Nehal Patel¹, Ghosha Pandav¹, Rashmi Thanvi¹
1- GMERS Medical College and Hospital, India
Abstract Body
Background: Emicizumab prophylaxis has significantly improved bleed prevention in Hemophilia A through subcutaneous administration. However, real-world longitudinal data, especially from resource-constrained settings, remain limited.
Aim: To evaluate clinical and functional outcomes in 43 patients with Hemophilia A receiving Emicizumab prophylaxis, assessing changes in Annualized Bleeding Rate (ABR), target joints, Functional Independence Score in Hemophilia (FISH), Hemophilia Joint Health Score (HJHS), and EQ-VAS quality-of-life scores across varying treatment durations.
Methods: In this prospective observational study (July 2023–December 2025), 43 patients received Emicizumab at a modified maintenance dose of 1.5 mg/kg every two weeks. Clinical and functional parameters were recorded at baseline and follow-up. Patients were stratified by duration of prophylaxis for subgroup analysis.
Results: Emicizumab prophylaxis demonstrated a substantial reduction in bleeding rates across all groups. Overall ABR decreased from 18.5 to 0.44 (P value < 0.001). 100% reduction in target joint bleeds. Mean FISH increased from 23.84 to 30.7(P value < 0.001) indicating enhanced functional independence. The HJHS improved from 17 .5 to 1.7 (P value < 0.001). Quality of life improved significantly , with EQ-VAS scores rising from 45% to 91.1% (P value