Haemophilia A management with emicizumab: A survey of haematologists in the United States

Abstract

Abstract Background Emicizumab is a bispecific monoclonal antibody approved in the United States (US) for the treatment of people with haemophilia A (PwHA) with or without factor VIII (FVIII) inhibitors. Changes to haematologists’ practices since the approval of emicizumab are of interest to the haemophilia A community. Aim To identify the clinical characteristics of PwHA receiving emicizumab (PwHArE) in the real-world setting and gain insight into the disease management practices of haematologists treating PwHArE. Methods In total, 50 haematologists across the US completed a one-time, 30-minute, online, qualitative survey consisting of 55 questions (including 11 screening questions) in May 2019. Haematologists were required to be board-certified in haematology, practising in the US, ≥2 years post-residency experience, and currently treating ≥3 PwHA with emicizumab. Results Haematologists reported their PwHArE were mostly adults (aged ≥18 years; 66%) with severe phenotypes (66%), with and without FVIII inhibitors. Haematologists perceived that PwHArE had similar or better treatment adherence (40% and 50%, respectively) compared with PwHA on other treatments, sought the same or lower levels of routine care (72% and 14%, respectively), and were similarly or more physically active (52% and 32%, respectively). Additionally, most haematologists currently using immune tolerance induction (ITI) in PwHArE reported using lower doses of FVIII (73%) and shorter durations (45%) for ITI. Conclusions Availability of emicizumab has resulted in changes in the care of PwHArE, including bleed management, FVIII monitoring, activity guidance, surgery, and use of ITI. Understanding patterns of disease management can inform clinical care.