{"title":"Innovative Therapies for Acquired Hemophilia A.","authors":"Massimo Franchini, Daniele Focosi","doi":"10.1055/s-0044-1779737","DOIUrl":null,"url":null,"abstract":"<p><p>Acquired hemophilia A (AHA) is a rare autoimmune bleeding disorder which can be life-threatening. AHA is due to autoantibodies against coagulation factor VIII. Disease onset may be idiopathic (approximately half of the cases) or triggered by autoimmune disorders, cancers, drugs, infections, or pregnancy. Besides treating the underlying disorder, specific AHA treatments include management of bleeding and inhibitor eradication. Various first-line and second-line hemostatic and immunosuppressive agents are currently available for the management of AHA. Recently, the hemostatic drug emicizumab and the immunosuppressive drug rituximab have been the object of intense research from investigators as innovative promising therapies for AHA. This narrative review will be focused on the current status of the clinical use of these two off-label therapeutic agents in AHA.</p>","PeriodicalId":21673,"journal":{"name":"Seminars in thrombosis and hemostasis","volume":" ","pages":"68-72"},"PeriodicalIF":3.6000,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Seminars in thrombosis and hemostasis","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1055/s-0044-1779737","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/2/23 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Acquired hemophilia A (AHA) is a rare autoimmune bleeding disorder which can be life-threatening. AHA is due to autoantibodies against coagulation factor VIII. Disease onset may be idiopathic (approximately half of the cases) or triggered by autoimmune disorders, cancers, drugs, infections, or pregnancy. Besides treating the underlying disorder, specific AHA treatments include management of bleeding and inhibitor eradication. Various first-line and second-line hemostatic and immunosuppressive agents are currently available for the management of AHA. Recently, the hemostatic drug emicizumab and the immunosuppressive drug rituximab have been the object of intense research from investigators as innovative promising therapies for AHA. This narrative review will be focused on the current status of the clinical use of these two off-label therapeutic agents in AHA.
获得性血友病 A（AHA）是一种罕见的自身免疫性出血性疾病，可危及生命。AHA 是由针对凝血因子 VIII 的自身抗体引起的。发病原因可能是特发性的（约占半数），也可能由自身免疫性疾病、癌症、药物、感染或妊娠引发。除了治疗潜在的疾病外，AHA 的具体治疗方法还包括控制出血和根除抑制剂。目前有多种一线和二线止血药和免疫抑制剂可用于治疗 AHA。最近，止血药埃米珠单抗（emicizumab）和免疫抑制剂利妥昔单抗（rituximab）作为治疗 AHA 的有前途的创新疗法，成为研究人员热衷研究的对象。本综述将重点介绍这两种标签外治疗药物在 AHA 中的临床应用现状。
期刊介绍：
Seminars in Thrombosis and Hemostasis is a topic driven review journal that focuses on all issues relating to hemostatic and thrombotic disorders. As one of the premiere review journals in the field, Seminars in Thrombosis and Hemostasis serves as a comprehensive forum for important advances in clinical and laboratory diagnosis and therapeutic interventions. The journal also publishes peer reviewed original research papers.
Seminars offers an informed perspective on today''s pivotal issues, including hemophilia A & B, thrombophilia, gene therapy, venous and arterial thrombosis, von Willebrand disease, vascular disorders and thromboembolic diseases. Attention is also given to the latest developments in pharmaceutical drugs along with treatment and current management techniques. The journal also frequently publishes sponsored supplements to further highlight emerging trends in the field.
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