PB0530 - Joint Health and Quality of Life in Children with Hemophilia A with Inhibitor on Emicizumab prophylaxis

Background: Emicizumab is a novel nonfactor VIII product developed to address the unmet needs of clotting factor replacement therapy and has become the benchmark for optimal prophylaxis in managing patients with hemophilia A with or without inhibitors. However, real-world data and study data regarding prophylactic therapy with emicizumab in pediatric patients with hemophilia A with inhibitors is scarce in Egypt.

Aims: to assess the efficacy of emicizumab prophylaxis on the joint health and quality of life in children with hemophilia A with inhibitors

Methods: This prospective study included 37 children with severe hemophilia A with inhibitors who received emicizumab prophylaxis for more than 1 year.
The median age of patients included in the study years was 7 (Interquartile range3-12) years. Full medical history and meticulous clinical examination was done. In addition, Annual bleeding rates (ABR), Hemophilia Joint Health Score (HJHS), Functional Independence Score in Hemophilia (FISH) and EQ-5D-Y dimensions were assessed before receiving emicizumab prophylaxis and after 12 months of regular emicizumab prophylaxis

Results: A significant reduction was observed in median ABR, from 40 (IQR 36-48) to 0 (IQR 0.0-2), all bleeding were trauma related and half of them result from hemarthrosis. HJHS, markedly improved from median 16 (IQR 4-29) to 9 (IQR 4-14). Moreover, there is statistically significant improvement in FISH score after one year of prophylactic therapy with emicizumab in hemophilic children (p-value < 0.001).Patients quality of life was estimated with EQ-5D Y, after one year of emicizumab prophylaxis, most patients were independent in their daily life(n=21,72%) only 20% were unhappy and have some anxiety and 56% of the children have no pain. In general, there is marked improvement of the five dimensions of health profiles in the EQ-5D questionnaire.

Conclusion(s): Emicizumab prophylaxis markedly decreases bleeding episodes, improves the quality of life and joint status in children with severe hemophilia A with inhibitors.