Monitoring tyrosinaemia type I: Blood spot test for nitisinone (NTBC)

Clin Chim Acta. 2011 Jan 14;412(1-2):134-8. doi: 10.1016/j.cca.2010.09.027. Epub 2010 Sep 29.

Abstract

Background: Quantification of nitisinone, 2-(nitro-4-trifluoromethylbenzoyl)1,3-cyclohexanedione (NTBC) has been repeatedly described. Nevertheless monitoring of NTBC has not yet become part of routine therapy surveillance in tyrosinaemia type I (OMIM 276700). We developed a blood spot test to facilitate collection and transport of samples. Furthermore, the test material can be used for determination of other parameters like tyrosine and succinylacetone.

Method: For quantification of NTBC in blood spots filter paper discs of 3.2mm diameter were extracted with 150 μL methanol containing mesotrione as internal standard (IS). Analysis was done by UPLC-MS/MS on a Xevo mass spectrometer (ESI+), (MRM). Parent ions were 330.05 for NTBC and 340.05 for IS, daughter ions were m/z 217.95 and m/z 125.95 for NTBC, and m/z 227.95 and m/z 103.95 for IS.

Results: The calibration curve for NTBC in blood spots was linear from 0.1 μmol/L to 100 μmol/L. Recovery exceeded 73.1%, CV intraday and interday were below 9.6%. Instrumental run time was 2.5 min. Sensitivity of the method was 0.1 μmol/L. NTBC concentrations in plasma were higher than in blood spots by a factor of 1.56 ± 0.13.

Conclusion: As demonstrated in patients with tyrosinaemia type I quantification of NTBC by UPLC-MS/MS in blood spots is feasible and gives valuable information for monitoring NTBC treatment.

MeSH terms

  • Blood Chemical Analysis / methods*
  • Cyclohexanones / blood*
  • Cyclohexanones / isolation & purification
  • Cyclohexanones / therapeutic use
  • Follow-Up Studies
  • Humans
  • Male
  • Nitrobenzoates / blood*
  • Nitrobenzoates / isolation & purification
  • Nitrobenzoates / therapeutic use
  • Specimen Handling / methods*
  • Treatment Outcome
  • Tyrosinemias / blood*
  • Tyrosinemias / drug therapy

Substances

  • Cyclohexanones
  • Nitrobenzoates
  • nitisinone