Clinical outcomes and safety profile of emicizumab and other treatment options for the management of children and adults with severe haemophilia A in the UAE: A retrospective study at two tertiary hospitals

Abstract Introduction More data on the clinical outcomes of emicizumab and other treatment options would guide the appropriate positioning of these agents among haemophilia management approaches. The purpose of this study was to review the clinical outcomes of emicizumab prophylaxis and other treatment options in children and adults with severe haemophilia A (HA) at Sheikh Khalifa Medical City (SKMC) and Tawam Hospital in the United Arab Emirates (UAE). Methods This retrospective study consisted of charts review of all individuals with HA diagnosed and treated with emicizumab and other treatment options at both centres between January 2019 and December 2023. The primary endpoint was annualised bleeding rate (ABR) from baseline. Secondary endpoints included the presence of FVIII inhibitors, joint bleed episodes, health-related quality of life (HRQoL), need for rescue medication, and Emergency Department visits at Month 12 after switching to emicizumab. Results All persons were males; mean age was 16.8±11.4 years. Mean duration of emicizumab therapy was 11±2.3 months. The ABR decreased significantly after therapy initiation (2.93±2.48 versus 0.0±0.0; p<0.001). None of the persons required rescue medication during emicizumab therapy (p<0.001) and FVIII inhibitors significantly decreased (p<0.001). After emicizumab therapy, number of Emergency Department visits decreased significantly (3.3±2.8 versus 0.1±0.3; p <0.001), no joint bleed episodes had been reported (p<0.001) and HRQoL index scores were significantly improved (p<0.001). Conclusion Emicizumab was associated with improved clinical outcomes in terms of ABR, joint bleeding episodes, use of rescue medications, and FVIII inhibitors level. Emicizumab prophylaxis also improved HRQoL.