Background: Hemophilia A is a congenital bleeding disorder characterized by factor VIII (FVIII) deficiency, leading to recurrent bleeding and joint damage. Emicizumab, a bispecific monoclonal antibody mimicking FVIII function, offers prophylaxis through subcutaneous administration, effective even in inhibitor-positive patients. Objective: To evaluate the efficacy, safety, and tolerability of emicizumab prophylaxis compared to conventional factor VIII therapy in patients with hemophilia A and comparative cost analysis. Methods: This was a prospective, randomized, controlled, non-blinded clinical trial conducted at a tertiary care center in Rajasthan, India. Sixty-six patients with confirmed hemophilia A were randomized into two equal groups: Group A received emicizumab (loading: 3 mg/kg weekly × 4 weeks; then maintenance: 6 mg/kg every 4 weeks), and Group B continued on-demand factor VIII therapy. Primary outcomes included annualized bleeding rate (ABR) and activated partial thromboplastin time (aPTT) changes over six months. Secondary outcomes included joint bleed incidence and adverse events. Results: Group A demonstrated a significant reduction in mean ABR ( from 17 ± 9.25 to 0.03 ± 0.17)compared to Group B (p<0.001). Mean aPTT values normalized in Group A (from 122.88 ± 16.54 s to 29.95 ± 7.03 s; p < 0.001) but remained prolonged in Group B. Adverse events in Group A were minimal, with only mild injection site reactions reported. Conclusion: Emicizumab prophylaxis significantly reduces bleeding rates and improves joint health in patients with hemophilia A, with a favorable safety profile, and is feasible in government-sponsored healthcare settings in India.

Emicizumab, factor VIII inhibitors,Hemophilia A,India, prophylaxis