Department of Endocrinology, Bone Diseases, Genetics, and Gynaecology, Centre de Référence du Syndrome de Prader-Willi, Children's Hospital, Toulouse, France.
J Clin Endocrinol Metab. 2010 Oct;95(10):4600-8. doi: 10.1210/jc.2009-1831.
Children with Prader-Willi syndrome (PWS) are routinely treated with GH and have a response comparable with that observed in children with GH deficiency (GHD).
The objective of the study was to compare changes in serum IGF-I, IGF binding protein 3 (IGFBP-3), IGF-I to IGFBP-3 molar ratio, and growth velocity during the first 2 yr of GH therapy in PWS and GHD children.
Thirty-three children with PWS (14 boys, 4.9 ± 3.8 yr) and 591 with GHD (351 boys, 9.6 ± 3.6 yr), all naive to GH treatment, were included in this study. Serum IGF-I and IGFBP-3 were measured at 0, 6, 12, and 24 months of GH therapy. The mean initial dose of GH was 0.9 and 1 mg/m(2) · d in the PWS and GHD groups, respectively.
Mean ± SD IGF-I sdscore (SDS) and IGFBP-3 SDS were significantly higher in PWS compared with GHD. The IGF-I to IGFBP-3 molar ratio was significantly lower at baseline and subsequently not different. Despite significantly lower GH doses in PWS children at 6, 12, and 24 months (P = 0.021, P = 0.021, P = 0.001), IGF-I reached 2.8 ± 1.2 SDS at 24 months (72% of values > 2 SDS), and remained at 0.7 ± 1.6 SDS in GHD children (17% of values > 2 SDS). IGFBP-3 did not exceed 2 SDS in either group. There was no significant change in the IGF-I to IGFBP-3 molar ratio.
IGF-I SDS increases to a greater extent in PWS than GHD. Bioavailable IGF-I is apparently not different, suggesting that any possible safety issues related to elevated IGF-I are similar in both groups.
患有普拉德-威利综合征（PWS）的儿童通常接受生长激素（GH）治疗，其反应与生长激素缺乏症（GHD）儿童相似。
本研究旨在比较 PWS 和 GHD 儿童在 GH 治疗的前 2 年内血清 IGF-I、IGF 结合蛋白 3（IGFBP-3）、IGF-I 与 IGFBP-3 摩尔比以及生长速度的变化。
本研究纳入了 33 名 PWS 儿童（男 14 名，4.9 ± 3.8 岁）和 591 名 GHD 儿童（男 351 名，9.6 ± 3.6 岁），所有儿童均为 GH 治疗初治者。在 GH 治疗的 0、6、12 和 24 个月时测量血清 IGF-I 和 IGFBP-3。PWS 和 GHD 组的初始 GH 平均剂量分别为 0.9 和 1 mg/m2·d。
PWS 组的 IGF-I 标准差评分（SDS）和 IGFBP-3 SDS 均显著高于 GHD 组。IGF-I 与 IGFBP-3 摩尔比在基线时显著较低，随后无差异。尽管 PWS 儿童在 6、12 和 24 个月时的 GH 剂量显著较低（P = 0.021、P = 0.021、P = 0.001），但 IGF-I 在 24 个月时达到 2.8 ± 1.2 SDS（72%的数值> 2 SDS），而 GHD 儿童则保持在 0.7 ± 1.6 SDS（17%的数值> 2 SDS）。两组 IGFBP-3 均未超过 2 SDS。IGF-I 与 IGFBP-3 摩尔比无显著变化。
PWS 中 IGF-I SDS 的增加幅度大于 GHD。生物可利用 IGF-I 似乎没有差异，这表明与 IGF-I 升高相关的任何可能的安全性问题在两组中相似。
