Molecular Genetics and Metabolism Reports (Dec 2015)

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

DOI
https://doi.org/10.1016/j.ymgmr.2015.09.003
Journal volume & issue
Vol. 5, no. C
pp. 12 – 14

Abstract

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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.

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