University of Maryland, Baltimore, Md., USA.
Horm Res Paediatr. 2013;80(4):242-51. doi: 10.1159/000354126. Epub 2013 Sep 10.
Several models have been developed to predict growth response to growth hormone (GH) based on auxological and biochemical parameters for children with non-GH-deficient, idiopathic short stature (ISS).
To demonstrate if an individualized, formula-based, target-driven GH regimen for children with ISS would lead to a height (Ht) gain to -1.3 SDS during the first 24 months of treatment of this 4-year study, with less variability than with standard weight-based dosing.
A 4-year, open-label, multi-center, randomized, two-arm study comparing formula-based dosing of Genotropin® GH from 0.18 to 0.7 mg/kg/week versus standard FDA-approved ISS dosing of Genotropin® (0.37 mg/kg/week). Subjects (n = 316, 89 females) were prepubertal, 3-14 years of age, bone age 3-10 years (m) and 3-9 years (f), naive to GH treatment, Ht SDS -3 to -2.25, Ht velocity <25th percentile for bone age, and peak GH >10 ng/ml.
The majority (83%) of subjects had Ht SDS within the normal range by 2 years. All subjects displayed catch-up growth consistent with other studies of GH treatment of ISS.
The formula-based therapy did not meet the primary endpoint achieving targeted gain with lower variability. No new safety concerns were found.
已经开发出了几种模型，可基于儿童非生长激素缺乏性特发性身材矮小症（ISS）的生长和生化参数来预测生长激素（GH）的生长反应。
证明对于 ISS 儿童，采用个体化、基于公式、以目标为导向的 GH 治疗方案，是否会导致在这项为期 4 年的研究的前 24 个月内身高（Ht）增长达到-1.3 SDS，且变异性小于标准体重剂量。
一项为期 4 年、开放标签、多中心、随机、双臂研究，比较基于公式的 Genotropin® GH 剂量（0.18 至 0.7 mg/kg/周）与 Genotropin® 的标准 FDA 批准的 ISS 剂量（0.37 mg/kg/周）。受试者（n = 316，女性 89 例）为青春期前，年龄 3-14 岁，骨龄 3-10 岁（男）和 3-9 岁（女），GH 治疗初治，Ht SDS -3 至-2.25，Ht 速度<骨龄第 25 百分位，和峰值 GH >10ng/ml。
大多数（83%）受试者在 2 年内 Ht SDS 达到正常范围。所有受试者均表现出与其他 GH 治疗 ISS 研究一致的追赶生长。
基于公式的治疗方案未达到主要终点，无法实现靶向增长，且变异性降低。未发现新的安全问题。
