Manuel Rodríguez-López, Servicio de Hematología y Hemoterapia, Hospital Universitario Álvaro Cunqueiro, EOXI Vigo, Vigo, Pontevedra, España
Ramiro J. Núñez-Vázquez, Sección de Trombosis y Hemostasia, Hospital Universitario Virgen del Rocío, Sevilla, España
María T. Álvarez-Román, Unidad de Trombosis y Hemostasia, Servicio de Hematología y Hemoterapia, Hospital Universitario La Paz, Madrid, España
The arrival of new subcutaneous products for prophylaxis in hemophilia has focused on preventing bleeding by normalizing thrombin generation. Emicizumab has been a paradigm shift, and while some experts prefer prophylaxis with coagulation factor VIII (CFVIII), others consider emicizumab as the reference treatment due to its efficacy and safety. CFVIII extended half-life concentrates have improved previous treatments, but their effectiveness has been limited by their half-life, restricted by von Willebrand factor. New products, such as efanesoctocog alfa, approved in 2023, offer an ultra-long half-life, reducing the treatment burden. This recombinant product presents modifications that extend its plasma half-life and improve thrombin generation. The clinical efficacy of weekly prophylaxis has been demonstrated in the XTEND-1 and XTEND-Kids studies, showing good results in efficacy and tolerance, with a low incidence of inhibitors. Indirect comparisons suggest that efanesoctocog alfa could be superior to emicizumab in bleeding prophylaxis in adults and adolescents without inhibitors. In conclusion, efanesoctocog alfa inaugurates a new class of ultra-long half-life CFVIII, offering a significant therapeutic opportunity for hemophilia by normalizing thrombin generation in a physiological manner. However, more studies are needed to evaluate its efficacy and safety in different clinical scenarios.
Keywords: Prophylaxis. Ultra-long half-life. Von Willebrand factor. XTEN. Efficacy. Innovation.