Adoption of emicizumab (Hemlibra®) for hemophilia A in Europe: Data from the 2020 European Association for Haemophilia and Allied Disorders survey

Abstract

Background Emicizumab, a bispecific monoclonal antibody administered subcutaneously, mimicking the action of activated coagulation factor VIII, has been approved in Europe for use in patients with severe hemophilia of all ages. Aims To assess availability, acceptance, adverse events, efficacy and laboratory monitoring of emicizumab and the effect of the coronavirus disease 2019 (COVID‐19) pandemic on its use. Methods Online questionnaire sent to 144 hemophilia treatment centres (November 2020 to January 2021). Results Forty‐six physicians from 21 countries responded, with a total of 3420 patients with severe HA under their care. Emicizumab was widely available, for 100% of inhibitor patients and 88% of non‐inhibitor patients. No major adverse events were reported. Four reported deaths in patients on emicizumab were not thought to be related to emicizumab. An annualized bleeding rate (ABR) of zero was achieved in 73% of inhibitors patients. Haemostasis was satisfactory in the majority of minor (93.7%) and major (90.7%) surgical procedures performed while on emicizumab. Inhibitor titers were monitored in 78.4% of inhibitor patients on emicizumab, but chromogenic FVIII assay was only available in 73% of centres. The COVID‐19 pandemic did not have a major impact on the adoption of emicizumab in most centres (64.9%). Conclusion Three years after its rollout in Europe, emicizumab is widely available. Clinical efficacy and safety were evaluated to be very good, keeping in mind the inherent limitations of the study. Unmet needs include establishment of treatment guidelines for surgery and breakthrough bleeding, limited expertise, especially in young children, and availability of laboratory assays.

Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A

“…( 9 ) In your opinion, which future PPX therapies have the potential to become mainstream choices for people with hemophilia A without inhibitors? ( 10 ) If EHL FVIII concentrate was available at the same payer price as SHL FVIII, would you consider switching a well-controlled person with hemophilia A to EHL? and ( 11 ) In your opinion, how can QoL be improved in the future for people with hemophilia A?…”

“…In 2018, the first non-factor replacement therapy, emicizumab, was approved for use in Europe for long-term PPX in people of all ages who have congenital hemophilia A with FVIII inhibitors or severe congenital hemophilia A (FVIII<1%) without FVIII inhibitors [ 4 , 9 , 10 ]. Emicizumab, a bispecific monoclonal antibody that mimics the activity of FVIII by binding activated factors IX and X, maintains a level of hemostatic activity estimated at 9–10% of FVIII activity [ 11 , 12 ] and thus offers the potential for a clinically meaningful reduction of bleeding episodes in patients with hemophilia A who have developed inhibitors compared to on-demand/prophylactic use of BPAs [ 4 , 9 , 10 ]. Its weekly subcutaneous dosing schedule has been reported to provide health-related quality of life (QoL) and health status benefits [ 9 , 13 ].…”

Impact of novel hemophilia therapies around the world

“…The Humanitarian Aid Program has distributed 40 673 500 units of EHL FVIII and FIX products to 37 and 18 countries, respectively 72 . Recently, Krumb et al 73 published data on the adoption of emicizumab in Europe. Of the 144 contacted hemophilia treatment centers, 46 responded to the survey, representing 21 countries.…”

Emicizumab treatment in chronic intermittent haemodialysis

“…Although emicizumab is widely used for bleeding prophylaxis to date 4 there are still limited data about the use of emicizumab in patients with comorbidities, 5 particularly in patients with advanced CKD with or without kidney replacement therapy.…”