尿苯乙酰谷氨酰胺（U-PAGN）浓度作为接受苯丁酸钠甘油酯治疗的尿素循环障碍（UCD）患者依从性的生物标志物。
Urinary phenylacetylglutamine (U-PAGN) concentration as biomarker for adherence in patients with urea cycle disorders (UCD) treated with glycerol phenylbutyrate.
作者信息
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
机构信息
Horizon Pharma, Brisbane, CA, USA.
Mount Sinai School of Medicine, New York, NY, USA.
出版信息
Mol Genet Metab Rep. 2015 Sep 27;5:12-14. doi: 10.1016/j.ymgmr.2015.09.003. eCollection 2015 Dec.
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 m, and <5000 μg/mL for > 2 years of age with BSA > 1.3 m) were determined as an approach to identify patients for whom increased dosing and/or adherence to prescribed dosing should be assessed.
摘要
在68名健康成年人以及66名参与甘油苯丁酸盐（GPB）临床试验的成年和儿科尿素循环障碍患者中，分析了即时尿样中的尿苯乙酰谷氨酰胺（U-PAGN）浓度，将其作为GPB给药期间的剂量生物标志物。确定了按年龄和体表面积（BSA）划分的即时U-PAGN浓度第25百分位数截止点（2岁以下患者<9000μg/mL，2岁以上且BSA≤1.3m的患者<7000μg/mL，2岁以上且BSA>1.3m的患者<5000μg/mL），以此作为一种方法来识别那些应评估增加剂量和/或遵医嘱给药情况的患者。
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3
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