Kenet Gili, Martinowitz Uri
The Israeli National Hemophilia Center, Institute of Thrombosis and Hemostasis, Sheba Medical Center, Tel-Hashomer, Israel.
Semin Hematol. 2008 Apr;45(2 Suppl 1):S38-41. doi: 10.1053/j.seminhematol.2008.03.011.
Recombinant activated factor VII (rFVIIa; NovoSeven, Novo Nordisk, Bagsvaerd, Denmark) is well established as an effective hemostatic agent for the management of hemorrhage in hemophilia patients with inhibitors. Its use in prophylaxis is currently being investigated. On-demand treatment schedules usually involve multiple bolus doses of 90 to 120 microg/kg administered every 2 to 3 hours, but recent evidence from randomized controlled trials suggests that the use of higher single doses is equally safe and effective when used for the home treatment of hemarthroses. Consequently, the use of a single dose of 270 microg/kg rFVIIa for the treatment of bleeds in inhibitor patients has recently been approved by the European Medicines Agency (EMEA). Such high-dose regimens may overcome the rapid clearance rate observed in pediatric patients, and may be more convenient for patients with poor venous access. It also has been suggested that individually tailored single-dose therapies might be beneficial in selected groups of patients, although proper monitoring of such patients is advised. Further research should contribute towards more effective dose optimization of rFVIIa in hemophilic inhibitor patients.
重组活化凝血因子 VII（rFVIIa；诺其，丹麦诺和诺德公司， Bagsvaerd）作为治疗有凝血因子抑制物的血友病患者出血的有效止血剂已得到广泛认可。目前正在研究其在预防方面的应用。按需治疗方案通常包括每2至3小时多次推注90至120微克/千克，但最近随机对照试验的证据表明，用于关节积血家庭治疗时，使用更高的单剂量同样安全有效。因此，欧洲药品管理局（EMEA）最近已批准使用单剂量270微克/千克的rFVIIa治疗有凝血因子抑制物患者的出血。这种高剂量方案可能克服儿科患者中观察到的快速清除率，对于静脉通路不佳的患者可能更方便。也有人提出，尽管建议对这类患者进行适当监测，但个体化的单剂量疗法可能对特定患者群体有益。进一步的研究应有助于更有效地优化血友病有凝血因子抑制物患者的rFVIIa剂量。
