الفهرس 
Assessment of prophylaxis on the outcome of severe hemophilia patients with and without inhibitors
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Abstract
Background: Deficiency of factor VIII cause hemophilia A. On-demand or preventive treatments replace clotting factors. Prophylaxis inhibits spontaneous joint and other hemophilia bleeding. PWH were given clotting factor concentrates may develop ”inhibitors”. Musculoskeletal damage is the most common PWH complication, as measured by the WFH physical score, HEAD-US, and MRI Denver score. Ultrasound checks healing, rebleeding, synovitis, and cartilage degeneration. Aim of work: Assessing individuals with severe Hemophilia with or without inhibitors before and after prophylaxis to assess its influence on joint state, and QOL. Subjects and Methods: This observational prospective study was carried out at Pediatric Hematology Unit, Cairo University Children Hospital. The study had included 39 patients of severe hemophilia A patients with and without inhibitors on prophylaxis therapy and followed up for a minimum of 6 months duration. ISTHBAT, haem QOL, HEAD-US, MRI Denver score were done at baseline, 3months, 6months respectively. Prophylaxis Regimen: Hemophilia A patients with high titer inhibitors (˃5 Bethesda units) received Emicizumab therapy subcutaneous loading dose (3mg/kg/dose) once weekly for first 4 weeks then maintenance dose (3mg/kg/dose) every 2 weeks while HA patients with inhibitor titer ≤5 Bethesda units received double dose of FVIII concentrate, patients negative for inhibitors were on prophylaxis with recombinant FVIII concentrates biweekly (25-30 IU/kg/dose). Results: There was a statistically significance improvement in ISTHBAT bleeding score, Haem QOL and Musculoskeletal score among patients on Emicizumab and Recombinant FVIII concentrate prophylaxis. Conclusion: Emicizumab and Recombinant FVIII concentrate had a positive impact with preserving joint status and improving quality of life in PWH. However, Emicizumab is more efficacious as patients on Emicizumab prophylaxis showed zero hospital admissions and zero annual bleeding rates after starting on it while patients receiving Recombinant FVIII concentrate showed decreased annual bleeding rate but not zero. Prophylactic therapy should be considered as standard of care even in countries with low resources.