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

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 surfac...

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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2015
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5471406/
https://www.ncbi.nlm.nih.gov/pubmed/28649536
http://dx.doi.org/10.1016/j.ymgmr.2015.09.003
Descripción
Sumario: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 m(2), and <~5000 μg/mL for > 2 years of age with BSA > 1.3 m(2)) 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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