The availability of recombinant activated factor VII (rFVIIa, eptacog alfa activated) has greatly advanced the care of patients with haemophilia A or B who have developed inhibitors against the infused replacement factor. Recombinant FVIIa is licensed for the on-demand treatment of bleeding episodes and the prevention of bleeding in surgery or invasive procedures in patients with congenital haemophilia with inhibitors. This article attempts to review in detail the extensive evidence of rFVIIa in congenital haemophilia patients with inhibitors. Patients with acute bleeding episodes are best treated on demand at home, to achieve the short- and long-term benefits of rapid bleed control. Key prospective studies have shown that rFVIIa achieves consistently high efficacy rates in the management of acute (including joint) bleeds in inhibitor patients in the home treatment setting. Substantial post-approval data from key registries also support the on-demand efficacy profile of rFVIIa established by the prospective clinical trials. The availability of rFVIIa has allowed major surgery to become a reality for inhibitor patients. Studies in key surgery, including orthopaedic procedures, have found that rFVIIa provides consistently high efficacy rates. Importantly, the wealth of data does not raise any unexpected safety concerns surrounding rFVIIa use; this is likely because rFVIIa is a recombinant product with a localised mechanism of action at the site of vascular injury. In summary, rFVIIa is established as an effective and well-tolerated first-line treatment for on-demand bleeding control and bleed prevention during minor and major (including elective orthopaedic) surgery in inhibitor patients. Use of rFVIIa has been a major step towards narrowing the gap in outcomes between inhibitor patients and non-inhibitor patients.

中文翻译：

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重组活化因子VII用于治疗出血和使用抑制剂预防先天性血友病中与手术有关的出血。

重组激活因子VII（rFVIIa，阿帕金酸α激活）的可用性大大提高了对A或B型血友病患者的治疗，这些患者已开发出针对输注替代因子的抑制剂。重组FVIIa被许可用于先天性血友病患者使用抑制剂按需治疗出血发作并在外科手术或侵入性手术中预防出血。本文试图详细回顾rFVIIa在先天性血友病患者中使用抑制剂的广泛证据。急性出血发作的患者最好在家中按需治疗，以达到快速控制出血的短期和长期利益。关键的前瞻性研究表明，rFVIIa在家庭治疗环境中抑制抑制剂患者的急性（包括关节）出血的治疗中一直获得很高的疗效。来自主要注册机构的大量批准后数据也支持前瞻性临床试验建立的rFVIIa的按需疗效概况。rFVIIa的可用性已使大手术成为抑制剂患者的现实。对关键外科手术（包括整形外科手术）的研究发现，rFVIIa始终如一地提供很高的疗效。重要的是，丰富的数据不会引起使用rFVIIa的任何意外的安全隐患；这可能是因为rFVIIa是在血管损伤部位具有局部作用机制的重组产物。总之，rFVIIa被确立为一种有效且耐受性良好的一线治疗药物，用于抑制患者的轻度和重度（包括选择性骨科手术）手术中的按需出血控制和出血预防。使用rFVIIa是缩小抑制剂患者与非抑制剂患者之间结局差距的重要一步。