Morita J, Yoshino M, Watari H, Yoshida I, Motohiro T, Yamashita F, Okano Y, Hashimoto T
Department of Pediatrics and Child Health, Kurume University School of Medicine, Fukuoka, Japan.
Dev Pharmacol Ther. 1992;19(1):6-9. doi: 10.1159/000457456.
Wilson's disease is an autosomal recessive disorder characterized by an accumulation of a toxic amount of copper in the body. Triethylene tetramine dihydrochloride (trientine, 2HCl) is a new chelating agent that may be effective in the removal of excess copper but long-term efficacy has not yet been investigated. Here we report the use of trientine over more than 8 years in 2 patients with Wilson's disease who could not tolerate D-penicillamine. We found no significant side effect, except a decreased serum iron concentration without clinical symptoms of anemia. In annual examinations at a steady state, the serum copper levels remained below 20 micrograms/100 ml. The 24-hour urinary copper excretion was less than that found using D-penicillamine, while the basal copper excretion, after 5 days abstinence from trientine, was maintained below 100 micrograms/day. Both hepatic and neurological manifestations except bulbar symptoms were recovered without any initial deterioration.
威尔逊氏病是一种常染色体隐性疾病，其特征是体内蓄积有毒量的铜。三乙烯四胺二盐酸盐（曲恩汀，2HCl）是一种新型螯合剂，可能对去除过量铜有效，但尚未对其长期疗效进行研究。在此，我们报告了2例无法耐受D-青霉胺的威尔逊氏病患者使用曲恩汀超过8年的情况。我们发现除血清铁浓度降低但无贫血临床症状外，无明显副作用。在稳定状态下的年度检查中，血清铜水平保持在20微克/100毫升以下。24小时尿铜排泄量低于使用D-青霉胺时的水平，而在停用曲恩汀5天后的基础铜排泄量维持在100微克/天以下。除延髓症状外，肝脏和神经方面的表现均得到恢复，且初期无任何恶化。
