Poon M-C, D'Oiron R, Von Depka M, Khair K, Négrier C, Karafoulidou A, Huth-Kuehne A, Morfini M
Centre d'Hémophiles, AP-HP Hôpital Bicêtre, Le Kremlin Bicêtre, France.
J Thromb Haemost. 2004 Jul;2(7):1096-103. doi: 10.1111/j.1538-7836.2004.00767.x.
Antibodies to glycoprotein (GP) IIb-IIIa and/or HLA may render platelet transfusions ineffective to stop bleeding or to cover surgery in patients with Glanzmann's thrombasthenia (GT). Anecdotal reports suggest recombinant factor (rF)VIIa might be a therapeutic alternative in these situations.
An international survey was conducted to evaluate further the efficacy and safety of rFVIIa in GT patients.
We analyzed the use of rFVIIa during 34 surgical/invasive procedures and 108 bleeding episodes in 59 GT patients including 29 with current or previous antiplatelet antibodies, and 23 with a history of refractoriness to platelet transfusion.
rFVIIa was effective in 29 of the 31 evaluable procedures, and in 77 of the 103 evaluable bleeding episodes of which eight had a recurrence. A significantly higher success rate was observed in severe bleeding episodes when an arbitrarily defined 'optimal regimen' derived from the Canadian pilot study results (> or = 80 micro g kg(-1) rFVIIa/injection, dosing interval < or = 2.5 h, three or more doses before failure declaration) was used compared with other regimens (77%; 24/31 vs. 48%, 19/40; chi(2), P = 0.010). Patients given maintenance doses had significantly fewer recurrences within 48 h of bleed cessation compared with those not given any (Fisher's exact test, P = 0.022). One thromboembolic event and one blood clot in the ureter occurring in surgical patients following prolonged continuous infusion of high-dose rFVIIa and antifibrinolytic drug use have been previously reported.
rFVIIa seems a potential alternative to platelet transfusion in GT patients, particularly in those with antiplatelet antibodies and/or platelet refractoriness.
糖蛋白（GP）IIb-IIIa抗体和/或人类白细胞抗原（HLA）抗体可能会使血小板输注对于患有Glanzmann血小板无力症（GT）的患者止血或覆盖手术无效。轶事报道表明重组因子（rF）VIIa在这些情况下可能是一种治疗选择。
进行一项国际调查以进一步评估rFVIIa在GT患者中的疗效和安全性。
我们分析了59例GT患者在34例外科手术/侵入性操作和108次出血事件中rFVIIa的使用情况，其中包括29例有当前或既往抗血小板抗体的患者，以及23例有血小板输注难治史的患者。
rFVIIa在31例可评估的操作中有29例有效，在103例可评估的出血事件中有77例有效，其中8例复发。当使用源自加拿大初步研究结果（≥80μg·kg⁻¹ rFVIIa/注射，给药间隔≤2.5小时，在宣布失败前给予三剂或更多剂）的任意定义的“最佳方案”时，与其他方案相比，严重出血事件的成功率显著更高（77%；24/31对48%，19/40；χ²检验，P = 0.010）。与未给予维持剂量的患者相比，给予维持剂量的患者在出血停止后48小时内复发明显更少（Fisher精确检验，P = 0.022）。先前曾报道过在高剂量rFVIIa持续输注和使用抗纤溶药物后，外科手术患者发生1例血栓栓塞事件和1例输尿管血凝块。
rFVIIa似乎是GT患者血小板输注的一种潜在替代方法，特别是对于那些有抗血小板抗体和/或血小板难治性的患者。
