Dutch Growth Research Foundation/Erasmus MC Rotterdam, Westzeedijk 106, 3016 AH Rotterdam, The Netherlands.
J Clin Endocrinol Metab. 2012 Jul;97(7):2307-14. doi: 10.1210/jc.2012-1182. Epub 2012 Apr 16.
Knowledge about the effects of GH treatment on cognitive functioning in children with Prader-Willi syndrome (PWS) is limited.
Fifty prepubertal children aged 3.5 to 14 yr were studied in a randomized controlled GH trial during 2 yr, followed by a longitudinal study during 4 yr of GH treatment. Cognitive functioning was measured biennially by short forms of the WPPSI-R or WISC-R, depending on age. Total IQ (TIQ) score was estimated based on two subtest scores.
During the randomized controlled trial, mean sd scores of all subtests and mean TIQ score remained similar compared to baseline in GH-treated children with PWS, whereas in untreated controls mean subtest sd scores and mean TIQ score decreased and became lower compared to baseline. This decline was significant for the Similarities (P = 0.04) and Vocabulary (P = 0.03) subtests. After 4 yr of GH treatment, mean sd scores on the Similarities and Block design subtests were significantly higher than at baseline (P = 0.01 and P = 0.03, respectively), and scores on Vocabulary and TIQ remained similar compared to baseline. At baseline, children with a maternal uniparental disomy had a significantly lower score on the Block design subtest (P = 0.01) but a larger increment on this subtest during 4 yr of GH treatment than children with a deletion. Lower baseline scores correlated significantly with higher increases in Similarities (P = 0.04) and Block design (P < 0.0001) sd scores.
Our study shows that GH treatment prevents deterioration of certain cognitive skills in children with PWS on the short term and significantly improves abstract reasoning and visuospatial skills during 4 yr of GH treatment. Furthermore, children with a greater deficit had more benefit from GH treatment.
关于生长激素（GH）治疗对 Prader-Willi 综合征（PWS）患儿认知功能影响的知识有限。
50 名 3.5 至 14 岁的青春期前儿童参加了一项为期 2 年的随机对照 GH 试验，随后进行了 4 年的 GH 治疗纵向研究。认知功能通过 WPPSI-R 或 WISC-R 的简短形式每两年测量一次，具体取决于年龄。总智商（TIQ）得分基于两个子测试得分进行估算。
在随机对照试验中，与基线相比，接受 GH 治疗的 PWS 患儿所有子测试的平均标准差（sd）评分和平均 TIQ 评分保持相似，而未接受治疗的对照组中，平均子测试 sd 评分和平均 TIQ 评分下降，与基线相比变得更低。这种下降在相似性（P = 0.04）和词汇（P = 0.03）子测试中具有统计学意义。经过 4 年的 GH 治疗后，相似性和积木设计子测试的平均 sd 评分明显高于基线（P = 0.01 和 P = 0.03），词汇和 TIQ 评分与基线相比保持相似。在基线时，母源单亲二体性的儿童在积木设计子测试中的得分明显较低（P = 0.01），但在 4 年 GH 治疗期间的增长幅度大于缺失的儿童。较低的基线评分与相似性（P = 0.04）和积木设计（P < 0.0001）子测试中更高的增量显著相关。
我们的研究表明，GH 治疗可在短期内预防 PWS 儿童某些认知技能的恶化，并在 4 年 GH 治疗期间显著改善抽象推理和视觉空间技能。此外，具有更大缺陷的儿童从 GH 治疗中获益更多。
