Current and Future Options of Haemophilia A Treatments

Authors: Miesbach W; Eladly, F.

Affiliations: Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany; Department of Haemostaseology and Haemophilia Center, Internal Medicine, Frankfurt, Germany.

Publication: Expert Opinion on Biological Therapy; 2021. 1–8 ; April 2021

Abstract: INTRODUCTION: The standard treatment of hemophilia A consists of the prophylactic administration of a coagulation factor concentrate, to be administered intravenously several times a week. Newly approved factor concentrates, and non-factor products reduce the frequency of injection and offer better protection against bleeding. AREAS COVERED: New treatment options for hemophilia A are either coagulation factor concentrates based on innovative active principles extending half-life (EHL) or non-factor products allowing subcutaneous application with an extended half-life, so that their broader application only needs to be made every one to four weeks. Other new therapeutic options are still in clinical studies, such as the inhibition of TFPI (tissue factor pathway inhibitor) or small interfering mRNA molecule against antithrombin and gene therapy for hemophilia A. EXPERT OPINION: It can be expected that patients with hemophilia will benefit significantly from the new treatment options and that the protection against bleeding and joint damage as well as the quality of life will increase. The availability of alternatives to classical replacement therapy will require the development of treatment algorithms for patients with hemophilia. It is still unclear to what extent factor substitution will be challenged by the new therapies as first-line therapy.