Department of Pediatrics, University of Wisconsin, American Family Children's Hospital, 600 Highland Avenue H4-436, Madison, Wisconsin 53792, USA.
J Clin Endocrinol Metab. 2010 Mar;95(3):1131-6. doi: 10.1210/jc.2009-1389. Epub 2010 Jan 8.
Children with Prader-Willi syndrome (PWS) have decreased muscle mass, hypotonia, and impaired linear growth. Recombinant human GH (hGH) treatment reportedly improves body composition and physical function in children with PWS, but these studies lack long-term control data. To assess the impact of hGH therapy begun early in life on the natural history of PWS, we compared height, body composition, and strength in similar-age children with PWS naïve to hGH with those treated with hGH for 6 yr.
Forty-eight children with PWS were studied: 21 subjects (aged 6-9 yr) treated with hGH for 6 yr (beginning at 4-32 months, mean 13 +/- 6 months) were compared with 27 children of similar age (5-9 yr) prior to treatment with hGH. Percent body fat, lean body mass, carbohydrate/lipid metabolism, and motor strength were compared using analysis of covariance.
PWS children treated with hGH demonstrated lower body fat (mean, 36.1 +/- 2.1 vs. 44.6 +/- 1.8%, P < 0.01), greater height (131 +/- 2 vs. 114 +/- 2 cm; P < 0.001), greater motor strength [increased standing broad jump 22.9 +/- 2.1 vs. 14.6 +/- 1.9 in. (P < 0.001) and sit-ups 12.4 +/- 0.9 vs. 7.1 +/- 0.7 in 30 sec (P < 0.001)], increased high-density lipoprotein cholesterol (58.9 +/- 2.6 vs. 44.9 +/- 2.3 mg/dl, P < 0.001), decreased low-density lipoprotein (100 +/- 8 vs. 131 +/- 7 mg/dl, P < 0.01), and no difference in fasting glucose or insulin.
hGH treatment in children with PWS, begun prior to 2 yr of age, improves body composition, motor function, height, and lipid profiles. The magnitude of these effects suggests that long-term hGH therapy favorably alters the natural history of PWS to an extent that exceeds risks and justifies consideration for initiation during infancy.
患有普拉德-威利综合征（PWS）的儿童肌肉量减少、肌张力低、线性生长受损。据报道，重组人生长激素（hGH）治疗可改善 PWS 儿童的身体成分和身体功能，但这些研究缺乏长期对照数据。为了评估早期开始的 hGH 治疗对 PWS 自然史的影响，我们比较了未经 hGH 治疗的 PWS 儿童（年龄 6-9 岁）与接受 hGH 治疗 6 年的 PWS 儿童（年龄 4-32 个月，平均 13±6 个月）的身高、身体成分和力量。使用协方差分析比较体脂百分比、瘦体重、碳水化合物/脂质代谢和运动力量。
研究了 48 名 PWS 儿童：21 名（6-9 岁）接受 hGH 治疗 6 年（开始于 4-32 个月，平均 13±6 个月）的受试者与 27 名未接受 hGH 治疗的同龄儿童（5-9 岁）进行比较。使用协方差分析比较体脂百分比、瘦体重、碳水化合物/脂质代谢和运动力量。
在 2 岁之前开始对 PWS 儿童进行 hGH 治疗可改善身体成分、运动功能、身高和血脂谱。这些影响的程度表明，长期 hGH 治疗可显著改变 PWS 的自然史，其益处超过风险，值得考虑在婴儿期开始治疗。
