Tokugawa Tazuko
Department of Hematology, Hyogo Medical University Hospital.
Rinsho Ketsueki. 2022;63(9):1212-1222. doi: 10.11406/rinketsu.63.1212.
Replacement therapy is the basic treatment for hemophilia by infusing deficient clotting factors, including replacement therapy for prophylaxis (i.e., prevention of breakthrough bleeding for physical activity), episodic replacement therapy, replacement therapy during and after procedures and surgery, and replacement therapy for physical activity, each administered at doses and intervals appropriate for the purpose and the product used. Although emicizumab is increasingly used for prophylaxis in severe hemophilia A, the combination of replacement therapy is necessary, especially during severe bleeding, highly invasive procedures, and major surgery. Furthermore, the usual APTT measurements cannot be used for monitoring the replacement therapy and detecting the presence of inhibitors while patients are receiving emicizumab. Hemostatic management of patients with inhibitors should be implemented based on the purpose of the therapy, the latest inhibitor titer, and the clinical response to the product, with the choice of inhibitor neutralization or bypass agent therapy. When bypassing agents are used in patients with hemophilia A with emicizumab inhibitor during and after bleeding, procedures, and surgery, the choice of agent and dose adjustment should be made with attention to thrombotic complications.
替代疗法是血友病的基本治疗方法，通过输注缺乏的凝血因子进行，包括预防性替代疗法（即预防因体力活动导致的突破性出血）、按需替代疗法、手术及操作期间和之后的替代疗法以及体力活动时的替代疗法，每种疗法的给药剂量和间隔均根据治疗目的和所用产品进行调整。尽管艾美赛珠单抗越来越多地用于重度甲型血友病的预防，但替代疗法的联合使用仍是必要的，尤其是在严重出血、高侵入性操作和大手术期间。此外，在患者接受艾美赛珠单抗治疗时，通常的活化部分凝血活酶时间（APTT）测量不能用于监测替代疗法和检测抑制剂的存在。对于有抑制剂的患者，应根据治疗目的、最新的抑制剂滴度以及对产品的临床反应进行止血管理，选择抑制剂中和或旁路制剂疗法。当在出血、手术及操作期间和之后，对接受艾美赛珠单抗治疗且有抑制剂的甲型血友病患者使用旁路制剂时，应注意血栓形成并发症，选择合适的制剂并调整剂量。
