{"title":"[血友病:治疗方案正在发生革命?]","authors":"Michael Spannagl","doi":"10.1007/s00108-024-01712-4","DOIUrl":null,"url":null,"abstract":"<p><p>Prophylactic replacement therapy for hemophilia A (hereditary factor VIII deficiency) is a success story of the production of coagulation factor concentrates from donor plasma. Recombinant factor concentrates, which are also produced with modified gene constructs for coagulation factor VIII in order to improve pharmacological properties, have since proven their worth. This successful development over many years of factor concentrates for the successful treatment of hemophilia patients has now been followed by the innovation of a factor VIII mimetic in the form of a monoclonal antibody, which was developed in Japan already some years back. Emicizumab is a humanized, bispecific monoclonal antibody for therapeutic use in hemophilia A. With this therapeutic agent, the treatment of the hereditary coagulation defect is based, for the first time, on a completely new active principle. The specific antibody simulates the properties of coagulation factor VIII as a cofactor for the formation of the tenase complex with the coagulation factors IX and X. As a result under steady state conditions almost normal thrombin and thus fibrin formation can be achieved.</p>","PeriodicalId":73385,"journal":{"name":"Innere Medizin (Heidelberg, Germany)","volume":" ","pages":"1040-1043"},"PeriodicalIF":0.0000,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"[Hemophilia: is a revolution in treatment options taking place?]\",\"authors\":\"Michael Spannagl\",\"doi\":\"10.1007/s00108-024-01712-4\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Prophylactic replacement therapy for hemophilia A (hereditary factor VIII deficiency) is a success story of the production of coagulation factor concentrates from donor plasma. Recombinant factor concentrates, which are also produced with modified gene constructs for coagulation factor VIII in order to improve pharmacological properties, have since proven their worth. This successful development over many years of factor concentrates for the successful treatment of hemophilia patients has now been followed by the innovation of a factor VIII mimetic in the form of a monoclonal antibody, which was developed in Japan already some years back. Emicizumab is a humanized, bispecific monoclonal antibody for therapeutic use in hemophilia A. With this therapeutic agent, the treatment of the hereditary coagulation defect is based, for the first time, on a completely new active principle. The specific antibody simulates the properties of coagulation factor VIII as a cofactor for the formation of the tenase complex with the coagulation factors IX and X. As a result under steady state conditions almost normal thrombin and thus fibrin formation can be achieved.</p>\",\"PeriodicalId\":73385,\"journal\":{\"name\":\"Innere Medizin (Heidelberg, Germany)\",\"volume\":\" \",\"pages\":\"1040-1043\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2024-10-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Innere Medizin (Heidelberg, Germany)\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1007/s00108-024-01712-4\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/8/30 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Innere Medizin (Heidelberg, Germany)","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1007/s00108-024-01712-4","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/8/30 0:00:00","PubModel":"Epub","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
摘要
血友病 A(遗传性第八因子缺乏症)的预防性替代疗法是利用供体血浆生产凝血因子浓缩物的成功案例。重组因子浓缩物也是用改良的凝血因子 VIII 基因构建物生产的,目的是改善药理特性。在浓缩因子多年来成功治疗血友病患者的基础上,日本早在几年前就开发出了单克隆抗体形式的 VIII 因子模拟物。Emicizumab 是一种用于治疗 A 型血友病的人源化双特异性单克隆抗体。特异性抗体模拟凝血因子 VIII 的特性,作为辅助因子与凝血因子 IX 和 X 形成tenase 复合物。
[Hemophilia: is a revolution in treatment options taking place?]
Prophylactic replacement therapy for hemophilia A (hereditary factor VIII deficiency) is a success story of the production of coagulation factor concentrates from donor plasma. Recombinant factor concentrates, which are also produced with modified gene constructs for coagulation factor VIII in order to improve pharmacological properties, have since proven their worth. This successful development over many years of factor concentrates for the successful treatment of hemophilia patients has now been followed by the innovation of a factor VIII mimetic in the form of a monoclonal antibody, which was developed in Japan already some years back. Emicizumab is a humanized, bispecific monoclonal antibody for therapeutic use in hemophilia A. With this therapeutic agent, the treatment of the hereditary coagulation defect is based, for the first time, on a completely new active principle. The specific antibody simulates the properties of coagulation factor VIII as a cofactor for the formation of the tenase complex with the coagulation factors IX and X. As a result under steady state conditions almost normal thrombin and thus fibrin formation can be achieved.