Williamson M L, Koch R, Azen C, Chang C
Pediatrics. 1981 Aug;68(2):161-7.
The Collaborative Study of Children Treated for Phenylketonuria was initiated in 1967. The data presented are based on findings in 132 children treated from near birth to 6 years of age. Two treatment groups were randomly formed. The target for blood phenylalanine was 1.0 to 5.4 mg/100 ml for group 1 and 5.5 to 9.9 mg/100 ml for group 2. Although mean blood phenylalanine levels for both groups initially fell within the prescribed ranges, a steady increase over time resulted in mean six year levels of 11.4 mg/100 ml and 13.0 mg/100 ml for the two groups, respectively. Because it was not possible to maintain the prescribed differences in blood phenylalanine levels between the two groups, they were combined for further analyses. The mean IQ of the total sample at age 6 years was 98 on the Stanford-Binet Intelligence Scale. Multiple regression analysis showed that, among selected treatment and psychosocial factors, the most important predictors of IQ for 6-year-old children were: (1) mothers' intellectual ability (as measured on the Wechsler Adult Intelligence Scale); (2) age at which the subjects were first treated; and (3) how well the subjects adhered to the phenylalanine-restricted diet. It was concluded that optimal early treatment will result in normal levels of intelligence at 6 years of age.
苯丙酮尿症患儿协作研究始于1967年。所呈现的数据基于132名从出生后不久至6岁接受治疗的儿童的研究结果。随机形成了两个治疗组。第一组血苯丙氨酸的目标值为1.0至5.4毫克/100毫升，第二组为5.5至9.9毫克/100毫升。尽管两组的平均血苯丙氨酸水平最初都落在规定范围内，但随着时间的推移稳步上升，两组6年时的平均水平分别为11.4毫克/100毫升和13.0毫克/100毫升。由于无法维持两组之间规定的血苯丙氨酸水平差异，因此将它们合并进行进一步分析。在斯坦福-比奈智力量表上，总样本6岁时的平均智商为98。多元回归分析表明，在选定的治疗和心理社会因素中，6岁儿童智商的最重要预测因素是：（1）母亲的智力能力（以韦氏成人智力量表衡量）；（2）受试者首次接受治疗的年龄；（3）受试者遵守苯丙氨酸限制饮食的情况。得出的结论是，最佳的早期治疗将使6岁时的智力水平正常。
