Professor of Pediatrics Children's Hospital Los Angeles, University of Southern California Keck School of Medicine Los Angeles, United States
There are now 3 categories for treatment of hemophilia B--factor replacement therapy, gene therapy, and nonfactor therapy. Factor replacement therapy has been the mainstay of treatment for decades and currently there are both standard half-life and extended half-life version. These must be given intravenously typically at least once weekly and don't offer steady levels. Gene therapy is a newer treatment aimed at being a single dose therapy increasing FIX levels, however, eligibility criteria including age over 18 years limits this option to a relatively small group of patients. The newest option for treating patients with hemophilia B are nonfactor therapies with the recent approvals of marstacimab and concizumab and the pending approval of fitusiran. All 3 have been demonstrated to be effective in preventing bleeding and have largely been shown to be safe. Importantly, they are all administered subcutaneously either daily (concizumab), weekly (marstacimab) or monthly or every other month (fitusiran). In this debate, I will argue that nonfactor therapies are the ideal treatment for bleed prevention in hemophilia B given that they can be given to all ages, are delivered subcutaneously, provide steady levels of their effect, and are effective and safe.
Learning Objectives:
The newest option for treating patients with hemophilia B are nonfactor therapies with the recent approvals of marstacimab and concizumab and the pending approval of fitusiran. In this debate, I will argue that nonfactor therapies are the ideal treatment for bleed prevention in hemophilia B given that they can be given to all ages, are delivered subcutaneously, provide steady levels of their effect, and are effective and safe. All 3 have been demonstrated to be effective in preventing bleeding and have largely been shown to be safe. Importantly, they are all administered subcutaneously either daily (concizumab), weekly (marstacimab) or monthly or every other month (fitusiran).
