尼替西农用于1型遗传性酪氨酸血症药物治疗的经验。
Experience of nitisinone for the pharmacological treatment of hereditary tyrosinaemia type 1.
作者信息
Santra Saikat, Baumann Ulrich
机构信息
Birmingham Children's Hospital, The Liver Unit, Steelhouse Lane, Birmingham, B4 6NH, UK.
出版信息
Expert Opin Pharmacother. 2008 May;9(7):1229-36. doi: 10.1517/14656566.9.7.1229.
BACKGROUND
Hereditary tyrosinaemia type 1 is a rare inherited metabolic condition, which leads to a fatal multisystemic disease in childhood. Since 1992, nitisinone - a compound developed from work on triketone herbicides - has become an effective pharmacological treatment by inhibiting the enzyme 4-hydroxyphenylpyruvate dioxygenase.
OBJECTIVES
This review examines recent pharmacological and clinical literature on nitisinone, and assesses its impact as a pharmacological treatment for hereditary tyrosinaemia type 1.
METHODS
English language literature from MedLine and EmBase for nitisinone was searched from 1990 to 2008 for all papers relevant to the use of nitisinone in hereditary tyrosinaemia type 1.
CONCLUSIONS
Nitisinone can prevent the development of liver disease and significantly reduce the risk of developing hepatocellular carcinoma; however, vigorous surveillance for the development of HCC needs to be continued lifelong.
背景
1型遗传性酪氨酸血症是一种罕见的遗传性代谢疾病，可导致儿童期致命的多系统疾病。自1992年以来，尼替西农——一种从三酮类除草剂研究中开发出的化合物——通过抑制4-羟基苯丙酮酸双加氧酶，已成为一种有效的药物治疗方法。
目的
本综述研究了关于尼替西农的近期药理学和临床文献，并评估其作为1型遗传性酪氨酸血症药物治疗的影响。
方法
检索1990年至2008年MedLine和EmBase中关于尼替西农的英文文献，查找所有与尼替西农用于1型遗传性酪氨酸血症相关的论文。
结论
尼替西农可预防肝脏疾病的发展，并显著降低肝细胞癌的发生风险；然而，需要终身持续对肝细胞癌的发生进行严格监测。
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