Giovannini Marcello, Riva Enrica, Salvatici Elisabetta, Cefalo Graziella, Radaelli Giovanni
a Department of Paediatrics , San Paolo Hospital, University of Milan , Milan , ITALY.
J Am Coll Nutr. 2014;33(2):103-10. doi: 10.1080/07315724.2013.857281.
To examine whether a phenylalanine-free protein substitute with prolonged release may be beneficial to the protein status of children with phenylketonuria (PKU) compared to conventional substitutes.
Sixty children with PKU, 7 to 16 years of age, were randomly allocated to receive either a prolonged-release (test) or the current conventional protein substitute for 30 days. Subjects were additionally sex and age matched with 60 subjects with mild hyperphenylalaninemia and 60 unaffected subjects. The protein status in children with PKU was assessed by albumin, transthyretin, and retinol-binding protein (RBP), and changes throughout the trial period were the primary outcome measures.
Children with PKU did not differ in anthropometry from children with mild hyperphenylalaninemia or unaffected children but they ingested lower amounts of proteins (p < 0.01). No differences occurred throughout the trial between or within children with PKU who received the test or conventional substitute for macronutrient intake. Albumin and RBP concentrations were within the age-specific reference range for all children. The rate of protein insufficiency (transthyretin concentration less than 20 mg/dL) did not differ statistically between children receiving test or conventional substitute (recruitment 51.8% vs 53.6%; end of the trial 44.4% vs 50.0%) but mean transthyretin recovered over 20 mg/dL in children who received the test substitute, increasing from 19.1 to 20.7 mg/dL (mean change, 1.6 mg/dL; 95% confidence interval 0.4 to 2.8 mg/dL). In children receiving conventional substitute mean transthyretin changed from 19.0 to 19.2 mg/dL (0.2; -0.2 to 0.6) mg/dL.
Protein substitutes with prolonged release might be beneficial to protein status in children with phenylketonuria.
与传统替代品相比，研究一种具有缓释作用的无苯丙氨酸蛋白质替代品是否对苯丙酮尿症（PKU）患儿的蛋白质状况有益。
60名7至16岁的PKU患儿被随机分配接受缓释（试验）或当前传统蛋白质替代品30天。另外，研究对象在性别和年龄上与60名轻度高苯丙氨酸血症患儿和60名未受影响的患儿相匹配。通过白蛋白、转甲状腺素蛋白和视黄醇结合蛋白（RBP）评估PKU患儿的蛋白质状况，整个试验期间的变化是主要结局指标。
PKU患儿在人体测量学方面与轻度高苯丙氨酸血症患儿或未受影响的患儿没有差异，但他们摄入的蛋白质含量较低（p < 0.01）。在接受试验或传统替代品的PKU患儿之间或内部，整个试验期间的常量营养素摄入量没有差异。所有儿童的白蛋白和RBP浓度均在特定年龄的参考范围内。接受试验或传统替代品的儿童中，蛋白质不足率（转甲状腺素蛋白浓度低于20 mg/dL）在统计学上没有差异（招募时分别为51.8%和53.6%；试验结束时分别为44.4%和50.0%），但接受试验替代品的儿童中转甲状腺素蛋白平均恢复到超过20 mg/dL，从19.1 mg/dL增加到20.7 mg/dL（平均变化，1.6 mg/dL；95%置信区间0.4至2.8 mg/dL）。接受传统替代品的儿童中转甲状腺素蛋白平均从19.0 mg/dL变为19.2 mg/dL（0.2；-0.2至0.6）mg/dL。
具有缓释作用的蛋白质替代品可能对苯丙酮尿症患儿的蛋白质状况有益。
