(MED-TH-07.1) Factor replacement

FACTOR REPLACEMENT
Bulent ZULFİKAR, MD
Hemophilia A, a hereditary bleeding disorder resulting from a deficiency in coagulation Factor VIII, necessitates vigilant management to prevent both acute bleeding episodes and long-term musculoskeletal complications. Factor replacement therapy has been the cornerstone of Hemophilia A treatment for decades, providing an established and efficacious strategy to mitigate bleeding tendencies, enhance quality of life, and prevent irreversible joint damage. Regular prophylactic infusions of Factor VIII have proven to significantly diminish bleeding events, thereby preventing chronic joint damage and other sequelae, ultimately improving long-term patient outcomes. Furthermore, routine factor replacement has been shown to mitigate the development of osteoporosis and prevent joint destruction, both of which are prevalent concerns in hemophilia patients. These benefits contribute substantially to long-term improvements in patient quality of life, decreasing both the physical burden of joint disease and the risk of fractures.
Factor replacement therapy was introduced in Turkiye in 1993 and reimbursement for prophylaxis of all hemophiliacs began in 2007. Over the past 10 years, in Istanbul University Inherited Bleeding Disorders Center, annual factor consumption has gradually reached to 50.000.000 IU, treatment strategies have become more effective, and the efficacy of prophylaxis therapy has been enhanced. In recent years, 65% of prescriptions written for hemophilia A cases in our center have been EHL, 30% PD, and 5% standard recombinant CFC.
Recently added to hemophilia treatment, efanesoctocog alfa (EFA) has shown remarkable success in clinical experience. EFA has demonstrated high efficacy in bleeding control and prophylaxis with the extended half-life.
One of the main challenges in hemophilia treatment is the difficulty of administering the therapy. Specifically, adherence to treatment can negatively affect patients' lifestyles. The risk of infection transmission remains a significant concern, especially when PD CFC are used.
In conclusion, despite the ongoing development of alternative therapies, factor replacement remains the gold standard for managing Hemophilia A. Given the current limitations of non-factor and gene therapies, factor replacement continues to be the cornerstone of effective Hemophilia A management.