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首页> 外文期刊>Journal of Blood Medicine >Critical appraisal of the role of recombinant activated factor VII in the treatment of hemophilia patients with inhibitors
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Critical appraisal of the role of recombinant activated factor VII in the treatment of hemophilia patients with inhibitors

机译:关键评估重组活化因子VII在血友病患者中使用抑制剂的作用

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Abstract: Hemophilia patients with inhibitors faced the constraint of inadequate treatment for several years before the era of recombinant factor VIIa (rFVII). Initially, rFVIIa was used in the compassionate-use programs. After a worldwide license was issued, more than 1.5 million doses were administered. Bleeding of joints and muscles was controlled effectively by means of an early home treatment program, with either a standard dose of 90 μg/kg every 2 to 3 hours for a few doses or a single dose of 270 μg/kg. For more serious bleeding episodes or minor surgery, an initial dose of 90 μg/kg was given every 2 hours for 24 to 48 hours followed by increased intervals of 3 to 6 hours according to the severity of bleeding and efficacy of bleeding control. In cases of major surgery such as orthopedic procedures, the same regimen can be applied except for a higher initial dose of 120 to 180 μg/kg. However, increasing the dose should be considered if there are unexpected bleeding complications since the half-life and clearance of rFVIIa differ between individuals. In addition, prophylaxis is administered to a small number of patients. Finally, the reported thromboembolic events found in hemophilia patients with inhibitors receiving rFVIIa are extremely low, much less than 1%.
机译:摘要:带有抑制剂的血友病患者在重组因子VIIa(rFVII)时代来临之前面临着治疗不足的局限。最初,rFVIIa用于同情使用程序。在全球范围内颁发许可证后,已施用了超过150万剂。通过早期的家庭治疗程序,可以有效地控制关节和肌肉的出血,标准剂量为每2至3小时90μg/ kg,持续数次,或单剂量270μg/ kg。对于更严重的出血事件或较小的手术,根据出血的严重程度和出血控制的有效性,每2小时给予一次90μg/ kg的初始剂量,持续24至48小时,然后增加3至6小时的间隔。对于大手术(例如整形外科手术),可以采用相同的治疗方案,但初始剂量较高,为120至180μg/ kg。但是,如果出现意外的出血并发症,则应考虑增加剂量,因为rFVIIa的半衰期和清除率因人而异。另外,对少数患者进行预防。最后,在接受rFVIIa抑制剂治疗的血友病患者中发现的血栓栓塞事件极低,远远少于1%。

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