Abstract：
Hemophilia is a plasma bleeding disorder characterized by a deficiency of certain blood clotting factors. The most common forms of this disease, i.e., type A and type B, affect approximately 400,000 people worldwide. Without appropriate treatment ensuring the proper coagulation cascade, this disease may lead to serious disability. Minimizing patient discomfort is possible via replacement therapy, consisting of the substitution of a missing coagulation factor via intravenous administration. Frequent medication and the risk related to factor inhibitors are significant disadvantages, necessitating the improvement of current therapies or the development of novel ones. This review examines the humanized bispecific antibody Emicizumab which ensures hemostasis by mimicking the action of the coagulation factor VIII, a deficiency of which causes type A hemophilia. The paper outlines the topic and then summarizes available clinical trials on Emicizumab in type A hemophilia. Several interventional clinical trials have found Emicizumab to be effective in decreasing bleeding episodes and raising patient satisfaction among various hemophilia A populations. Current Emicizumab-related trials are forecast to be completed between 2024 and 2030, and in addition to congenital hemophilia A, the trials cover acquired hemophilia A and patients playing sports. Providing a more comprehensive understanding of Emicizumab may revolutionize the management of hemophilia type A and improve quality of life. Conclusively, Emicizumab is a gentler therapy owing to subcutaneous delivery and fewer injections, which reduces injection-site reactions and makes therapy less burdensome, ultimately decreasing hospital visits and indirect costs.
摘要：
血友病是一种血浆出血性疾病，其特征是缺乏某些凝血因子。这种疾病最常见的形式，即，A型和B型，影响全球约40万人。如果没有适当的治疗，确保适当的凝血级联，这种疾病可能导致严重的残疾。通过替代疗法可以最大限度地减少患者的不适，包括通过静脉给药替代缺失的凝血因子。频繁的用药和与因子抑制剂相关的风险是显著的缺点，需要改进当前的疗法或开发新的疗法。这篇综述研究了人源化双特异性抗体Emicizumab，它通过模拟凝血因子VIII的作用来确保止血。导致A型血友病的缺乏。本文概述了该主题，然后总结了Emicizumab在A型血友病中的可用临床试验。一些介入临床试验已经发现Emicizumab在各种血友病A人群中可有效减少出血事件并提高患者满意度。目前艾美珠单抗相关试验预计将在2024年至2030年之间完成，除了先天性血友病A，试验涵盖获得性血友病A和参加体育运动的患者.提供对Emicizumab的更全面了解可能会彻底改变A型血友病的管理并改善生活质量。最后，Emicizumab是一种温和的治疗，由于皮下给药和较少的注射，这减少了注射部位的反应，减少了治疗的负担，最终减少医院就诊次数和间接成本。
