NBDF’s Bleeding Disorders Conference welcomed many people new to bleeding disorders, and offered preconference sessions to help these new attendees acquire some basic knowledge to assist them on their education journey throughout the conference. “Hemophilia Treatment Landscape,” on Thursday, August 21, 2025, gave attendees a comprehensive overview in the current ways of treating hemophilia.
Mark Reding, MD, Director of the Center for Bleeding and Clotting Disorders at the University of Minnesota traced the significant changes in hemophilia care over the past 30 years. The introduction of recombinant products in the 1990s was key milestone. “When we found recombinant products we thought we had solved it,” he said. “But then we realized that the burden on the patient to maintain prophylaxis was really just hard for a lot of people.”
Current Treatment Categories
Dr. Reding covered three main treatment approaches: factor replacement therapy, non-factor therapy, and gene therapy. He explained the differences between standard half-life, extended half-life, and newer extended half-life factor products, providing context for how each works and when they might be appropriate.
Non-factor therapies represent a different approach entirely. Factor mimetics, such as emecizimab, work by mimicking factor VIII to help activate the clotting process. They are also delivered subcutaneously, rather than intravenously, and require dosing less frequently. “it was the first nonfactor drug, the first subcutaneous drug, and the first highly effective prophylaxis option for hemophilia with inhibitors,” he said. “It changed the way you think about treating hemophilia.”
Rebalancing agents—including recently approved treatments like marstacimab, concizumab, and fitusiran—take yet another approach by adjusting the body's natural balance of clotting and bleeding factors.
Dr. Reding addressed gene therapy developments, including the use of adeno-associated viral vectors to potentially provide long-term treatment. He discussed both the potential benefits and current limitations, including variable patient responses, questions about durability, and the challenge that patients currently cannot receive repeat doses due to immune responses.
Dr. Reding emphasized that treatment selection should be individualized based on factors like bleeding risk, preferred method of administration, inhibitor status, and access to care. It is not a decision you need to make alone, he said. “I would just encourage you to make sure that you make the time and make your treatment team have those conversations with you so you can figure out the right therapy for you.”
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