Urinary phenylacetylglutamine (U-PAGN) concentration as biomarker for adherence in patients with urea cycle disorders (UCD) treated with glycerol phenylbutyrate.
Urinary phenylacetylglutamine (U-PAGN) concentration as biomarker for adherence in patients with urea cycle disorders (UCD) treated with glycerol phenylbutyrate.
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DOI：
10.1016/j.ymgmr.2015.09.003
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发表时间：
2015-12
影响因子：
1.9
通讯作者：
Scharschmidt BF
中科院分区：
文献类型：
--
作者：
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
对参加 GPB 临床试验的 68 名健康成人和 66 名患有尿素循环障碍的成人和儿童患者进行苯丁酸甘油酯 (GPB) 给药期间，分析了现场尿样中的尿苯乙酰谷氨酰胺 (U-PAGN) 浓度，作为给药生物标志物。点 U-PAGN 浓度的年龄和体表面积 (BSA) 特定第 25 个百分位截止点（<2 岁患者 <~9000 μg/mL，> 2 岁且 BSA ≤ 1.3 m2 的患者 <7000 μg/mL，> 2 岁且 BSA > 1.3 m2 的 <~5000 μg/mL）确定为一种确定应评估增加剂量和/或遵守处方剂量的患者的方法。
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 <~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.