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

Emicizumab is a bispecific antibody approved for prophylaxis in people with haemophilia A (PwHA).

To describe from the physician perspective the characteristics and treatment experiences of PwHA receiving emicizumab.

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.

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.

Among PwHA receiving emicizumab, rates and severity of bleeds decreased after emicizumab initiation, regardless of the clinical setting.