An LC-MS/MS-Based Method for the Quantification of Pyridox(am)ine 5'-Phosphate Oxidase Activity in Dried Blood Spots from Patients with Epilepsy

Anal Chem. 2017 Sep 5;89(17):8892-8900. doi: 10.1021/acs.analchem.7b01358. Epub 2017 Aug 17.

Abstract

We report the development of a rapid, simple, and robust LC-MS/MS-based enzyme assay using dried blood spots (DBS) for the diagnosis of pyridox(am)ine 5'-phosphate oxidase (PNPO) deficiency (OMIM 610090). PNPO deficiency leads to potentially fatal early infantile epileptic encephalopathy, severe developmental delay, and other features of neurological dysfunction. However, upon prompt treatment with high doses of vitamin B6, affected patients can have a normal developmental outcome. Prognosis of these patients is therefore reliant upon a rapid diagnosis. PNPO activity was quantified by measuring pyridoxal 5'-phosphate (PLP) concentrations in a DBS before and after a 30 min incubation with pyridoxine 5'-phosphate (PNP). Samples from 18 PNPO deficient patients (1 day-25 years), 13 children with other seizure disorders receiving B6 supplementation (1 month-16 years), and 37 child hospital controls (5 days-15 years) were analyzed. DBS from the PNPO-deficient samples showed enzyme activity levels lower than all samples from these two other groups as well as seven adult controls; no false positives or negatives were identified. The method was fully validated and is suitable for translation into the clinical diagnostic arena.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Area Under Curve
  • Case-Control Studies
  • Child
  • Child, Preschool
  • Chromatography, High Pressure Liquid / methods*
  • Dried Blood Spot Testing
  • Epilepsy / diagnosis*
  • Epilepsy / drug therapy
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Pyridoxal Phosphate / blood
  • Pyridoxamine / analogs & derivatives
  • Pyridoxamine / blood
  • Pyridoxaminephosphate Oxidase / metabolism*
  • ROC Curve
  • Tandem Mass Spectrometry / methods*
  • Vitamin B 6 / chemistry
  • Vitamin B 6 / metabolism
  • Vitamin B 6 / therapeutic use
  • Young Adult

Substances

  • Pyridoxal Phosphate
  • Pyridoxamine
  • Vitamin B 6
  • Pyridoxaminephosphate Oxidase
  • pyridoxamine phosphate