Double blind placebo control trial of large neutral amino acids in treatment of PKU: Effect on blood phenylalanine
Double blind placebo control trial of large neutral amino acids in treatment of PKU: Effect on blood phenylalanine
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DOI：
10.1007/s10545-007-0556-4
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发表时间：
2007-04-01
影响因子：
4.2
通讯作者：
Guttler, F.
中科院分区：
文献类型：
--
作者：
Matalon, R.;Michals-Matalon, K.;Guttler, F.
大中性氨基酸 (LNAA) 已用于少数苯丙酮尿症 (PKU) 患者，目的是减少苯丙氨酸 (Phe) 流入大脑。在一项使用 LNAA 的开放标签研究中，俄罗斯、乌克兰和美国代谢治疗中心的 PKU 患者的血液 Phe 浓度出现了惊人的下降。为了验证从该试验中获得的数据，在米兰、帕多瓦和里约热内卢的另外三个代谢中心的参与下，使用 LNAA 对 PKU 患者进行了一项短期双盲安慰剂对照研究。短期试验的结果显示，血液 Phe 浓度较基线平均显着降低 39%。双盲安慰剂对照的数据令人鼓舞，为口服 LNAA 在降低 PKU 患者血液 Phe 浓度方面的作用提供了原理证明。需要进行长期研究来验证此类治疗的可接受性、有效性和安全性。
Large neutral amino acids (LNAA) have been used on a limited number of patients with phenylketonuria (PKU) with the purpose of decreasing the influx of phenylalanine (Phe) to the brain. In an open-label study using LNAA, a surprising decline of blood Phe concentration was found in patients with PKU in metabolic treatment centres in Russia, the Ukraine, and the United States. To validate the data obtained from this trial, a short-term double-blind placebo control study was done using LNAA in patients with PKU, with the participation of three additional metabolic centres Milan, Padua and Rio de Janeiro. The results of the short trial showed significant lowering of blood Phe concentration by an average of 39% from baseline. The data from the double-blind placebo control are encouraging, establishing proof of principle of the role of orally administered LNAA in lowering blood Phe concentrations in patients with PKU. Long-term studies will be needed to validate the acceptability, efficacy and safety of such treatment.