Patients: Previously treated males (n=160) with severe hemophilia A (FVIII activity <1%) participating in pathfinder2 (n=102) and pathfinder5 (n=58) trials.
Study design: Multicenter, multinational, open-label, nonrandomized, interventional trial. Patients received Esperoct® prophylaxis dosed every 7 days (Q7D), twice weekly (BIW), or three times weekly (TIW), with dosing interval based on previous regimen and bleeding tendency. All patients on the Q7D regimen were aged ≥12 years, and the dose was 75 IU/kg. Dosing for BIW was 50 IU/kg (patients aged ≥12 years) or 60 IU/kg (patients aged <12 years). TIW dosing was 50 IU/kg (all ages).
Primary endpoint: Number of reported adverse events.
Secondary endpoints: Efficacy endpoints included number of treated bleeds on prophylaxis, pre-dose FVIII activity levels on prophylaxis, and treatment satisfaction. A secondary safety endpoint was the incidence of FVIII inhibitors.