Petru A M, Azimi P H, Cummins S K, Sjoerdsma A
Department of Infectious Diseases, Children's Hospital Oakland, CA 94609.
Am J Dis Child. 1988 Feb;142(2):224-8. doi: 10.1001/archpedi.1988.02150020126047.
The traditional treatment of African sleeping sickness (trypanosomiasis) with central nervous system involvement is an organic arsenical compound, melarsoprol, which is associated with severe and even life-threatening side effects. A polyamine biosynthesis inhibitor, eflornithine (chemical name, DL-alpha-difluoromethylornithine, supplied as monohydrochloride monohydrate), was used to treat a 3 1/2-year-old child with newly diagnosed severe trypanosomiasis that had been acquired more than two years previously in Zaire or the Congo. Treatment consisted of 300 to 400 mg/kg/d of eflornithine by continuous intravenous infusion for 25 days followed by 300 mg/kg/d of eflornithine by mouth divided in four equal doses daily for 17 days. The child's recovery was dramatic, with eradication of blood and cerebrospinal fluid parasites in the first week. Cerebrospinal fluid pleocytosis resolved completely. Her generalized adenopathy and fever gradually resolved. Severe ataxia, inability to walk or to change posture on her own, marked language regression, and lethargy all improved during and after her therapy. The drug was well tolerated; the only noted adverse effect was transient thrombocytopenia during the fourth week of therapy. Eflornithine was a safe and effective agent for treatment of trypanosomiasis with central nervous system involvement in this child.
对于累及中枢神经系统的非洲昏睡病（锥虫病），传统治疗方法是使用一种有机砷化合物美拉胂醇，但它会引发严重甚至危及生命的副作用。一种多胺生物合成抑制剂依氟鸟氨酸（化学名称为DL-α-二氟甲基鸟氨酸，以盐酸盐一水合物形式供应）被用于治疗一名3岁半的儿童，该儿童新诊断为重度锥虫病，两年多前在扎伊尔或刚果感染。治疗方法为持续静脉输注依氟鸟氨酸，剂量为300至400毫克/千克/天，持续25天，随后口服依氟鸟氨酸，剂量为300毫克/千克/天，分四等份，每日一次，持续17天。患儿恢复显著，第一周时血液和脑脊液中的寄生虫被清除。脑脊液细胞增多症完全消退。她的全身淋巴结病和发热逐渐消退。严重的共济失调、无法自行行走或改变姿势、明显的语言退化和嗜睡在治疗期间及治疗后均有所改善。该药物耐受性良好；唯一观察到的不良反应是治疗第四周出现的短暂性血小板减少。依氟鸟氨酸是治疗该例累及中枢神经系统锥虫病患儿的一种安全有效的药物。
