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, Cary Harding, C. Le Mons, D. F. Coakley, B. Lee, B. F. Scharschmidt

Research output: Contribution to journalArticle

1 Citation (Scopus)

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 ( 2 years with BSA ≤ 1.3 m2 and 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

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Inborn Urea Cycle Disorder
Body Surface Area
Patient Compliance
Biomarkers
Clinical Trials
Urine
Pediatrics
phenylacetylglutamine
glycerol phenylbutyrate

Keywords

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

ASJC Scopus subject areas

  • Endocrinology
  • Genetics
  • Molecular Biology

Cite this

Urinary phenylacetylglutamine (U-PAGN) concentration as biomarker for adherence in patients with urea cycle disorders (UCD) treated with glycerol phenylbutyrate. / Mokhtarani, M.; Diaz, G. A.; Lichter-Konecki, U.; Berry, S. A.; Bartley, J.; McCandless, S. E.; Smith, W.; Harding, Cary; Le Mons, C.; Coakley, D. F.; Lee, B.; Scharschmidt, B. F.

In: Molecular Genetics and Metabolism Reports, Vol. 5, 01.12.2015, p. 12-14.

Research output: Contribution to journalArticle

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