Role of Emicizumab Therapy in treatment of Children with Hemophilia A Inhibitors

Background: Hemophilia A is a hereditary condition that results in a lack of coagulation factor VIII. Emicizumab (ACE910) is a recombinant, humanized, bispecific monoclonal antibody that has been developed as a potential alternative treatment for hemophilia A in patients with inhibitors. Aim: To assess efficacy, safety of Emicizumab in treatment of children with hemophilia A of factor VIII inhibitors. Patients and methods: This prospective study was conducted on twenty patients with hemophilia A Inhibitors, admitted to pediatric department of El Helal insurance hospital, Sohag between 1st September 2023, 28 th February 2024. Results: 45% of patients had a history of serious conditions before starting treatment. Common problems due to the disease included knee joint pain (60%) and elbow joint pain (30%). Positive antibodies to Factor VIII were found in 40% of cases The mean age of starting Emicizumab was ±3.21 years. 70% received Emicizumab due to high antibody titre to factor VIII, 30% due to uncontrolled bleeding, 25% due to no response to factor VIII and 5% received Emicizumab without receiving Factor VIII. 65% of patient had no side effects, 35% had mild skin rash after treatment. 20% experienced serious conditions, including accidents and adenoidectomy operations. Patients visits decreased from three to two visits per month after emicizumab treatment. Conclusion: Emicizumab, a safe, effective treatment hemophilia, has no side effects, but common symptoms include mild bruises and a history of serious conditions.