Real-World Experience With Emicizumab for Haemophilia A From the Physician Perspective in a Real-World Clinical Setting.

INTRODUCTION: Emicizumab is a bispecific antibody approved for prophylaxis in people with haemophilia A (PwHA). AIM: To describe from the physician perspective the characteristics and treatment experiences of PwHA receiving emicizumab. METHODS: The Adelphi Real World Haemophilia Disease Specific Programme is a cross-sectional, retrospective data collection from US haematology and haematology-oncology specialists between July 2023 and April 2024. Male PwHA with or without inhibitors were included if they had baseline factor VIII levels <5% and were not currently participating in a clinical trial. Surveys captured demographic characteristics and clinical outcomes, including bleeding events. RESULTS: Fifty-four physicians provided data for 293 PwHA, of which 115 were seen outside federally funded haemophilia treatment centres (HTCs), 77 were treated with emicizumab and 49 had pre- and post-emicizumab initiation data available. More Black, African American, African or Caribbean PwHA received emicizumab outside federally funded HTCs than at federally funded HTCs (46.2% vs. 16.7%). Most emicizumab doses were taken on time (mean [SD], 90.7% [14.6%]), and 64.0% of PwHA took >80% of the prescribed dose. More PwHA had zero treated bleeds (76.9% vs. 42.9%), with fewer bleeding events (mean [SD], 1.3 [1.9] vs. 0.4 [1.1]; p = 0.0001) during the post-emicizumab period (median [IQR] follow-up, 12.0 [1.3-12.0] months) than in the 12 months pre-emicizumab. Post-emicizumab outcomes were similarly improved across settings, whether at federally funded HTCs or elsewhere. CONCLUSION: Among PwHA receiving emicizumab, rates and severity of bleeds decreased after emicizumab initiation, regardless of the clinical setting.