Department of Pediatrics, Leiden University Medical Center, NL-2300 Leiden, The Netherlands.
Horm Res Paediatr. 2013;79(5):257-70. doi: 10.1159/000351025. Epub 2013 May 28.
The goal of growth hormone (GH) treatment in a short child is to attain a fast catch-up growth toward the target height (TH) standard deviation score (SDS), followed by a maintenance phase, a proper pubertal height gain, and an adult height close to TH. The short-term response variable of GH treatment, first-year height velocity (HV) (cm/year or change in height SDS), can either be compared with GH response charts for diagnosis, age and gender, or with predicted HV based on prediction models. Three types of prediction models have been described: the Kabi International Growth Hormone Study models, the Gothenburg models and the Cologne model. With these models, 50-80% of the variance could be explained. When used prospectively, individualized dosing reduces the variation in growth response in comparison with a fixed dose per body weight. Insulin-like growth factor-I-based dose titration also led to a decrease in the variation. It is uncertain whether adding biochemical, genetic or proteomic markers may improve the accuracy of the prediction. Prediction models may lead to a more evidence-based approach to determine the GH dose regimen and may reduce the drug costs for GH treatment. There is a need for user-friendly software programs to make prediction models easily available in the clinic.
生长激素（GH）治疗矮小儿童的目标是实现快速追赶生长，达到目标身高（TH）标准偏差评分（SDS），然后进入维持阶段，实现适当的青春期身高增长，并接近成人身高。GH 治疗的短期反应变量，即第一年身高速度（HV）（厘米/年或身高 SDS 的变化），可以与 GH 反应图表进行比较，用于诊断、年龄和性别，或与基于预测模型的预测 HV 进行比较。已经描述了三种类型的预测模型：Kabi 国际生长激素研究模型、哥德堡模型和科隆模型。这些模型可以解释 50-80%的变异。当前瞻性使用时，与按体重固定剂量相比，个体化剂量可减少生长反应的变异。基于胰岛素样生长因子-I 的剂量滴定也导致变异减少。添加生化、遗传或蛋白质组学标志物是否能提高预测的准确性还不确定。预测模型可能会使确定 GH 剂量方案更具循证依据，并可能降低 GH 治疗的药物成本。需要用户友好的软件程序，以便在临床中方便地使用预测模型。
