Department of Public Health, School of Public Health, Hamadan University of Medical Sciences, Shaheed Fahmideh Ave., Hamadan, Iran.
Social Determinants of Health Research Center, Hamadan University of Medical Sciences, Hamadan, Iran.
Orphanet J Rare Dis. 2021 Nov 12;16(1):475. doi: 10.1186/s13023-021-02094-8.
Children with Phenylketonuria (PKU) need a special diet to avoid a variety of physical and psychological complications. The aim of this study was to compare and assess the effects of two interventions on and levels of phenylalanine and micronutrients in children with PKU.
Forty-six children with PKU (ages 1-12 years) and their caregivers were randomly assigned to one of two 10-week interventions: a caregiver educational intervention based on the Integrative Model of Behvioral Prediction (IMBP) and supplementary low-protein-modified foods. Outcomes consisted of Children's plasma phenylalanine and micronutrients (i.e., vitamin B12, vitamin D3, and zinc) and hemoglobin levels. To assess the sustainability of outcomes, we also compared the children's phenylalanine level at five-time points including baseline and 10-week, 6 months, 12 months, and 24 months follow-ups. In addition, caregivers of both groups were asked to complete a questionnaire to assess how well the interventions were implemented as well as satisfaction with interventions.
While a large percentage of children had phenylalanine levels out of range indicating non-adherence (69.6% and 81% in the educational and food items group, respectively), micronutrient deficiencies were not prevalent in the patient cohort. The levels of phenylalanine in both groups decreased significantly over time. However, conducting a repeated-measures ANOVA to evaluating the change in groups across five-time points, revealed a significant difference between groups (F = 4.68, p = 0.03). That is, the educational intervention was more effective in lowering the children's phenylalanine level. At 24-month follow-up, the percentage of children with a normal range of phenylalanine level in the educational and food items groups increased to 73.9 and 57.1 percent, respectively, from 26 and 38 percent at baseline. There were no significant changes in children's micronutrients level following the interventions, except in the hemoglobin. In this way, at 10-week follow-up, the mean hemoglobin of children in the educational group reduced significantly (P = 0.041). However, there was no significant difference between the two groups. In general, all caregivers completed the process evaluation checklist, the feedback was largely positive.
The results of this study demonstrated that both educational and providing food item interventions resulted in a significant reduction in phenylalanine levels. Empowering caregivers of patients, creating and fortifying social networks, providing favorable social supports, and providing access to special food items may be effective in controlling PKU.
Iranian Registry of Clinical Trials (IRCT20180506039548N1). Registered 6th Jun 2018, https://www.irct.ir/trial/30977 .
患有苯丙酮尿症（PKU）的儿童需要特殊饮食以避免各种身体和心理并发症。本研究旨在比较和评估两种干预措施对 PKU 儿童苯丙氨酸水平和微量营养素的影响。
46 名 PKU 儿童（年龄 1-12 岁）及其照顾者被随机分配到两种为期 10 周的干预措施之一：基于综合行为预测模型（IMBP）的照顾者教育干预和补充低蛋白改良食品。结果包括儿童血浆苯丙氨酸和微量营养素（即维生素 B12、维生素 D3 和锌）和血红蛋白水平。为了评估结果的可持续性，我们还比较了儿童在五个时间点的苯丙氨酸水平，包括基线和 10 周、6 个月、12 个月和 24 个月随访。此外，两组的照顾者都被要求完成一份问卷，以评估干预措施的实施情况以及对干预措施的满意度。
虽然很大比例的儿童苯丙氨酸水平超出了正常范围，表明不遵守饮食规定（教育组和食物组分别为 69.6%和 81%），但患者队列中并没有出现微量营养素缺乏的情况。两组的苯丙氨酸水平随着时间的推移都显著下降。然而，进行重复测量方差分析以评估五个时间点的组间变化，结果显示组间存在显著差异（F=4.68，p=0.03）。也就是说，教育干预在降低儿童苯丙氨酸水平方面更为有效。在 24 个月的随访中，教育组和食物组中苯丙氨酸水平正常的儿童比例分别从基线时的 26%和 38%增加到 73.9%和 57.1%。干预后儿童微量营养素水平没有显著变化，除了血红蛋白。在这种情况下，在 10 周随访时，教育组儿童的血红蛋白显著降低（P=0.041）。然而，两组之间没有显著差异。总的来说，所有照顾者都完成了过程评估检查表，反馈大多是积极的。
本研究结果表明，教育和提供食物干预都能显著降低苯丙氨酸水平。赋予患者照顾者权力、建立和强化社交网络、提供有利的社会支持以及提供特殊食物可能有助于控制 PKU。
伊朗临床试验注册中心（IRCT20180506039548N1）。注册于 2018 年 6 月 6 日，https://www.irct.ir/trial/30977 。
