[NEW INNOVATIONS IN THE TREATMENT OF HEMOPHILIA].

Abstract

Introduction: Hemophilia is a congenital bleeding disorder with a deficiency of coagulation factor 8 or 9 (hemophilia A or B, respectively) and a tendency for recurrent bleeding, especially into muscles and joints, which may cause orthopedic damage and necessitate joint replacement surgeries at a young age. In recent years, there has been a huge breakthrough in the treatment of hemophilia. Until recently, the only available therapy was based on repeated intravenous injection of factor concentrates (replacement therapy). Nowadays, new therapeutic options are being developed, some already registered and approved and others are still in clinical studies. The new molecules either enhance the coagulation system or inhibit coagulation inhibitors, promoting faster and improved clot formation, and are administered subcutaneously. These developments have had an enormous impact on the patients' quality of life. In the last decade, the option of complete (genetic) cure of the disease has been explored for both hemophilia A and hemophilia B, and multiple clinical gene therapy trials are currently being conducted. In this review, we discuss the novel therapies currently available for hemophilia. We will elaborate on extended half-life long acting factor concentrates, subcutaneous non-replacement therapies and gene therapy.