Kreuz Wolfhart, Meili Esther, Peter-Salonen Kristiina, Haertel Sabine, Devay Jan, Krzensk Udo, Egbring Rudolf
Department of Paediatrics, University of Frankfurt/Main, Germany.
Transfus Apher Sci. 2005 Jun;32(3):247-53. doi: 10.1016/j.transci.2004.08.003.
The efficacy and tolerability of a pasteurised human fibrinogen concentrate were assessed in an open, multi-centre, non-controlled retrospective study in patients with congenital fibrinogen deficiency. Haemostatic efficacy was assessed by laboratory investigation and clinical observation. The study included 12 patients (afibrinogenaemia, n = 8; hypofibrinogenaemia, n = 3; dysfibrinogenaemia combined with hypofibrinogenaemia, n = 1). Fibrinogen substitution was indicated: to stop an ongoing bleed; as prophylaxis before surgery; or for routine prophylaxis to prevent spontaneous bleeding. In total, 151 fibrinogen infusions were recorded. The median single dosage was 63.5mg/kg body weight for bleeding events or surgery and 76.9 mg/kg for prophylaxis. The median total dose per event for bleeding events or surgery was 105.6 mg/kg. Fibrinogen was administered in 26 bleeding episodes; 11 surgical operations; and 89 prophylactic infusions, of which 86 were received by one patient. The median response (n = 8) was 1.5 mg/dl per substituted mg of fibrinogen per kg body weight (0.8-2.3). The median in vivo recovery (n = 8) was 59.8% (32.5-93.9). Clinical efficacy was very good in all events with the exception of one surgical procedure, where it was moderate. No intercurrent bleeding occurred during prophylaxis. All but one infusion was well tolerated; the patient, who was administered 86 prophylactic infusions, experienced an anaphylactic reaction after the 56th infusion. In addition, one patient developed deep vein thrombosis and non-fatal pulmonary embolism with treatment for osteosynthesis after collum femoris fracture. Fibrinogen substitution could not be excluded as a contributing factor in this high-risk patient. Substitution with pasteurised human fibrinogen concentrate in patients with congenital fibrinogen deficiencies is efficient and generally well tolerated.
在一项针对先天性纤维蛋白原缺乏症患者的开放性、多中心、非对照回顾性研究中，评估了一种巴氏消毒的人纤维蛋白原浓缩物的疗效和耐受性。通过实验室检查和临床观察评估止血效果。该研究纳入了12例患者（无纤维蛋白原血症，n = 8；低纤维蛋白原血症，n = 3；异常纤维蛋白原血症合并低纤维蛋白原血症，n = 1）。进行纤维蛋白原替代治疗的指征为：停止正在进行的出血；作为手术前的预防措施；或用于预防自发性出血的常规预防。总共记录了151次纤维蛋白原输注。出血事件或手术时单次剂量的中位数为63.5mg/kg体重，预防时为76.9mg/kg。出血事件或手术时每次事件的总剂量中位数为105.6mg/kg。纤维蛋白原用于26次出血发作、11次外科手术以及89次预防性输注，其中86次预防性输注由一名患者接受。反应的中位数（n = 8）为每千克体重每替代1mg纤维蛋白原增加1.5mg/dl（0.8 - 2.3）。体内回收率的中位数（n = 8）为59.8%（32.5 - 93.9）。除了一台手术效果为中等外，所有事件的临床疗效都非常好。预防期间未发生并发出血。除一次输注外，所有输注耐受性良好；接受86次预防性输注的患者在第56次输注后发生过敏反应。此外，一名患者在股骨颈骨折后进行骨固定治疗时发生深静脉血栓形成和非致命性肺栓塞。不能排除纤维蛋白原替代是该高危患者发病的一个因素。先天性纤维蛋白原缺乏症患者使用巴氏消毒的人纤维蛋白原浓缩物进行替代治疗是有效的，且一般耐受性良好。
