Department of Functional Genomics, Medical University of Lodz, Żeligowskiego 7/9, 90-752, Lodz, Poland.
Department of General Surgery & Laboratory of Gastric Cancer, State Key Laboratory of Biotherapy/Collaborative Innovation Center of Biotherapy and Cancer Center, West China Hospital, Sichuan University, Chengdu, China.
Curr Protein Pept Sci. 2024;25(9):719-737. doi: 10.2174/0113892037294674240509094418.
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 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.
血友病是一种血浆出血性疾病，其特征是某些凝血因子缺乏。这种疾病最常见的形式，即 A 型和 B 型，影响着全球约 40 万人。如果没有适当的治疗来确保适当的凝血级联反应，这种疾病可能会导致严重的残疾。通过替代疗法，即静脉内给予缺失的凝血因子，可以最大限度地减少患者的不适。频繁的药物治疗和与因子抑制剂相关的风险是显著的缺点，这需要改进现有的治疗方法或开发新的治疗方法。本综述考察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，而因子 VIII 的缺乏会导致 A 型血友病。本文首先概述了这一主题，然后总结了 Emicizumab 在 A 型血友病中的可用临床试验。几项干预性临床试验发现，Emicizumab能有效减少出血发作，并提高各种 A 型血友病患者的满意度。目前的 Emicizumab 相关试验预计将于 2024 年至 2030 年完成，除了先天性 A 型血友病外，这些试验还涵盖了获得性 A 型血友病和运动型患者。更全面地了解 Emicizumab 可能会彻底改变 A 型血友病的管理方式，并提高生活质量。总之，由于皮下给药和注射次数减少，Emicizumab 是一种更温和的治疗方法，这减少了注射部位反应，使治疗负担减轻，最终减少了医院就诊次数和间接成本。
