Kriström Berit, Aronson A Stefan, Dahlgren Jovanna, Gustafsson Jan, Halldin Maria, Ivarsson Sten A, Nilsson Nils-Osten, Svensson Johan, Tuvemo Torsten, Albertsson-Wikland Kerstin
Göteborg Pediatric Growth Research Center, Department of Pediatrics, Institute of Clinical Science, The Sahlgrenska Academy at University of Gothenburg, Göteborg, Sweden.
J Clin Endocrinol Metab. 2009 Feb;94(2):483-90. doi: 10.1210/jc.2008-1503. Epub 2008 Nov 11.
Weight-based GH dosing results in a wide variation in growth response in children with GH deficiency (GHD) or idiopathic short stature (ISS).
The hypothesis tested was whether individualized GH doses, based on variation in GH responsiveness estimated by a prediction model, reduced variability in growth response around a set height target compared with a standardized weight-based dose.
A total of 153 short prepubertal children diagnosed with isolated GHD or ISS (n = 43) and at least 1 SD score (SDS) below midparental height SDS (MPH(SDS)) were included in this 2-yr multicenter study.
The children were randomized to either a standard (43 microg/kg.d) or individualized (17-100 microg/kg.d) GH dose.
We measured the deviation of height(SDS) from individual MPH(SDS) (diffMPH(SDS)). The primary endpoint was the difference in the range of diffMPH(SDS) between the two groups.
The diffMPH(SDS) range was reduced by 32% in the individualized-dose group relative to the standard-dose group (P < 0.003), whereas the mean diffMPH(SDS) was equal: -0.42 +/- 0.46 and -0.48 +/- 0.67, respectively. Gain in height(SDS) 0-2 yr was equal for the GH-deficient and ISS groups: 1.31 +/- 0.47 and 1.36 +/- 0.47, respectively, when ISS was classified on the basis of maximum GH peak on the arginine-insulin tolerance test or 24-h profile.
Individualized GH doses during catch-up growth significantly reduce the proportion of unexpectedly good and poor responders around a predefined individual growth target and result in equal growth responses in children with GHD and ISS.
基于体重的生长激素（GH）给药导致生长激素缺乏症（GHD）或特发性矮小（ISS）儿童的生长反应差异很大。
检验的假设是，与基于体重的标准化剂量相比，基于预测模型估计的GH反应性变化的个体化GH剂量是否能减少围绕设定身高目标的生长反应变异性。
本为期2年的多中心研究纳入了153名青春期前矮小儿童，他们被诊断为孤立性GHD或ISS（n = 43），且身高低于父母平均身高标准差（MPH(SDS)）至少1个标准差分数（SDS）。
将儿童随机分为标准（43微克/千克·天）或个体化（17 - 100微克/千克·天）GH剂量组。
我们测量了身高（SDS）与个体MPH(SDS)的偏差（diffMPH(SDS)）。主要终点是两组之间diffMPH(SDS)范围的差异。
个体化剂量组的diffMPH(SDS)范围相对于标准剂量组减少了32%（P < 0.003），而平均diffMPH(SDS)相等：分别为-0.42 ± 0.46和-0.48 ± 0.67。当根据精氨酸 - 胰岛素耐量试验或24小时曲线的最大GH峰值对ISS进行分类时，GHD组和ISS组0 - 2年的身高（SDS）增长相等：分别为1.31 ± 0.47和1.36 ± 0.47。
追赶生长期间的个体化GH剂量显著降低了围绕预定义个体生长目标的意外良好和不良反应者的比例，并导致GHD和ISS儿童的生长反应相等。
