Journal of Blood Medicine (Dec 2021)
Clinical Utility of Subcutaneous Factor VIII Replacement Therapies in Hemophilia A: A Review of the Evidence
Abstract
Yesim Dargaud,1,2 Maissa Janbain3 1UR4609 Hemostase et Thrombose, Université Claude Bernard Lyon 1, Faculté de Médecine Lyon Est, Lyon, France; 2Unité d’Hémostase Clinique, Hôpital Cardiologique Louis Pradel, Hospices Civils de Lyon, Lyon, France; 3Hematology Department, Tulane School of Medicine, New Orleans, LA, USACorrespondence: Yesim DargaudUR4609 Hemostase&Thrombose, Université Claude Bernard Lyon 1, Faculte de Medecine Lyon Est, 7, rue Guillaume Paradin, Lyon, 69008, FranceTel +3378778797Email [email protected]: Hemophilia therapies have tremendously improved over the last decades with the development of prolonged half-life factor VIII (FVIII) and FIX concentrates, non-factor therapies, such as emicizumab, anti-TFPI antibodies or siRNA antithrombin and gene therapy. All of these new molecules significantly reduced the burden of the disease and improved the quality of life of patients with severe hemophilia. Emicizumab, a non-factor therapy, is currently the only subcutaneous molecule available for prophylactic treatment of severe hemophilia A. Because of the subcutaneous route of delivery and similar efficacy to FVIII replacement therapy, emicizumab has been rapidly adopted by patients and their families. This clinical observation emphasizes the relevance and need for the development of subcutaneous FVIII concentrates. Here, we report evidence-based advantages and interest in the subcutaneous route of administration for the treatment of hemophilia A and review the stages of development of the different subcutaneous FVIII molecules.Keywords: hemophilia A, factor VIII, recombinant von Willebrand factor fragment, prophylaxis, subcutaneous injection
