Initial experience of Emicizumab prophylaxis in children with inhibitor positive severe hemophilia A from a single center in India

摘要

BackgroundAlthough improvement in the access to clotting factor concentrates and provision of comprehensive care has dramatically improved the quality of life of patients with severe hemophilia, inhibitor-positive patients still develop significant morbidity in terms of joint involvement, progression to disability and life threatening bleeds due to poor access to bypassing agents and immune tolerance induction.ObjectiveTo analyze the response of patients with severe hemophilia A and high-titer inhibitors started on Emicizumab prophylaxis.MethodsPatients with inhibitor-positive severe Hemophilia A were followed up for bleed frequency, joint involvement and health-related quality-of-life assessment (HR-QOL) using PedHAL questionnaire. The data from 1 year prior to start of Emicizumab or from diagnosis of inhibitor status (if shorter period) was compared to that 4 weeks after start of the drug. Emicizumab was administered as per standard guidelines.Results4 patients were initiated on Emicizumab prophylaxis and followed for a median period of 34 weeks (19–57 weeks). The average bleed frequency reduced from 2 to 3.5 per month to 0 after initiation of prophylaxis. No significant adverse events were reported. Improvement in raw and normalized data for all 7 domains of PedHAL which measures activities of daily living was observed. Functional improvement was noted as early as 4 weeks into treatment.ConclusionEmicizumab is a promising agent for prophylaxis of patients with severe hemophilia A and high titre inhibitors.