MacDonald A, Lilburn M, Davies P, Evans S, Daly A, Hall S K, Hendriksz C, Chakrapani A, Lee P
Birmingham Children's Hospital, Steelhouse Lane, Birmingham, BH4 6NH, UK.
J Inherit Metab Dis. 2006 Aug;29(4):526-31. doi: 10.1007/s10545-006-0234-y. Epub 2006 May 30.
In phenylketonuria (PKU), compliance with taking protein substitute is an issue in teenage and older patients. A 'ready to drink' protein substitute may overcome many of the practical issues associated with its administration.
To investigate the efficacy of a liquid protein substitute in a 6-week, three-part, randomized, crossover, controlled study.
27 subjects (15 female; 12 male) with PKU with a median age of 30 years (range 8-49 years) were recruited. One subject withdrew from the study. Their median daily dose of protein equivalent was 60 g (range 45-75 g). In parts 1 and 2, subjects were randomized to either a liquid or a powder protein substitute with the same nutritional composition per unit (each 130 ml liquid pouch or 25 g powder sachet contained 15 g protein equivalent). In part 3, subjects chose liquid, powder or a combination of both. Weekly blood phenylalanine (Phe) concentrations were estimated, and during weeks 2, 4 and 6 subjects completed a daily questionnaire on administration issues.
All but one of 26 subjects chose the liquid in part 3 as either their sole (69%, n = 18) or partial source (28%, n = 7) of protein substitute. Blood Phe concentrations were significantly better on the liquid (p = 0.03). With the liquid protein substitute, subjects were less self-consciousness (p = 0.003) and found it easier to take away from home (p = 0.001). Overall, the liquid was easier (p < 0.0001), more convenient (p = 0.002) and resulted in less wastage of protein substitute (p = 0.001).
Liquid protein substitute was popular and efficacious, reduced self-consciousness and overall improved compliance of teenagers and adults with PKU.
在苯丙酮尿症（PKU）患者中，青少年及成年患者服用蛋白质替代物的依从性是个问题。一种“即饮型”蛋白质替代物或许能克服许多与其服用相关的实际问题。
在一项为期6周、分为三个阶段且随机、交叉、对照的研究中，调查一种液体蛋白质替代物的疗效。
招募了27名苯丙酮尿症患者（15名女性；12名男性），中位年龄为30岁（范围8 - 49岁）。一名受试者退出研究。他们每日蛋白质当量的中位剂量为60克（范围45 - 75克）。在第1和第2阶段，受试者被随机分为服用液体或粉末状蛋白质替代物，每单位营养成分相同（每130毫升液体袋或25克粉末包含有15克蛋白质当量）。在第3阶段，受试者可选择液体、粉末或两者组合。每周估算血苯丙氨酸（Phe）浓度，在第2、4和6周，受试者完成一份关于服用问题的每日问卷。
26名受试者中除一人外，其余所有人在第3阶段都选择了液体作为蛋白质替代物的唯一（69%，n = 18）或部分来源（28%，n = 7）。服用液体时血苯丙氨酸浓度显著更佳（p = 0.03）。使用液体蛋白质替代物时，受试者的自我意识降低（p = 0.003），且发现更容易携带出门（p = 0.001）。总体而言，液体更易于服用（p < 0.0001）、更方便（p = 0.002），且蛋白质替代物的浪费更少（p = 0.001）。
液体蛋白质替代物受欢迎且有效，可降低自我意识，并总体上提高苯丙酮尿症青少年和成人的依从性。
