Abstract
Background. Regular treatment to prevent bleeding and consequent joint deterioration (prophylaxis) is the standard of care for persons with severe hemophilia A, traditionally based on intravenous infusions of the deficient clotting FVIII concentrates (CFCs). In recent years, extended half-life (EHL) CFCs and the non-replacement agent emicizumab, subcutaneously administered, have reduced the treatment burden. Methods. To compare and integrate the opinions on the different therapies available, eight hemophilia specialists were involved in drafting items of interest and relative statements through the Estimate-Talk-Estimate (ETE) method (“mini-Delphi”), in this way reaching consensus. Results. Eighteen items were identified, then harmonized to 10, and a statement was generated for each. These statements highlight the importance of personalized prophylaxis regimens. CFCs, particularly EHL products, seem more suitable for this, despite the challenging intravenous (i.v.) administration. Limited real-world experience, particularly in some clinical settings, and the lack of evidence on long-term safety and efficacy of non-replacement agents, require careful individual risk/benefit assessment and multidisciplinary data collection. Conclusions. The increased treatment options extend the opportunities of personalized prophylaxis, the mainstay of modern management of hemophilia. Close, long-term clinical and laboratory follow-up of patients using newer therapeutic approaches by specialized hemophilia treatment centers is needed.
Highlights
Hemophilia A is a rare X-linked coagulation disorder (1:5000 male live births) caused by gene variants affecting the synthesis or function of factor VIII (FVIII), an essential component of the intrinsic pathway of blood coagulation [1,2]
18 points of interest regarding the treatment of severe hemophilia A patients without inhibitors
Regular long-term prophylaxis to prevent bleeding, preserve joint status, and ensure a quality of life equal to peers without hemophilia is the treatment of choice in patients of all ages with severe hemophilia A or severe hemorrhagic phenotype
Summary
Hemophilia A is a rare X-linked coagulation disorder (1:5000 male live births) caused by gene variants affecting the synthesis or function of factor VIII (FVIII), an essential component of the intrinsic pathway of blood coagulation [1,2]. Different approaches based on alternative hemostatic agents to substitute for CFC have arisen in the last few years [20] These innovative products (i.e., non-factor replacement therapies) act by mimicking FVIII in tenase complex formation, or inhibiting naturally occurring anticoagulant proteins or inhibitors of activation of coagulation, enhancing thrombin generation and rendering fibrin clots more resistant; importantly, their prolonged half-life and subcutaneous (s.c.) administration route may play a crucial role for prophylaxis implementation and adherence, especially for patients with poor venous access [20,21]. Clinical experience is still limited in such a novel treatment, and data on its long-term safety and efficacy are lacking [8,28,29]
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