Clinical assessment of Optivate®, a high-purity concentrate of factor VIII (FVIII) with von Willebrand factor (VWF), in the management of patients with haemophilia A

International audience; Factor VIII concentrates have revolutionised the treatment of patients with haemophilia A. Concerns over the transmission of viral infections through these products have been addressed through stringent, donor screening procedures and robust antiviral manufacturing steps. BPL has developed a high-purity FVIII product with von Willebrand factor (VWF), Optivate®. Its safety, tolerability and efficacy as prophylaxis and treatment of bleeds has been established in long term studies. Seventy previously treated patients with severe haemophilia A, with > 20 exposure days (ED), were recruited into two long term, multicentre, open-label studies. The protocols were virtually identical. Patients received Optivate® either prophylactically or on demand. A mean of 159.0 EDs were experienced over 11,320 infusions. Under both conditions, Optivate® was well tolerated. Only 10% of patients experienced a treatment related adverse event (AE); the most commonly reported were headache (4% of patients) and dizziness (3% of patients). The mean number of bleeds/patient over the 2 year treatment period was 23.5 during prophylactic use and 70.4 during on demand use. In patients treated prophylactically, clinical responses to breakthrough bleeds were rated by physician as excellent or good and as very helpful or helpful by patients in 95% of bleeds. Clinical responses by physicians in on-demand patients were rated as excellent or good and helpful or very helpful by the patients for 91% of bleeds. There were no viral transmissions or inhibitors. The studies confirm the clinical efficacy and safety of Optivate® in both prophylactic and on-demand management of patients with haemophilia A.