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Validation of a therapeutic range for nitisinone in patients treated for tyrosinemia type 1 based on reduction of succinylacetone excretion.
JIMD Rep 2019; 46(1):75-78JR

Abstract

The drug nitisinone (NTBC; Orfadin, Vienna, Austria) has been used for the treatment of hereditary tyrosinemia type-1 since 1991. Nitisinone effectively blocks the metabolism of tyrosine to prevent the formation of the toxic compound succinylacetone (and precursor fumarylacetoacetate) in affected children. Monitoring of plasma drug levels and urine succinylacetone can be used to assess compliance and adequate dose of drug. We present retrospective data from patient monitoring for over 10 years that provide validation of a target therapeutic range for nitisinone of 40 to 60 μmol/L. The target nitisinone range is justified as valid based on reduction of succinylacetone excretion. There was no statistical significance in succinylacetone excretion in mmol/mol creatinine above a level of 40 μmol/L plasma NTBC (P > 0.05).

Authors+Show Affiliations

Department of Laboratories Seattle Children's Hospital Seattle Washington. Department of Laboratory Medicine University of Washington Seattle Washington.Division of Genetic Medicine, Department of Pediatrics University of Washington School of Medicine Seattle Washington.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

31240158

Citation

Jack, Rhona M., and C Ronald Scott. "Validation of a Therapeutic Range for Nitisinone in Patients Treated for Tyrosinemia Type 1 Based On Reduction of Succinylacetone Excretion." JIMD Reports, vol. 46, no. 1, 2019, pp. 75-78.
Jack RM, Scott CR. Validation of a therapeutic range for nitisinone in patients treated for tyrosinemia type 1 based on reduction of succinylacetone excretion. JIMD Rep. 2019;46(1):75-78.
Jack, R. M., & Scott, C. R. (2019). Validation of a therapeutic range for nitisinone in patients treated for tyrosinemia type 1 based on reduction of succinylacetone excretion. JIMD Reports, 46(1), pp. 75-78. doi:10.1002/jmd2.12023.
Jack RM, Scott CR. Validation of a Therapeutic Range for Nitisinone in Patients Treated for Tyrosinemia Type 1 Based On Reduction of Succinylacetone Excretion. JIMD Rep. 2019;46(1):75-78. PubMed PMID: 31240158.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Validation of a therapeutic range for nitisinone in patients treated for tyrosinemia type 1 based on reduction of succinylacetone excretion. AU - Jack,Rhona M, AU - Scott,C Ronald, Y1 - 2019/03/14/ PY - 2019/6/27/entrez PY - 2019/6/27/pubmed PY - 2019/6/27/medline KW - NTBC KW - Orfadin KW - nitisinone KW - succinylacetone KW - tyrosinemia SP - 75 EP - 78 JF - JIMD reports JO - JIMD Rep VL - 46 IS - 1 N2 - The drug nitisinone (NTBC; Orfadin, Vienna, Austria) has been used for the treatment of hereditary tyrosinemia type-1 since 1991. Nitisinone effectively blocks the metabolism of tyrosine to prevent the formation of the toxic compound succinylacetone (and precursor fumarylacetoacetate) in affected children. Monitoring of plasma drug levels and urine succinylacetone can be used to assess compliance and adequate dose of drug. We present retrospective data from patient monitoring for over 10 years that provide validation of a target therapeutic range for nitisinone of 40 to 60 μmol/L. The target nitisinone range is justified as valid based on reduction of succinylacetone excretion. There was no statistical significance in succinylacetone excretion in mmol/mol creatinine above a level of 40 μmol/L plasma NTBC (P > 0.05). SN - 2192-8304 UR - https://www.unboundmedicine.com/medline/citation/31240158/Validation_of_a_therapeutic_range_for_nitisinone_in_patients_treated_for_tyrosinemia_type_1_based_on_reduction_of_succinylacetone_excretion L2 - https://dx.doi.org/10.1002/jmd2.12023 DB - PRIME DP - Unbound Medicine ER -
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