Immunohematology and Transfusion Center, University Hospital of Parma, Parma, Italy.
Semin Thromb Hemost. 2009 Nov;35(8):806-13. doi: 10.1055/s-0029-1245113. Epub 2010 Feb 18.
The mainstay of therapy in patients with congenital hemophilia is factor replacement. However, the development of inhibitors in these patients is a major complication that represents an important challenge in hemophilia care. Development of inhibitors complicates the clinical course of severe hemophilia in up to 30% of patients with hemophilia A and up to 5% of patients with hemophilia B. Although the main short-term objective of the treatment of alloantibodies against factors VIIII and IX is to control the bleeding diathesis, the eradication of the inhibitor is the leading long-term goal. The management of severe bleeding episodes and the definitive eradication of the autoantibody are also the two main options of the clinical management of patients with acquired hemophilia, a rare but life-threatening hemorrhagic condition. The most recent options available for treating patients with acquired hemophilia and congenital hemophilia with inhibitors are addressed in this review.
在患有先天性血友病的患者中,治疗的主要方法是因子替代。然而,这些患者产生抑制剂是一个主要的并发症,这是血友病治疗中的一个重要挑战。在多达 30%的血友病 A 患者和多达 5%的血友病 B 患者中,抑制剂的产生使严重血友病的临床病程复杂化。尽管针对因子 VIII 和 IX 的同种抗体的治疗的主要短期目标是控制出血倾向,但抑制剂的消除是主要的长期目标。严重出血发作的管理和自身抗体的最终消除也是获得性血友病患者的临床管理的两个主要选择,获得性血友病是一种罕见但危及生命的出血性疾病。本综述介绍了治疗患有获得性血友病和伴有抑制剂的先天性血友病患者的最新选择。