Castaman Giancarlo, Jimenez-Yuste Victor, Mahlangu Johnny
Center for Bleeding Disorders and Coagulation, Department of Heart, Lungs, and Vessels, Careggi University Hospital, Largo Brambilla, 3, 50134 Florence, Italy.
Haematology Department, Hospital Universitario La Paz-IdiPaz, Autónoma University, Paseo de la Castellana, 261, 28046 Madrid, Spain.
J Clin Med. 2025 Apr 25;14(9):2961. doi: 10.3390/jcm14092961.
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.
尽管在开发用于甲型和乙型血友病（HA、HB）患者的治疗药物方面取得了巨大进展，但仍存在一些未满足的需求。这些需求与疾病和治疗相关。凝血因子替代预防是金标准，但在有抑制剂的HA和HB患者中不可行。虽然有抑制剂的HA患者可以接受模拟因子药物艾美赛珠单抗的预防，但由于没有可用的分子，尚无推荐用于有抑制剂的HB患者进行预防的药物。康西珠单抗是一种新型的皮下预防药物，适用于有抑制剂的HA或HB患者，可能会改善长期预后。在此，我们综述了关于康西珠单抗的现有数据，并讨论其在治疗有抑制剂的血友病药物库中的可能定位。
