Hemophilia A is a rare bleeding disorder caused by deficiency of clotting factor VIII (FVIII). The current standard of care for patients with severe hemophilia A centers on replacement therapy with FVIII concentrate, either on demand when bleeding occurs or prophylactically (replacement therapy). Potential complications from replacement therapy include the development of inhibitory antibodies that attack the clotting factor in approximately one-fourth of patients and viral infections from human clotting factors. Moreover, traditional clotting factor replacement formulations require frequent and time-consuming administration, which in turn has limited use of prophylactic regimens to maintain adequate levels of clotting factor activity.
In the past several years, new therapies have become available that have the potential to dramatically alter the approach to chronic disease management for the first time in three decades. These new agents offer the opportunity to improve the frequency and convenience of replacement therapy, while achieving an unprecedented degree of protection over time. Furthermore, agents that eliminate use of clotting factor entirely, including factor VIII (FVIII) mimetics and drugs aimed at rebalancing the coagulation cascade via antithrombin, may be useful not only in overcoming inhibitors, but also in preventing their formation.
This activity features a patient with hemophilia A expressing his real-world experiences throughout his treatment journey. The patient shares his story with regards to diagnosis, treatment challenges, impact on quality of life and how they have overcome obstacles living with hemophilia A.