To the Editor: Hemophilia A (HA) is an X-linked hereditary bleeding disorder caused by deficiency of coagulation factor (F) VIII activity. Emicizumab is a bispecific monoclonal antibody that mimics the cofactor function of activated FVIII and prevents bleeds in patients with hemophilia A regardless of inhibitor status. Emicizumab prophylaxis is expected to reduce the risk of severe bleeds with their subsequent complications in such patients. However, data about its safety and efficacy in Egyptian patients with hemophilia A is limited. We conducted a prospective cohort study on 88 hemophilia A patients to evaluate safety and efficacy of Emicizumab prophylaxis in them. We found a successful reduction in annualized bleeding rate during Emicizumab prophylaxis where the median annualized bleeding rate was 48 before Emicizumab vs. 0 after Emicizumab prophylaxis. which is comparable to previous studies [
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4]. In addition, all adverse events that developed during Emicizumab prophylaxis were mild and resolved without any treatment. No thrombotic events were reported in our study cohort. This is in agreement with an another study that reported the most frequent treatment-related adverse event as injection-site reaction with no thrombotic events or development of de-novo antidrug antibodies [
3]. We conclude that safety and efficacy data of Emicizumab are consistent with the findings of previous studies in hemophilia A patients. Emicizumab was effective in terms of reduction of annual bleeding rate where the majority of patients had 0 treated bleeds. Also, Emicizumab continued to demonstrate a favorable safety profile, with no discontinuations due to adverse events. Emicizumab prophylaxis seems to be an effective as well as safe treatment option for patients with hemophilia A. …
