Experience of nitisinone for the pharmacological treatment of hereditary tyrosinaemia type 1.

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Santra S, Baumann U

Experience of nitisinone for the pharmacological treatment of hereditary tyrosinaemia type 1.

Expert Opin Pharmacother. 2008 May;9(7):1229-36. doi: 10.1517/14656566.9.7.1229.

PubMed ID

18422479 [ View in PubMed

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Abstract

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.

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Drug Targets

Drug	Target	Kind	Organism	Pharmacological Action	Actions
Nitisinone	4-hydroxyphenylpyruvate dioxygenase	Protein	Humans	Yes	Inhibitor	Details