Atkison P R, Jardine L, Williams S, Barr R M, Quan D, Wall W
Children's Hospital of Western Ontario, London Health Sciences Centre, University of Western Ontario, London, Ontario, Canada.
Transplant Proc. 2005 Mar;37(2):1091-3. doi: 10.1016/j.transproceed.2004.11.067.
Several reports have suggested a benefit for recombinant Factor VIIa (rFVIIa) in nonhematological conditions, including liver disease and transplantation. However, there are few reports of its use in children with liver failure. Recently, we used rFVIIa in four patients with liver failure and severe coagulopathy with bleeding who demonstrated significant laboratory and clinical improvement following its use with no side effects.
All four patients were hospitalized with liver failure, coagulopathy, and bleeding that was controlled with fresh frozen plasma, platelets, and other therapies, as indicated. Their international normalization ratios (INR) ranged from 1.7 to 5.8 (normal 0.9-1.1). All four patients received rFVIIa for bleeding episodes that were not responding to their usual therapy, for procedures with a high risk of bleeding, or both. The dose of rFVIIa ranged from 0.067 to 0.3 mg/kg. The INR improved to normal or near normal in all four patients. In all cases, bleeding stopped within 10 minutes of receiving the rFVIIa, and there were no complications observed.
rFVIIa provided significant benefit in these children with liver failure and severe coagulopathy, in terms of clinical and laboratory improvement in their bleeding and coagulation profiles. There were no obvious side effects from the rFVIIa. This drug may be an important tool in the treatment of children with liver failure and more study is needed to define the optimal dosing for children.
多项报告表明重组凝血因子VIIa（rFVIIa）在非血液系统疾病中具有益处，包括肝脏疾病和移植。然而，关于其在肝衰竭儿童中的应用报告较少。最近，我们对4例肝衰竭且伴有严重凝血障碍并出血的患者使用了rFVIIa，用药后这些患者在实验室检查和临床症状方面均有显著改善，且未出现副作用。
所有4例患者均因肝衰竭、凝血障碍和出血住院，根据需要采用新鲜冰冻血浆、血小板及其他疗法控制出血。他们的国际标准化比值（INR）范围为1.7至5.8（正常范围0.9 - 1.1）。所有4例患者均因常规治疗无效的出血发作、具有高出血风险的手术或两者兼有的情况而接受rFVIIa治疗。rFVIIa的剂量范围为0.067至0.3 mg/kg。所有4例患者的INR均改善至正常或接近正常。在所有病例中，接受rFVIIa后10分钟内出血停止，且未观察到并发症。
就出血和凝血指标的临床及实验室改善而言，rFVIIa对这些肝衰竭且伴有严重凝血障碍的儿童具有显著益处。rFVIIa未产生明显副作用。该药可能是治疗肝衰竭儿童的重要工具，需要更多研究来确定儿童的最佳给药剂量。
