Wu Ying, Zhang Lai, Ma Haipeng, Wang Peng, Lu Ming, Xv Xinle, Yu Sheng
Department of Pediatric, Linping District Maternal and Child Health Hospital, Hangzhou City, Zhejiang Province 311199, China.
Department of Pediatric, Linping District Maternal and Child Health Hospital, Hangzhou City, Zhejiang Province 311199, China.
Growth Horm IGF Res. 2025 Sep 12;82:101664. doi: 10.1016/j.ghir.2025.101664.
This study evaluates the efficacy and safety of optimized PEG-rhGH dosing in pre-pubertal and pubertal children with Childhood-Onset Growth Failure due to growth hormone deficiency (GHD) or non-GHD causes.
This study employed a combined retrospective (n = 144) and prospective (n = 14) design to examine the PEG-rhGH dosing strategies' impact. A total of 158 children were enrolled in the study, of whom 130 were included in the analysis after completing a minimum of one year of follow-up. Participants were stratified into pre-pubertal and pubertal groups. PEG-rhGH therapy with dose titration was administered based on growth response and IGF-1 level. The primary goal of the study was to evaluate the effect of individualized PEG-rhGH dosing, including clinically-based target height velocity and IGF-1 titration, on height velocity in children with GHD and non-GHD small children, stratified by puberty. Outcome measures included change in height, weight, BMI, and adverse events. Data were analyzed with SPSS 25.0.
Pre-pubertal children exhibited a significantly greater height increase compared to pubertal adolescents (9.75 cm vs. 9.01 cm, p = 0.0159). A dose-dependent effect on growth velocity was observed in both groups. In the pubertal group, growth velocity (GV) increased from 0.80 ± 0.20 cm/year at doses ≤0.200 mg/kg/week to 0.99 ± 0.38 cm/year at doses ≥0.220 mg/kg/week (p = 0.017). Similarly, in the pre-pubertal group, GV increased from 0.87 ± 0.23 cm/year at the lowest dose to 1.10 ± 0.24 cm/year at the highest dose (p = 0.048). These findings confirm a dose-response relationship, particularly at doses exceeding 0.200 mg/kg/week.
PEG-rhGH therapy was more effective in promoting height growth in pre-pubertal children compared to pubertal adolescents. A clear dose-dependent effect was observed in both groups, emphasizing the importance of individualized dosing for optimal growth outcomes.
本研究评估了优化聚乙二醇重组人生长激素（PEG-rhGH）剂量对因生长激素缺乏（GHD）或非GHD原因导致儿童期生长衰竭的青春期前和青春期儿童的疗效和安全性。
本研究采用回顾性（n = 144）和前瞻性（n = 14）相结合的设计，以检验PEG-rhGH给药策略的影响。共有158名儿童参与了该研究，其中130名在完成至少一年的随访后纳入分析。参与者被分为青春期前和青春期组。根据生长反应和胰岛素样生长因子-1（IGF-1）水平进行剂量滴定的PEG-rhGH治疗。该研究的主要目标是评估个体化PEG-rhGH剂量，包括基于临床的目标身高增长速度和IGF-1滴定，对GHD和非GHD矮小儿童身高增长速度的影响，并按青春期进行分层。观察指标包括身高、体重、体重指数（BMI）的变化以及不良事件。数据采用SPSS 25.0进行分析。
青春期前儿童的身高增长显著高于青春期青少年（9.75厘米对9.01厘米，p = 0.0159）。两组均观察到对生长速度的剂量依赖性效应。在青春期组中，生长速度（GV）从剂量≤0.200毫克/千克/周时的0.80±0.20厘米/年增加到剂量≥0.220毫克/千克/周时的0.99±0.38厘米/年（p = 0.017）。同样，在青春期前组中，GV从最低剂量时的0.87±0.23厘米/年增加到最高剂量时的1.10±0.24厘米/年（p = 0.048）。这些发现证实了剂量反应关系，特别是在剂量超过0.200毫克/千克/周时。
与青春期青少年相比，PEG-rhGH治疗在促进青春期前儿童身高增长方面更有效。两组均观察到明显的剂量依赖性效应，强调了个体化给药对实现最佳生长结果的重要性。
