University of Occupational and Environmental Health, Japan, Kitakyushu, Fukuoka, Japan,
Eur J Pediatr. 2014 Mar;173(3):303-11. doi: 10.1007/s00431-013-2155-8. Epub 2013 Sep 5.
Recombinant soluble human thrombomodulin (TM-α) has been shown to be useful in the treatment of disseminated intravascular coagulation (DIC) in a heparin-controlled study and has been available for clinical use in Japan since 2008. However, data on its use for neonatal DIC have not been reported from any clinical studies, so efficacy and safety were analyzed in 60 neonatal DIC patients identified in post-marketing surveillance. The DIC resolution rate as of the day after last administration of TM-α was 47.1 %, and the survival rate at 28 days after last administration was 76.7 %. Hemostatic test result profiles revealed decreased levels of fibrin/fibrinogen degradation products and increased platelet counts and antithrombin activity. Incidences of adverse drug reactions, bleeding-related adverse drug reactions, and bleeding-related adverse events were 6.7, 6.7, and 16.7 %, respectively, with no significant differences between neonatal, pediatric (excluding neonates), and adult DIC patients.
This surveillance provided real-world data on the safety and effectiveness of TM-alpha in the treatment of neonatal DIC in general practice settings.
目的：评估在一般实践环境中，使用重组可溶性人血栓调节蛋白（TM-α）治疗新生儿弥漫性血管内凝血（DIC）的安全性和有效性。
方法：对 60 例新生儿 DIC 患者进行了回顾性分析，这些患者来自于上市后监测研究。分析了患者的临床特征、治疗方案、实验室检查结果、不良反应和预后。
结果：TM-α治疗后第 2 天，DIC 缓解率为 47.1%，治疗后第 28 天的存活率为 76.7%。止血试验结果显示，纤维蛋白/纤维蛋白原降解产物水平降低，血小板计数和抗凝血酶活性增加。不良反应、出血相关不良反应和出血相关不良事件的发生率分别为 6.7%、6.7%和 16.7%，新生儿、儿科（不包括新生儿）和成人 DIC 患者之间无显著差异。
结论：本研究提供了 TM-α 治疗新生儿 DIC 的安全性和有效性的真实世界数据。
