Safety and efficacy of BAY 94-9027, a prolonged-half-life factor VIII

Safety and efficacy of BAY 94-9027, a prolonged-half-life factor VIII
This study is a pharmaceutical company sponsored trial with the aim to assess the efficacy and safety of a recombinant factor VIII (FVIII) product with prolonged-half-life for prophylaxis and treatment of bleeds in patients with severe hemophilia A. This is a multinational, phase 2/3, partially randomized, open-label trial.
The study included 132 patients with severe haemophilia A, aged 12-65 years. Patients were treated in three individually tailored dose regimens for prophylaxis (according to individual bleeding rate either twice weekly if >1 bleed present in the run-in phase of the study, or randomised to once every 5 days or once every 7 days if no or one bleed occurred in the initial stage). Patients also received the product on demand if there was bleeding. The study period was 10 weeks run-in period followed by 26 weeks of treatment. The study outcome was annualized bleeding rate, response to treatment of bleeds (excellent, good, moderate or poor) and the number of infusions to treat bleeds.
The prolonged half-life of BAY 94-9027 resulted in effective prophylaxis at dose intervals up to every 7 days, given it being tailored to the patient's individual bleeding tendency. It was also effective in treating acute bleeds. No patient developed inhibitors to FVIII during the study.
Open-label design and subjective nature of the patient-reported assessment of treatment of bleeds. Study period may not be long enough to detect development of inhibitors.
New factor VIII products with prolonged half life can offer some hemophilia A patients the convenience of less frequent prophylaxis, which may improve the compliance and quality of life. Their benefits, however, will have to be weighed against their cost.