Gkiougki Evangelia, Mitsiakos Georgios, Chatziioannidis Elias, Papadakis Emmanouel, Nikolaidis Nikolaos
Neonatal Intensive Care Unit and Neonatal Department, Aristotle University of Thessaloniki, Thessaloniki, Greece.
J Pediatr Hematol Oncol. 2013 Apr;35(3):221-6. doi: 10.1097/MPH.0b013e318286d27e.
To date, clinical experience with recombinant factor VIIa (rFVIIa) in neonates is rather limited because of the lack of controlled studies. ΑIM: The objective of this study was to present further experience from our center with regard to the use of rFVIIa in newborns with severe bleeding or coagulopathy resistant to conventional therapy and to determine factors affecting the clinical outcome.
We performed a retrospective data analysis of 29 neonates with intractable bleeding or severe coagulation disturbances. All patients received 100 μg/kg of rFVIIa per dose bolus intravenously (maximum of 23 doses), as rescue procedure after other interventions had failed to achieve hemostasis.
Fourteen neonates survived (group A), whereas 15 died (group B). There was no difference in birth weight, gestational age, and bleeding site and causes between the 2 groups. In the neonates who survived, rFVIIa had been administered earlier in the disease process (<24 h of beginning of bleeding) compared with those who died (P=0.009). In all 29 neonates, international normalized ratio was directly restored (from 2.99±1.4 before rFVIIa administration to 1.6±1.1 afterward, P<0.001) and prothrombin time and activated partial thromboplastin time were significantly decreased after administration of rFVIIa (from 28 to 16.4 and from 180 to 67, respectively; P=0.001 and 0.05, respectively). Blood products administered were significantly less in group A than in group B, as time from the beginning of bleeding to the administration of rFVIIa was significantly less in group A than in group B. Neither acute adverse events nor thromboembolic complications were observed.
In this neonatal group with intractable bleeding and/or severe coagulation disturbances, rFVIIa was more effective in early intervention as rescue therapy, without any adverse events in all neonates. Upon failure to achieve hemostasis with initial administration of blood products, fast intervention with rFVIIa could be considered in neonates with serious bleeding and coagulation disorders.
由于缺乏对照研究，迄今为止重组凝血因子VIIa（rFVIIa）在新生儿中的临床经验相当有限。目的：本研究的目的是介绍我们中心在使用rFVIIa治疗对传统治疗耐药的严重出血或凝血病新生儿方面的更多经验，并确定影响临床结局的因素。
我们对29例难治性出血或严重凝血障碍的新生儿进行了回顾性数据分析。所有患者均接受每剂100μg/kg的rFVIIa静脉推注（最大23剂），作为其他干预措施未能实现止血后的挽救措施。
14例新生儿存活（A组），15例死亡（B组）。两组在出生体重、胎龄、出血部位和原因方面无差异。与死亡的新生儿相比，存活的新生儿在疾病过程中更早接受了rFVIIa治疗（出血开始后<24小时）（P=0.009）。在所有29例新生儿中，国际标准化比值直接恢复（rFVIIa给药前为2.99±1.4，给药后为1.6±1.1，P<0.001），rFVIIa给药后凝血酶原时间和活化部分凝血活酶时间显著缩短（分别从28降至16.4和从180降至67；P分别为0.001和0.05）。A组输注的血液制品明显少于B组，因为A组从出血开始到给予rFVIIa的时间明显短于B组。未观察到急性不良事件或血栓栓塞并发症。
在这个难治性出血和/或严重凝血障碍的新生儿群体中，rFVIIa作为挽救治疗在早期干预中更有效，且所有新生儿均无不良事件。在初始输注血液制品未能实现止血的情况下，对于严重出血和凝血障碍的新生儿可考虑快速给予rFVIIa进行干预。
