ASH 2025 | Real‑world experience with marstacimab in severe and non‑severe hemophilia

Another hat that I don as a clinician is the center director for the Hemophilia Treatment Center, a bleeding disorder that affects boys and girls, predominantly males, with severe hemophilia A and B. For the longest time, the way we treated hemophilia was with an intravenous infusion of artificial clotting factor. Over the last three, two and a half, three years, we’ve had an advent of novel therapies that can be done in the subcutaneous route, not into the vein, but something that can be injected under the skin that makes lives for the patients and the caregivers for hemophilia very easy. The subcutaneous therapies have now evolved into self-administered fixed-dose pen devices that patients can actually carry in their pockets, store it at room temperature, allowing them to have a lot of mobility, flexibility and ease of administration. What we are researching and the data that we have shared about marstacimab which is a novel TFPI inhibitory agent which rebalances the coagulation cascade for people with hemophilia A and hemophilia B is a revolutionary new therapy that is available for people with hemophilia A and hemophilia B without inhibitors in the United States right now as a fixed dose pen. I’ve had the good fortune of having good success with that medicine with one of the largest number of patients in the world on commercially used marstacimab. But we are now through my research at ASH 2025 we are trying to push the needle forward to try to help think about giving such treatment options to people who are otherwise marginalized in the hemophilia community. These are women with hemophilia who classically are considered only as carriers but still bleed significantly and people with moderate hemophilia who continue to have a lot of bleeding throughout their life but per national and international guidelines don’t get offered prophylaxis therapy so how can we use these amazing new therapies that are easy to use and have changed the life of severe hemophilia boys how do we bring it to people who also suffer from severe bleeding disorder with a hemophilia diagnosis but are otherwise always neglected so my research has shown real-world utilization of these novel monoclonal antibodies and therapies in female patients and non-severe hemophilia patients with an amazing success rate that has changed their lives. So really happy that we were able to share the real world data to inspire other clinicians and help insurance companies look at authorization for such treatments more favorably.

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