CAS No. : 104206-65-7

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For research use only. Not Intended for Therapeutic Use!
Cat No:I000243
Molecular Formula:C14H10F3NO5
Molecular Weight:329.2
Target:4-Hydroxyphenylpyruvate Dioxygenase
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Appearance:Solid powder
Purity: > 98%
Cat No:I000243
Cas No:104206-65-7

Nitisinone(cas# 104206-65-7), also known as SC0735, is a reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase, an enzyme involved in the catabolism of tyrosine in mammals and the biosynthesis of plastoquinone in plants. 

Nitisinone has been evaluated as a herbicide, while also used in the treatment of hereditary tyrosinemia type 1, alkaptonuria, and oculocutaneous albinism, all of which involve a disorder in tyrosine metabolism.

1. Orphanet J Rare Dis. 2017 Sep 11;12(1):154. doi: 10.1186/s13023-017-0696-z.

Evaluation of pre-symptomatic nitisinone treatment on long-term outcomes in Tyrosinemia type 1 patients: a systematic review.

Geppert J(1), Stinton C(1), Freeman K(1), Fraser H(1), Clarke A(1), Johnson S(2), Sutcliffe P(1), Taylor-Phillips S(3).
Author information:
(1)Warwick Medical School, University of Warwick, Coventry, CV4 7AL, UK. (2)Warwick Library, University of Warwick, Coventry, CV4 7AL, UK. (3)Warwick Medical School, University of Warwick, Coventry, CV4 7AL, UK.
BACKGROUND: Tyrosinemia type 1 (TYR1) is a rare autosomal recessive disorder of amino acid metabolism that is fatal without treatment. With medication (nitisinone) and dietary restrictions outcomes are improved. We conducted a systematic review to investigate if treatment with nitisinone following screening provides better long-term outcomes than treatment with nitisinone following symptomatic detection.
METHODS: We searched Web of Science, Medline, Pre-Medline, and Embase up to 23rd September 2016 for journal articles comparing clinical outcomes of TYR1 patients receiving earlier versus later nitisinone treatment. Two reviewers independently screened titles and abstracts, assessed full texts, and appraised study quality. Data extraction was performed by a single reviewer and checked by a second. RESULTS: We included seven articles out of 470 unique records identified by our search. The seven articles included four studies (three cohort studies and one cross-sectional study). Study sample sizes ranged from 17 to 148. There is consistent evidence that nitisinone is an effective treatment for TYR1, and some evidence that earlier treatment with nitisinone and dietary restrictions within the first one or 2 months of life is associated with reduced need for liver transplantation, lower rates of renal dysfunction, fewer neurological crises, and fewer, shorter hospital admissions compared to later treatment. However, study quality was moderate to weak, with high risk of confounding and applicability concerns to the screening context. We conducted post hoc analyses to address these issues. Results suggested an association between earlier treatment and fewer liver transplants (earlier treatment: 0% of 10-24 patients; later treatment: 25-60% of 4-15 patients), but no impact on neurological crises. We found no effect of treatment timing on mortality in either the primary or post hoc analyses. Post hoc analyses of other health-related outcomes were not possible because of sample size or reporting.
CONCLUSIONS: There is some evidence from observational studies that earlier treatment with nitisinone might be beneficial but this is subject to bias. The applicability of our findings to the screening context or clinical practice is limited as not all early-treated patients were identified by screening and late-treated groups included patients born prior to the availability of nitisinone.

2. Appl Clin Genet. 2017 Jul 24;10:43-48. doi: 10.2147/TACG.S113310. eCollection 2017.

Clinical utility of nitisinone for the treatment of hereditary tyrosinemia type-1 (HT-1).

Das AM(1).
Author information:
(1)Department of Pediatrics, Hannover Medical School, Hannover, Germany.
Medical therapy for hereditary hepatorenal tyrosinemia (hereditary tyrosinemia type 1, HT-1) with nitisinone was discovered incidentally, and is a by-product of agrochemistry. It blocks the catabolic pathway of tyrosine, thereby leading to a reduction in the accumulation of toxic metabolites in HT-1. It has to be combined with a low-protein diet supplemented with amino acid mixtures devoid of tyrosine and phenylalanine. This treatment option has completely changed the clinical course of patients suffering from HT-1 who used to die in the first few months to years of life from liver failure, renal dysfunction, and/or hepatocellular carcinoma (HCC). It is essential to start nitisinone therapy early in life to avoid sequelae; beginning treatment in the newborn period is ideal. As initial clinical symptoms of HT-1 are often atypical and because there is a clinically latent phase during the first few months of life in many patients, newborn screening is required to secure early diagnosis. Succinylacetone in blood is a reliable screening parameter whereas tyrosine is neither specific nor sensitive. Especially HCC, but also liver and kidney dysfunction, rickets, and neurological crises can be prevented in most patients if nitisinone therapy is started in the newborn period. It is essential to adhere to a low-protein diet to avoid tyrosine toxicity. Reversible eye symptoms may occur as a side-effect of nitisinone, but other side effects are rare. Neurocognitive development is impaired in some patients, and the reason for this is unclear. Metabolic monitoring includes measurement of tyrosine, succinylacetone, and nitisinone concentrations in blood.

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

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

Santra S(1), Baumann U.
Author information:
(1)Birmingham Children/'s Hospital, The Liver Unit, Steelhouse Lane, Birmingham, B4 6NH, UK.
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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