Urinary phenylacetylglutamine (U-PAGN) concentration as biomarker for adherence in patients with urea cycle disorders (UCD) treated with glycerol phenylbutyrate

M. Mokhtarani, G. A. Diaz, U. Lichter-Konecki, S. A. Berry, J. Bartley, S. E. McCandless, W. Smith, C. Harding, C. Le Mons, D. F. Coakley, B. Lee, B. F. Scharschmidt

Research output: Contribution to journalArticle

2 Scopus citations

Abstract

Urinary phenylacetylglutamine (U-PAGN) concentrations in spot urine samples were analyzed as a dosing biomarker during glycerol phenylbutyrate (GPB) dosing in 68 healthy adults and 66 adult and pediatric patients with urea cycle disorders who participated in GPB clinical trials. Age- and body surface area (BSA)-specific 25th percentile cutoff points for spot U-PAGN concentrations (<∼9000 μg/mL for < 2 years old patients, < 7000 μg/mL for > 2 years with BSA ≤ 1.3 m2 and <sim;5000 μg/mL for > 2 years of age with BSA > 1.3 m2) were determined as an approach to identify patients for whom increased dosing and/or adherence to prescribed dosing should be assessed.

Original languageEnglish (US)
Pages (from-to)12-14
Number of pages3
JournalMolecular Genetics and Metabolism Reports
Volume5
DOIs
StatePublished - Dec 1 2015

Keywords

  • Biomarker
  • Glycerol phenylbutyrate
  • Phenylacetate
  • Phenylbutyrate
  • Urea cycle disorder
  • Urinary phenylacetylglutamine

ASJC Scopus subject areas

  • Molecular Biology
  • Genetics
  • Endocrinology

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    Mokhtarani, M., Diaz, G. A., Lichter-Konecki, U., Berry, S. A., Bartley, J., McCandless, S. E., Smith, W., Harding, C., Le Mons, C., Coakley, D. F., Lee, B., & Scharschmidt, B. F. (2015). Urinary phenylacetylglutamine (U-PAGN) concentration as biomarker for adherence in patients with urea cycle disorders (UCD) treated with glycerol phenylbutyrate. Molecular Genetics and Metabolism Reports, 5, 12-14. https://doi.org/10.1016/j.ymgmr.2015.09.003