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

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.

• Authors: Saikat Santra, Ulrich Baumann
• Journal: Expert opinion on pharmacotherapy (Expert Opin Pharmacother) Vol. 9 Issue 7 Pg. 1229-36 (May 2008) ISSN: 1744-7666 [Electronic] England
• PMID: 18422479 (Publication Type: Journal Article, Review)

Chemical References

• Cyclohexanones
• Enzyme Inhibitors
• Nitrobenzoates
• 4-Hydroxyphenylpyruvate Dioxygenase
• nitisinone

Topics

• 4-Hydroxyphenylpyruvate Dioxygenase (antagonists & inhibitors)
• Carcinoma, Hepatocellular (etiology, prevention & control)
• Child
• Cyclohexanones (adverse effects, pharmacology, therapeutic use)
• Enzyme Inhibitors (adverse effects, pharmacology, therapeutic use)
• Humans
• Liver Diseases (etiology, prevention & control)
• Liver Neoplasms (etiology, prevention & control)
• Monitoring, Physiologic
• Nitrobenzoates (adverse effects, pharmacology, therapeutic use)
• Tyrosinemias (complications, drug therapy, physiopathology)