Safety evaluation of emicizumab prophylaxis in individuals with haemophilia A.

Knowledge gaps include the efficacy and safety of emicizumab in younger age groups and those with mild or moderate hemophilia A. Future directions for research include exploring the risk of inhibitor recurrence in patients with a history of high titer inhibitor who have been successfully tolerized, who switch from factor prophylaxis to emicizumab, as well as conducting 'real world studies' to evaluate the patient's perception of emicizumab in regard to ease and tolerability in order to optimize individualized treatment plans.