VPB1164 - Outcome of Emicizumab in Management of Egyptian Children and adolescents with Hemophilia A with inhibitors: A 2 year single center prospective study

Background: Emicizumab (ACE900) has been recently extensively approved for the prophylaxis of HA patients with FVIII-inhibitors of all ages. Though few data(Phase 3 clinical trials and case series) are currently available in children they showed significantly reduced bleeding rates compared to previous treatments and had beneficial effects on health-related quality of life, However real-world patient studies are required to evaluate the long-term outcome of Emicizumab prophylaxis especially in HA children with inhibitors .

Aims: So our study describes real-world experience of Egyptian HA children and adolescents( PwHAs )with high titre inhibitors started on biweekly subcutaneous Emicizumab 3 mg/kg, following a 4-week induction

Methods: Outcome measures of Emicizumab in PwHAs included bleeding rate, ISTH -BAT and objective measures as joint physical examination (WFH musculoskeletal score) and joint imaging (HEAD- US and MRI Denver score of target joints) and HRQOL. Assessment was done initially and every 6 months thereafter over a 2 year period

Results: 14 severe HA male patients with a mean age of 9.2 years were enrolled .35.7% are of a consanguineous marriage and 9(64.2%) have a positive family history . Our enrolled cohort usually had 3 to 4 target joints. On baseline assessment the bleeding rate ranged from 6-8 events/ year and the ISTH –BAT ranged from 6 to 29 and both dropped to zero bleeds after 6 months on biweekly prophylactic Emicizumab in 11(78.6%) patients .Only 3 patients experienced at least one breakthrough bleed but none into target joints .The mean musculoskeletal score was 17 on initial assessment and dropped to 4 on treatment with Emicizumab .HRQOL is a patient-reported outcome that showed significant improvement . Radiological changes on HEAD US and Denver MRI scores were stationary

Conclusion(s): Long term outcome of Emicizumab in our described cohort of PwHAs with inhibitors showed significant improvements in bleeding rate , joint status and HRQOL.