Detection of beta-ureidopropionase deficiency with HPLC-electrospray tandem mass spectrometry and confirmation of the defect at the enzyme level

J Inherit Metab Dis. 2001 Dec;24(7):725-32. doi: 10.1023/a:1012997406132.

Abstract

The pyrimidine bases uracil and thymine are degraded via the consecutive action of three enzymes to beta-alanine and beta-aminoisobutyric acid, respectively. To date, a number of patients have been described with a deficiency of dihydropyrimidine dehydrogenase and dihydropyrimidinase, the first two enzymes of the pyrimidine degradation pathway. In this study, we demonstrate that the first patient presenting with N-carbamyl-beta-amino aciduria, due to a deficiency of beta-ureidopropionase, was easily diagnosed at the metabolite level using HPLC-tandem mass spectrometry. Urinary analysis showed strongly elevated levels of N-carbamyl-beta-alanine and N-carbamyl-beta-aminoisobutyric acid, with normal or moderately increased levels of the pyrimidine bases and the dihydropyrimidines, respectively. The deficiency of beta-ureidopropionase was confirmed by measuring all three enzymes of the pyrimidine degradation pathway. No activity of beta-ureidopropionase could be detected in a liver biopsy of the patient, while a normal activity of dihydropyrimidine dehydrogenase and dihydropyrimidinase was present. Thus, HPLC-tandem mass specrometry proved to be a powerful tool for the initial diagnosis of patients with deficiency of beta-ureidopropionase.

Publication types

  • Case Reports

MeSH terms

  • Amidohydrolases / analysis
  • Amidohydrolases / deficiency*
  • Aminoisobutyric Acids / analysis
  • Chromatography, High Pressure Liquid*
  • Dihydrouracil Dehydrogenase (NADP)
  • Female
  • Humans
  • Infant
  • Liver / enzymology*
  • Mass Spectrometry*
  • Oxidoreductases / analysis
  • Pyrimidines / metabolism
  • Spectrometry, Mass, Electrospray Ionization

Substances

  • Aminoisobutyric Acids
  • Pyrimidines
  • Oxidoreductases
  • Dihydrouracil Dehydrogenase (NADP)
  • Amidohydrolases
  • beta-ureidopropionase
  • dihydropyrimidinase
  • pyrimidine
  • 3-aminoisobutyric acid