Pediatric Hematologist Aflac Cancer and Blood Disorders Atlanta, United States
Haemophilia B is a relatively rate X-linked disorder leading to the deficiency or defective production of coagulation factor IX (FIX) that can be mild, moderate or severe in clinical severity. Without multidisciplinary care, persons affected can develop recurrent and bleeding that can lead to crippling severe arthropathy and/or life-threatening bleeding. Over the last four decades, advancements in haemophilia B management, include establishing the long term benefit of prophylaxis with clotting factor concentrates first with plasma derived concentrates, the advent of recombinant FIX and the more recently implemented extended half-life FIX products. The therapeutic landscape continues to evolve with the recent development and either approval or soon to be approved non-factor replacement therapies, including but not limited to anti- TFPI therapies, small interfering RNA technology targeting antithrombin and liver directed gene therapies.
In this debate, I will make the case for the concept of replacement clotting factor concentrates in the management and prevention of bleeding in persons with congenital FIX deficiency over non-factor therapies and the so-called rebalancing agents. We will highlight the track record of the hemostatic efficacy of primary and secondary factor prophylaxis, importance of extravascular distribution of FIX and the extrahemostatic benefits of FIX in humans.
Learning Objectives:
The focus of this lecture is to highlight why factor replacement is optimal for the management of hemophilia B patients as part of a 3 way debate on "What is the optimal therapy for hemophilia B?"
