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:10.1016/j.ymgmr.2015.09.003

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

Pediatrics - Genetics and Metabolism

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6 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 language	English (US)
Pages (from-to)	12-14
Number of pages	3
Journal	Molecular Genetics and Metabolism Reports
Volume	5
DOIs	https://doi.org/10.1016/j.ymgmr.2015.09.003
State	Published - Dec 1 2015

Bibliographical note

Publisher Copyright:
© 2015 Published by Elsevier Inc.

Keywords

Biomarker
Glycerol phenylbutyrate
Phenylacetate
Phenylbutyrate
Urea cycle disorder
Urinary phenylacetylglutamine

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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

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

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

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 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.",

keywords = "Biomarker, Glycerol phenylbutyrate, Phenylacetate, Phenylbutyrate, Urea cycle disorder, Urinary phenylacetylglutamine",

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

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doi = "10.1016/j.ymgmr.2015.09.003",

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AU - Diaz, G. A.

AU - Lichter-Konecki, U.

AU - Berry, S. A.

AU - Bartley, J.

AU - McCandless, S. E.

AU - Smith, W.

AU - Harding, C.

AU - Le Mons, C.

AU - Coakley, D. F.

AU - Lee, B.

AU - Scharschmidt, B. F.

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KW - Biomarker

KW - Glycerol phenylbutyrate

KW - Phenylacetate

KW - Phenylbutyrate

KW - Urea cycle disorder

KW - Urinary phenylacetylglutamine

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

Abstract

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Keywords

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