Anderson L A, Hakojarvi S L, Boudreaux S K
Multum Information Services, Inc., Denver, CO 80209, USA.
Ann Pharmacother. 1998 Jan;32(1):78-87. doi: 10.1345/aph.17075.
To briefly review the pathophysiology and diagnosis of Wilson's disease, and to evaluate the pharmacology, pharmacokinetics, clinical utility, adverse effects, dosing regimens, and pharmacoeconomics of zinc acetate therapy in Wilson's disease.
A MEDLINE search (December 1966-December 1996) of the English-language literature using the terms zinc and Wilson's disease was conducted to identify pertinent clinical trials, review articles, and case reports. Additional articles were selected from bibliographies of the reviewed literature.
Due to the rarity of the disease, all articles were considered for possible inclusion in this review. Single case reports are referenced, but were not selected for evaluation.
Wilson's disease, an inherited disorder of copper metabolism, is fatal if untreated. The chelating drugs penicillamine and trientine have been the mainstay of therapy; however, adverse reactions of chelators often interfere with successful treatment. Recently, zinc acetate was approved in the US for maintenance therapy in patients initially treated with a chelating agent. Although studies evaluating large populations are lacking zinc therapy has demonstrated exceptional safety and efficacy over a period of 40 years. Zinc acetate can be used during pregnancy and for the treatment of presymptomatic patients, although data do not support its use as monotherapy in patients with acute neurologic or hepatic disease.
Zinc acetate is an effective maintenance therapy for patients with Wilson's disease. Negligible toxicity, compared with that of previously approved treatments, is a major advantage.
简要回顾肝豆状核变性的病理生理学和诊断方法，并评估醋酸锌疗法在肝豆状核变性中的药理学、药代动力学、临床应用、不良反应、给药方案及药物经济学。
利用“锌”和“肝豆状核变性”等关键词对1966年12月至1996年12月期间的英文文献进行MEDLINE检索，以识别相关的临床试验、综述文章及病例报告。从所查阅文献的参考文献中选取其他文章。
由于该病罕见，所有文章均被考虑纳入本综述。引用了单病例报告，但未选作评估之用。
肝豆状核变性是一种遗传性铜代谢紊乱疾病，若不治疗会致命。螯合剂青霉胺和曲恩汀一直是主要治疗药物；然而，螯合剂的不良反应常常干扰治疗的成功进行。最近，醋酸锌在美国被批准用于经螯合剂初始治疗患者的维持治疗。尽管缺乏评估大量人群的研究，但锌疗法在40年期间已显示出卓越的安全性和有效性。醋酸锌可在孕期使用，也可用于治疗症状前患者，尽管数据不支持将其作为急性神经或肝脏疾病患者的单一疗法。
醋酸锌是肝豆状核变性患者有效的维持治疗药物。与先前批准的治疗方法相比，其毒性可忽略不计是一个主要优点。
