Consumption of BPA/CFC for treatment of breakthrough bleeds (EAS 1)
| Event . | Inhibitor . | Noninhibitor . | ||||||
|---|---|---|---|---|---|---|---|---|
| BPA prophylaxis (n = 19) . | Fitusiran 80 mg prophylaxis (n = 19) . | CFC prophylaxis (n = 46) . | Fitusiran 80 mg prophylaxis (n = 46) . | |||||
| aPCC (U/kg) . | rFVIIa (μg/kg) . | aPCC (U/kg) . | rFVIIa (μg/kg) . | FVIII (IU/kg)∗ . | FIX (IU/kg)† . | FVIII (IU/kg)∗ . | FIX (IU/kg)† . | |
| Mean annualized weigh-adjusted consumption, unit (SD) | 2353.0 (4317.0) | 7468.7 (5756.6) | 25.6 (82.6) | 236.4 (327.0) | 294.5 (366.6) | 288.6 (402.9) | 60.7 (148.3) | 17.8 (56.1) |
| Mean total weight-adjusted dose per bleed, unit (SD) | 199.8 (366.1) | 709.9 (1163.8) | 34.1 (16.1) | 38.2 (17.0) | 45.3 (41.8) | 73.6 (54.7) | 13.4 (5.5) | 26.2 (0.0) |
| Total number of injections, n | 260 | 159 | 7 | 19 | 159 | 30 | 56 | 3 |
| Total number of treated bleeds, n | 77 | 24 | 5 | 13 | 108 | 18 | 51 | 3 |
| Event . | Inhibitor . | Noninhibitor . | ||||||
|---|---|---|---|---|---|---|---|---|
| BPA prophylaxis (n = 19) . | Fitusiran 80 mg prophylaxis (n = 19) . | CFC prophylaxis (n = 46) . | Fitusiran 80 mg prophylaxis (n = 46) . | |||||
| aPCC (U/kg) . | rFVIIa (μg/kg) . | aPCC (U/kg) . | rFVIIa (μg/kg) . | FVIII (IU/kg)∗ . | FIX (IU/kg)† . | FVIII (IU/kg)∗ . | FIX (IU/kg)† . | |
| Mean annualized weigh-adjusted consumption, unit (SD) | 2353.0 (4317.0) | 7468.7 (5756.6) | 25.6 (82.6) | 236.4 (327.0) | 294.5 (366.6) | 288.6 (402.9) | 60.7 (148.3) | 17.8 (56.1) |
| Mean total weight-adjusted dose per bleed, unit (SD) | 199.8 (366.1) | 709.9 (1163.8) | 34.1 (16.1) | 38.2 (17.0) | 45.3 (41.8) | 73.6 (54.7) | 13.4 (5.5) | 26.2 (0.0) |
| Total number of injections, n | 260 | 159 | 7 | 19 | 159 | 30 | 56 | 3 |
| Total number of treated bleeds, n | 77 | 24 | 5 | 13 | 108 | 18 | 51 | 3 |
EAS 1 includes all participants who received BPA/CFC prophylaxis and at least 1 dose of 80 mg fitusiran before dose resumption (after the sponsor initiated pause in dosing).
aPCC, prothrombin complex concentrate; EHL, extended half-life; rFVIIa, recombinant activated factor VII; SHL, standard half-life.
SHL products.
Because of the very limited number of participants with FIX EHL product consumption for treatment of breakthrough bleeds (only 1 participant received FIX EHL), further distinction between SHL and EHL product consumption for breakthrough bleeds treatment was not feasible in this study.