Patients: Previously treated males (n=68) aged <12 years with severe hemophilia A (FVIII activity level of <1%) and a body weight ≥10 kg. Patients aged 0–5 years had at least 50 exposure days to other FVIII products. Patients aged 6–11 years had at least 150 exposure days.
Study design: Multinational, open-label, non-randomized, non-controlled trial evaluating the safety, clinical efficacy, and pharmacokinetics of prophylactic and treatment on demand of Esperoct®. For bleeding episodes, treatment dose was 20–75 IU/kg, depending on bleed severity and location. For prophylaxis, patients administered 50–75 IU/kg twice weekly for 26 weeks.
Prior to the start of routine prophylaxis, single-dose PK comparison was performed in 27 children between previous SHL products and Esperoct® at the same administered dose. Half-life comparison is based on estimated terminal half-life ratio using a population-based method.
Primary endpoint: Incidence of inhibitory antibodies against FVIII, indicated by a result of ≥0.6 BU (Bethesda units), as measured with the Nijmegen-modified Bethesda assay with heat inactivation of residual FVIII activity.
Secondary endpoint: Frequency of adverse events, including serious adverse effects, inhibitor formation against FVIII, allergic reactions, thromboembolic events, and medication errors.