{"title":"埃米珠单抗治疗获得性血友病 A:两个病例的报告和用药策略","authors":"Faiza Ahmed, Mariia Kasianchyk, Alejandro Moreno, Simone Chang, Satish Maharaj","doi":"10.1002/jha2.878","DOIUrl":null,"url":null,"abstract":"<p>Acquired hemophilia A (AHA) is a rare autoimmune bleeding disorder caused by autoantibodies against FVIII. Severe AHA is life-threatening. Currently, licensed hemostatic agents for the treatment of severe AHA have short half-lives and require intravenous administration, leading to a need for hospitalization, higher costs, and negative effects on quality of life. We present two cases of severe AHA with high inhibitor titers where emicizumab was safely and effectively used with intensive immunosuppression. These reports suggest in vivo efficacy even in high inhibitor environments. The optimal dosing regimen (accelerated vs. standard loading, maintenance frequency) is unknown and we discuss the current approaches.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2024-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.878","citationCount":"0","resultStr":"{\"title\":\"Emicizumab for acquired hemophilia A: Report of two cases and dosing strategies\",\"authors\":\"Faiza Ahmed, Mariia Kasianchyk, Alejandro Moreno, Simone Chang, Satish Maharaj\",\"doi\":\"10.1002/jha2.878\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>Acquired hemophilia A (AHA) is a rare autoimmune bleeding disorder caused by autoantibodies against FVIII. Severe AHA is life-threatening. Currently, licensed hemostatic agents for the treatment of severe AHA have short half-lives and require intravenous administration, leading to a need for hospitalization, higher costs, and negative effects on quality of life. We present two cases of severe AHA with high inhibitor titers where emicizumab was safely and effectively used with intensive immunosuppression. These reports suggest in vivo efficacy even in high inhibitor environments. The optimal dosing regimen (accelerated vs. standard loading, maintenance frequency) is unknown and we discuss the current approaches.</p>\",\"PeriodicalId\":72883,\"journal\":{\"name\":\"EJHaem\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2024-03-15\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.878\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"EJHaem\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1002/jha2.878\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"EJHaem","FirstCategoryId":"1085","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1002/jha2.878","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
摘要
获得性血友病 A(AHA)是一种罕见的自身免疫性出血性疾病,由抗 FVIII 的自身抗体引起。严重的 AHA 会危及生命。目前,用于治疗严重 AHA 的止血药半衰期较短,需要静脉给药,因此需要住院治疗,费用较高,对生活质量也有负面影响。我们介绍了两例抑制剂滴度较高的严重 AHA 病例,在强化免疫抑制的情况下,埃米珠单抗得到了安全有效的应用。这些报告表明,即使在高抑制剂环境中,埃米珠单抗也能发挥体内疗效。最佳给药方案(加速给药与标准给药、维持频率)尚不清楚,我们将讨论目前的方法。
Emicizumab for acquired hemophilia A: Report of two cases and dosing strategies
Acquired hemophilia A (AHA) is a rare autoimmune bleeding disorder caused by autoantibodies against FVIII. Severe AHA is life-threatening. Currently, licensed hemostatic agents for the treatment of severe AHA have short half-lives and require intravenous administration, leading to a need for hospitalization, higher costs, and negative effects on quality of life. We present two cases of severe AHA with high inhibitor titers where emicizumab was safely and effectively used with intensive immunosuppression. These reports suggest in vivo efficacy even in high inhibitor environments. The optimal dosing regimen (accelerated vs. standard loading, maintenance frequency) is unknown and we discuss the current approaches.