PB1319 - Efficacy, safety and satisfaction of using Emicizumab in Hemophilia A patients without Factor VIII Inhibitors: an integrative review

Background: Emicizumab is a bispecific monoclonal antibody that mimics factor VIII (FVIII) and is currently approved for the treatment of Hemophilia A (HA) with and without inhibitors. Emicizumab prophylaxis is considered for non-inhibitor patients with difficult venous access and with repeated bleeding episodes despite adequate prophylaxis with FVIII. There is limited evidence of patient outcomes treated with Emicizumab, particularly without inhibitors.

Aims: To evaluate the efficacy and safety of Emicizumab in HA patients without Factor VIII Inhibitors and their satisfaction with the current standard treatment.

Methods: The present study is an integrative review. The inclusion criteria were full original articles, in Portuguese, English or Spanish about the efficacy, safety and satisfaction of using Emicizumab in HA patients without Factor VIII Inhibitors. The exclusion criteria were Acquired Hemophilia and in vitro studies. The databases used were PubMed, LILACS, SciELO and Cochrane. The following descriptors were used with Boolean operators: (Hemophilia A) OR (Hemophilia A) AND (emicizumab) AND (without inhibitor). Figure 1 presents a flowchart for better elucidation.

Results: The characteristics of the studies included and the results are described in Table 1.

Conclusion(s): Subcutaneous Emicizumab prophylaxis of 3 mg/kg once per week for 4 weeks, followed by either 1.5 mg/kg weekly or 3 mg/kg every two weeks led to a significantly lower Bleeding Rate (BR) than previous factor VIII prophylaxis or no prophylaxis among patients with HA without inhibitors. Despite mild adverse effects, such as breakthrough bleeds and postoperative thrombosis, most HA experienced improved health and satisfaction with the treatment. The results of the analysis indicate that Emicizumab is safe and effective for the prevention of bleeding among infants and adults without FVIII inhibitors.