Results from the emicizumab clinical trial program (HAVEN 1-4)
| Clinical trial . | Population . | ABR, model based (95% CI) . | ABR, treated bleeds: emicizumab prophylaxis vs no prophylaxis . | Patients with 0 treated bleeds (%) . | ABR, treated bleeds: emicizumab prophylaxis vs prior prophylaxis in NIS . |
|---|---|---|---|---|---|
| HAVEN 1 (NCT02622321) | HA ≥ 12 y with FVIII inhibitors | 2.9 (1.7-5.0) (QW) | 87% reduction (QW) | 63 (QW), 6 (no prophylaxis) | 79% reduction with emicizumab QW vs prior BPA prophylaxis |
| HAVEN 2 (NCT02795767) | HA < 12 y with FVIII inhibitors | 0.2 (0.06-0.62) (QW) | N/A (no comparator) | 87 (QW) | 99% reduction with emicizumab QW vs prior BPA prophylaxis |
| HAVEN 3 (NCT02847637) | HA ≥ 12 y without FVIII inhibitors | 1.5 (0.9-2.5) (QW), 1.3 (0.8-2.3) (Q2W) | 96% reduction (QW), 97% reduction (Q2W) | 56 (QW), 60 (Q2W), 0 (no prophylaxis) | 68% reduction with emicizumab QW vs prior FVIII prophylaxis |
| HAVEN 4 (NCT03020160) | HA ≥ 12 y with or without FVIII inhibitors | 2.4 (1.4-4.3) (Q4W) | N/A (no comparator) | 56% (Q4W) | N/A (no comparator) |
| Clinical trial . | Population . | ABR, model based (95% CI) . | ABR, treated bleeds: emicizumab prophylaxis vs no prophylaxis . | Patients with 0 treated bleeds (%) . | ABR, treated bleeds: emicizumab prophylaxis vs prior prophylaxis in NIS . |
|---|---|---|---|---|---|
| HAVEN 1 (NCT02622321) | HA ≥ 12 y with FVIII inhibitors | 2.9 (1.7-5.0) (QW) | 87% reduction (QW) | 63 (QW), 6 (no prophylaxis) | 79% reduction with emicizumab QW vs prior BPA prophylaxis |
| HAVEN 2 (NCT02795767) | HA < 12 y with FVIII inhibitors | 0.2 (0.06-0.62) (QW) | N/A (no comparator) | 87 (QW) | 99% reduction with emicizumab QW vs prior BPA prophylaxis |
| HAVEN 3 (NCT02847637) | HA ≥ 12 y without FVIII inhibitors | 1.5 (0.9-2.5) (QW), 1.3 (0.8-2.3) (Q2W) | 96% reduction (QW), 97% reduction (Q2W) | 56 (QW), 60 (Q2W), 0 (no prophylaxis) | 68% reduction with emicizumab QW vs prior FVIII prophylaxis |
| HAVEN 4 (NCT03020160) | HA ≥ 12 y with or without FVIII inhibitors | 2.4 (1.4-4.3) (Q4W) | N/A (no comparator) | 56% (Q4W) | N/A (no comparator) |
N/A, not applicable; NIS, nonintervention study; QW, once weekly; Q2W, every 2 weeks; Q4W, every 4 weeks.