Adoption of emicizumab for hemophilia A in europe: Data from the 2020 european association for haemophilia and allied disorders survey

ABSTRACT

Background: Emicizumab is a bispecific monoclonal antibody, mimicking coagulation factor (F) VIII by linking factor IXa and X and thereby restoring steady-state hemostasis in patients with hemophilia A. It is administered subcutaneously and has been approved for use in hemophilia A patients with and without inhibitors. Aims: To assess availability, acceptance and safety of emicizumab for hemophilia A in Europe. Methods: An online questionnaire was sent to 144 European Haemophilia Comprehensive Care Centers (EHCCC) and European Haemophilia Treatment Centers (EHTC). The survey was open from November 17, 2020 to January 31, 2021. Descriptive statistics were performed using SPSS version 27. Results: Forty-six physicians from 21 countries responded. Emicizumab is reimbursed for all patients with inhibitors and the majority of patients without inhibitors. Reducing treatment burden was the main reason to switch both inhibitor and non-inhibitor patients to emicizumab. An annualized bleeding rate (ABR) of zero could be achieved in most patients with inhibitors on emicizumab (72.9%). Inhibitor titers were regularly monitored in 78.4% of inhibitor patients on emicizumab and chromogenic FVIII assay was not available in 27.0% of centers. Hemostasis was satisfactory in the majority of minor (93.7%) and major (90.7%) surgical procedures performed while on emicizumab. In the 35 centers that responded to the question, a total of four patients on emicizumab have died to date, although none of these deaths were directly linked to emicizumab. The Covid-19 pandemic did not have a considerable impact on the adoption of emicizumab in most centers (64.9%). Conclusions: Three years after its rollout in Europe, emicizumab is widely available for both inhibitor and non-inhibitor patients. Satisfaction of physicians with emicizumab is very good. Unmet needs include availability of laboratory assays and guidelines for cotreatment during surgery and in case of breakthrough bleeding.