肝豆状核变性(Wilson病)治疗策略的变革及思考

Shift of Therapeutic Strategy and Thinking on Treatment of Hepatolenticular Degeneration (Wilson's disease)

评析肝豆状核变性(WD)当前的治疗方法,主要有低铜饮食,铜的螯合剂青霉胺及曲恩汀,锌制剂和四硫钼酸胺,肝移植。介绍WD治疗策略的变革:不再提倡驱铜疗法以去除堆积的铜,而是使血液中的游离铜离子浓度常态化以逆转铜中毒,并结合2007年《柳叶刀》WD治疗专家共识进行思考。最后,展望未来对WD可能的根治疗法,即基因治疗、肝细胞移植及现在可施行的肝移植。

To briefly comment on current therapeutic methods of hepatolenticular degeneration （Wilson＇s disease, WD）, which mainly include low copper diet, bellacilline and trientine as copper chelators, zinc preparation and ammonium tetrathiomolybdate, and liver transplantation. To introduce shift of therapeutic strategy in treatment of WD： the treatment of symptomatic WD is no longer aimed at decoppering which means the removal of accumulated copper, but at the normalization of the free copper concentration in blood so as to reverse the copper poisoning. Moreover, thinking is carried out in combination with Panel recommendations for WD from medical treatment experts in LANCET at 2007. At last, the possible curative treatments for WE） in the future are prospected, which include genetic therapy, hepatocyte transplantation, as well as currently available liver transplantation.