Abstract
BAY 94-9027 (damoctocog alfa pegol, Jivi) is an extended-half-life recombinant factor
VIII (rFVIII) shown to be well-tolerated and efficacious in bleeding prevention in
previously treated patients with severe hemophilia A. During the PROTECT VIII study,
prophylaxis patients received BAY 94-9027 at intervals determined based on their bleeding
phenotype, observed during a 10-week run-in treatment period with twice-weekly dosing.
Those with ≤ 1 spontaneous joint or muscle bleed were randomized to either 45 to 60 IU/kg
every 5 days or 60 IU/kg every 7 days; patients could increase dosing frequency to
every 5 days or twice weekly in the case of bleeds. Those enrolled after the randomization
arms were full, and those with ≥ 2 bleeds in the run-in period, received 30 to 40 IU/kg
twice weekly. Patients completing the main study could receive open-label BAY 94-9027
in the extension phase. Dosing regimen, total, and joint annualized bleeding rates
were analyzed over three periods: prestudy, main study, and extension. A total of
80 patients who were on prophylaxis treatment prior to and during the study and had
prior bleed data available were evaluated in this post hoc analysis of PROTECT VIII.
Most patients (> 80%) required fewer infusions with BAY 94-9027 prophylaxis versus
their previous standard-half-life (SHL) rFVIII product. Lower bleeding and joint bleeding
rates were observed over time from the prestudy to the extension study period in all
treatment regimens. Compared with SHL FVIII, BAY 94-9027 prophylaxis allows patients
to reduce infusion frequency with maintained or improved protection from bleeds.
Keywords
annualized bleeding rate - hemophilia A - prophylaxis - recombinant factor VIII -
target joint
