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Concizumab, a Non-Replacement Therapy for Persons with Hemophilia with Inhibitors

Giancarlo Castaman1, Victor Jimenez-Yuste2, Johnny Mahlangu3

  • 1Center for Bleeding Disorders and Coagulation, Department of Heart, Lungs, and Vessels, Careggi University Hospital, Largo Brambilla, 3, 50134 Florence, Italy.

Journal of Clinical Medicine
|May 14, 2025
PubMed

Despite enormous progress in the development of therapeutic agents for persons with hemophilia A and B (HA, HB), several unmet needs persist. These are disease- and treatment-related. Prophylaxis with clotting factor replacement is the gold standard, but not feasible in HA and HB with inhibitors. Whereas persons with HA with inhibitors can receive prophylaxis with a factor-mimicking agent, emicizumab, there is no recommendation for the agents to use as prophylaxis in persons with HB with inhibitors as there are no available molecules. Concizumab is a novel, subcutaneous prophylaxis option in persons with HA or HB with inhibitors that can potentially improve long-term outcomes. Here, we review the available data on concizumab and discuss its possible positioning in the armamentarium to treat hemophilia with inhibitors.

Keywords:
concizumabhemophilia Ahemophilia Binhibitorsnon-replacement therapyrebalancing agentstissue factor pathway inhibitorunmet needs

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