我们以前已经报导过铁螯合剂-去铁胺能够阻止小鼠肝脏的损伤和癌前病变的发展，并且我们已经提出去铁胺可以作为抗癌药物。去铁胺具有抗增殖效应，且可以阻止细胞周期，并最终导致细胞凋亡。据我们所知还没有相关临床研究去评价肝癌患者应用去铁胺治疗的疗效。我们的研究有10名患者参与（6名男性和4名女性），他们均处于肝细胞癌晚期，并且对肝动脉灌注化疗与抗癌药物治疗反应差。他们的平均年龄是64岁（从43到77岁），进行研究前已取得他们的知情同意权，并且已获得山口大学医院的研究评审委员会的批准。其中7个患者的病因为丙肝病毒感染，2个为乙肝病毒感染，1个没有任何感染。1个患者的肿瘤阶段处于Ⅱ期，2个处于ⅣA期，7个处于ⅣB期。同时对他们进行Child–Pugh 分级，A级的有3名, B级的5名, C级的2名. (在 Child–Pugh分级中， A级说明最不严重, B级说明中度严重, C级说明最严重)。 患者们接受去铁胺的动脉灌输治疗 (剂量为 10-80 mg /Kg) 隔日一次。 注射去铁胺的次数平均是27次（9-78次），经治疗后，2个患者获得部分反应，3个患者疾病停止恶化，5个患者仍然进一步恶化. 总的应答率是20%。机体铁容易过量丢失，造成机体缺铁状态继而 引起缺铁症状从而对机体造成损伤。
吉林大学第一医院肝胆胰内科 董爱莲 摘译
本文首次发表于[N. Engl. J. Med. 2011 365(6):576-578]
Deferoxamine for Advanced Hepatocellular Carcinoma
The basis of research We have previously reported that the iron chelator deferoxamine can prevent liver injury as well as the development of preneoplastic lesions in rats, and we have proposed the use of deferoxamine as an anticancer drug.The antiproliferative effect of deferoxamine arrests the cell cycle and induces apoptosis. To our knowledge, no clinical study has been performed to evaluate deferoxamine therapy in patients with hepatocellular carcinoma. The process of research Our study involved 10 patients (6 men and 4 women) who had advanced hepatocellular carcinoma and did not have a response to hepatic arterial infusion chemotherapy with anticancer drugs. The average age of the patients was 64 years (range, 43 to 77). Written informed consent was obtained before the study, which was approved by the institutional review board of Yamaguchi University Hospital. Seven patients had hepatitis C virus infection, 2 patients had hepatitis B virus infection, and 1 patient did not have either type of infection. The tumor stages were classified as II, IVA, and IVB (according to the Liver Cancer Study Group of Japan criteria) for 1, 2, and 7 patients, respectively. The Child–Pugh class was A, B, and C for 3, 5, and 2 patients, respectively. (In the Child–Pugh classification of liver disease, class A indicates the least severe disease, class B moderately severe disease, and class C the most severe disease.) The patients received an arterial infusion of deferoxamine (at a dose of 10 to 80 mg per kilogram of body weight) over 24 hours on alternate days, through the injection port. The result of research Deferoxamine was administered an average of 27 times (range, 9 to 78). Two, three, and five patients had a partial response, stable disease, and progressive disease, respectively (according to the Eastern Cooperative Oncology Group criteria). The overall response rate was 20%.
