| On-demand | 5 | 100.0 | 73 | 93.2 | 6.8 | 79.5 | 94.5, 5.5, 0, 0 | 93.1 |
| Total | 71 | 69.0 | 207 | 62.3 | 34.8 | 75.8 | 87.9, 9.2, 2.4, 0.5 | 94.6 |
See text for trial design details
HSR haemostatic response, PRX prophylaxis, pts patients, qw once weekly, q2w every second week
aNumber of pts randomized/assigned (Paradigm 2) or treated at any time during the trial (Paradigm 4)
bIn Paradigm 4, numbers are based on the treatment group at the time of each bleeding episode
cHSR was rated by the patients as follows: excellent = abrupt pain relief and/or clear improvement in objective signs of bleeding within 8 h after a single injection; good = noticeable pain relief and/or improvement in signs of bleeding within 8 h after a single injection; moderate = probable or slight beneficial effect within the first 8 h after the first injection but requiring more than a single injection within 8 h; poor = either no improvement or worsening of symptoms within 8 h after 2 injections. Successful HSR was defined as an excellent or good HSR while a moderate or poor HSR was considered as failed HSR. Bleeding episodes with missing HSR were not included in the calculation
dRandomized treatment groups
eObserved rate, which is slightly higher than the estimated rate reported in text