PB1259 - Emicizumab prophylaxis: a novel alternative therapy for severe Hemophilia A patients with and without inhibitors.

Background: Emicizumab is a humanised, bispecific monoclonal antibody that connects active factor IX and factor X to replace the function of absent activated factor VIII, restoring hemostasis.

Aims: In this study, we assessed the efficacy in terms of bleeding, safety, and quality of life of emicizumab prophylaxis in persons who have hemophilia A (HA) with and without inhibitors.

Methods: In this prospective study, with severe HA patients were recruited from January to November 2022. Nineteen patients were inhibitor positive, while 17 patients were without inhibitors with annual bleeding rate (ABR) 8 or greater and past histories of severe bleeding type. Emicizumab loading dose was 3mg/kg in the first 4 weeks and the maintenance dose was started at week 5 at 6mg/kg/month.The five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) includes; mobility, Self care, usual activities, pain/discomfort, and anxiety/depression were used to evaluate patients’ HRQoL. Furthermore, Hemophilia Joint Health Score (HJHS) and Functional Independence Score in Hemophilia (FISH) scores were applied for the assessment of joints at different time points. Results were analysis by SPSS version 23.

Results: A total of 36 HA male patients with mean age of 19.7±14.42 years were recruited in the study with history of consanguinity in 30(83.3%). Patients clinically presented with common bleeding symptoms includes: hemarthrosis >95%, GI bleeding 13.8 %, bruises and gums bleeding 13.8%, with significant reduction was observed in the bleeding episodes after the therapeutic intervention and joints assessment and EQ VAS analog scores showed a significant improvement in health state after treatment, Table 1. Quality of life was assessed before and after evaluated by EQ-5D-5L questionnaire displayed in Figure 1.

Conclusion(s): Our results suggest that patients on prophylactic treatment with Emicizumab were less restricted and had improved quality of life, specially improved health and social lives. Similarly, it was well tolerated, and no participant discontinued because of adverse events.