Croom Katherine F, McCormack Paul L
Wolters Kluwer Health | Adis, Auckland, New Zealand.
BioDrugs. 2008;22(2):121-36. doi: 10.2165/00063030-200822020-00005.
Recombinant factor VIIa (NovoSeven; also known as recombinant activated factor VII or eptacog alfa) is structurally similar to human plasma-derived coagulation factor VIIa, but is manufactured using DNA biotechnology. Recombinant factor VIIa interacts with thrombin-activated platelets to produce a thrombin burst leading to accelerated fibrin clot formation localized to the site of vascular injury. It is approved in many countries for use as an intravenous hemostatic agent in patients with congenital hemophilia with inhibitors, and also for acquired hemophilia, factor VII deficiency, and Glanzmann thrombasthenia in some countries. Studies have shown it to be effective and generally well tolerated when used intravenously to treat bleeding episodes or provide hemostatic cover during surgery in patients with congenital hemophilia with inhibitors, acquired hemophilia, factor VII deficiency or Glanzmann thrombasthenia. Based on available data, its efficacy in terms of patient-assessed response may be similar to that of activated prothrombin complex concentrate (aPCC), but treatment with a single 270 microg/kg dose of recombinant factor VIIa might reduce the need for rescue therapy compared with aPCC. Recombinant factor VIIa is not immunogenic in patients with hemophilia, does not produce an anamnestic response in hemophilia patients with inhibitors, and has very low thrombogenicity. It is recommended in guidelines as the treatment of choice for bleeds in patients with hemophilia B with high-responding inhibitors and for patients with factor VII deficiency, and is also a first-line therapeutic option for high-responder hemophilia A patients with inhibitors and those with acquired hemophilia. Cost data from pharmacoeconomic analyses support its use in hemophilia patients with inhibitors. Thus, recombinant factor VIIa is a valuable treatment option for patients with these rare, but potentially serious, bleeding disorders.
重组凝血因子VIIa（诺其；也称为重组活化凝血因子VII或eptacog alfa）在结构上与人血浆来源的凝血因子VIIa相似，但采用DNA生物技术制造。重组凝血因子VIIa与凝血酶激活的血小板相互作用，产生凝血酶爆发，导致纤维蛋白凝块加速形成，定位于血管损伤部位。在许多国家，它被批准用作先天性血友病伴抑制剂患者的静脉内止血剂，在一些国家也用于获得性血友病、凝血因子VII缺乏症和血小板无力症。研究表明，对于先天性血友病伴抑制剂、获得性血友病、凝血因子VII缺乏症或血小板无力症患者，静脉内使用它治疗出血发作或在手术期间提供止血覆盖时，是有效且一般耐受性良好的。根据现有数据，其在患者评估反应方面的疗效可能与活化凝血酶原复合物浓缩物（aPCC）相似，但与aPCC相比，单次270μg/kg剂量的重组凝血因子VIIa治疗可能减少救援治疗的需求。重组凝血因子VIIa在血友病患者中无免疫原性，在有抑制剂的血友病患者中不会产生回忆反应，且血栓形成性非常低。指南推荐其作为B型血友病伴高反应性抑制剂患者和凝血因子VII缺乏症患者出血的首选治疗方法，也是A型血友病伴抑制剂的高反应者和获得性血友病患者的一线治疗选择。药物经济学分析的成本数据支持其在有抑制剂的血友病患者中的使用。因此，重组凝血因子VIIa对于这些罕见但可能严重的出血性疾病患者是一种有价值的治疗选择。
