Monthly Roundup

Emicizumab, the only approved factor VIII mimetic, has been shown to be effective for all ages and severities of hemophilia A. The HAVEN 7 study, which evaluated emicizumab in infants under 12 months, demonstrated a low annualized treated bleed rate of 0.4, with no new safety concerns. The study confirmed that emicizumab can be used to initiate prophylaxis from the point of hemophilia A diagnosis, potentially reducing bleeding risks, including intracranial hemorrhages. Several other factor VIII mimetics are in preclinical stages and the efficacy and safety data are eagerly anticipated.

Rebalancing agents, which block natural inhibitors of coagulation, have effectively reduced annualized bleeding rates in patients with hemophilia A and B. However, some adverse events, such as thromboembolic events and elevated liver enzymes, were observed. These issues decreased after making dose-specific adjustments.