^*Rated by the investigator at 3-month intervals on a 3-point scale: excellent (prophylaxis treatment completely prevented spontaneous musculoskeletal bleeding, no change in dosing regimen necessary) effective (adequately prevented spontaneous musculoskeletal bleeding episodes as demonstrated by a lower than expected incidence of spontaneous musculoskeletal bleeding episodes); or inadequate (inadequate prevention of bleeding requiring a change in dosing regimen).^2
†The response to secondary prophylaxis was assessed once every 3 months by the investigator using a 3-point scale: excellent (no spontaneous bleeding of any kind, no change in dosing regimen necessary); effective (no spontaneous musculoskeletal bleeding, no change in dosing regimen necessary) or inadequate (inadequate prevention of bleeding requiring a change in dosing regimen).³

FIX: factor IX; PTP: previously treated patient; PUP: previously untreated patient.

References:

1. Benefix Egyptian Drug Authority leaflet approval date: 14/11/2021. Revision Date: November 2020.
2. Shapiro AD, Di Paola J, Cohen A, et al. The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B. Blood. 2005;105(2):518-525.
3. Roth DA, Kessler CM, Pasi KJ, et al. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood. 2001;98(13):3600-3606.
4. Monahan PE, Leisner R, Sullivan ST, Ramirez ME, Kelly P, Roth DA. Safety and efficacy of investigator prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia B, Haemophilia. 2010;16(3):460-468.