Xia Wei, Wang Ting, Pan Jia-Yan
Department of Pediatrics, The First People's Hospital of Wuhu, Wuhu 241000, Anhui Province, China.
Department of Pediatric Endocrinology, The First People's Hospital of Wuhu, Wuhu 241000, Anhui Province, China.
World J Clin Cases. 2023 Oct 6;11(28):6715-6724. doi: 10.12998/wjcc.v11.i28.6715.
With the improvement of economy and living standards, the attention paid to short stature in children has been increasingly highlighted. Numerous causes can lead to short stature in children, among which growth hormone deficiency (GHD) is a significant factor.
To investigate the long-term efficacy and safety of different doses of long-acting polyethylene glycol recombinant human growth hormone (PEG-rhGH) in the treatment of GHD in children.
We selected 44 pediatric patients diagnosed with GHD who were treated at Wuhu First People's Hospital from 2014 to 2018. Total 23 patients were administered a high dose of long-acting PEG-rhGH at 0.2 mg/kg subcutaneously each week, forming the high-dose group. Meanwhile, 21 patients were given a lower dose of long-acting PEG-rhGH at 0.14 mg/kg subcutaneously each week, establishing the low-dose Group. The total treatment period was 2 years, during which we monitored the patients' height, annual growth velocity (GV), height standard deviation score (HtSDS), chronological age (CA), bone age (BA), and serum levels of insulin-like growth factor-1 (IGF-1) and insulin-like growth factor-binding protein-3 (IGFBP-3) before treatment and at 6 mo, 1 year, and 2 years after treatment initiation. We also monitored thyroid function, fasting plasma glucose, fasting insulin, and other side effects. Furthermore, we calculated the homeostatic model assessment for insulin resistance.
After 1 year of treatment, the GV, HtSDS, IGF-1, BA, and IGFBP-3 in both groups significantly improved compared to the pre-treatment levels ( < 0.05). Moreover, when comparing GV, HtSDS, IGF-1, BA, and IGFBP-3 between the two groups, there were no statistically significant differences either before or after the treatment ( > 0.05). During the treatment intervals of 0-1.0 years and 1.0-2.0 years, both patient groups experienced a slowdown in GV and a decline in HtSDS improvement ( < 0.05).
The use of PEG-rhGH in treating GHD patients was confirmed to be effective, with similar outcomes observed in both the high-dose group and low-dose groups, and no significant differences in the main side effects.
随着经济和生活水平的提高，儿童身材矮小问题日益受到关注。导致儿童身材矮小的原因众多，其中生长激素缺乏症（GHD）是一个重要因素。
探讨不同剂量长效聚乙二醇重组人生长激素（PEG-rhGH）治疗儿童GHD的长期疗效和安全性。
选取2014年至2018年在芜湖市第一人民医院接受治疗的44例确诊为GHD的儿科患者。其中23例患者每周皮下注射0.2mg/kg高剂量长效PEG-rhGH，形成高剂量组。同时，21例患者每周皮下注射0.14mg/kg低剂量长效PEG-rhGH，设立低剂量组。总治疗期为2年，在此期间，我们监测患者治疗前以及治疗开始后6个月、1年和2年时的身高、年生长速率（GV）、身高标准差积分（HtSDS）、实足年龄（CA）、骨龄（BA）以及胰岛素样生长因子-1（IGF-1）和胰岛素样生长因子结合蛋白-3（IGFBP-3）的血清水平。我们还监测甲状腺功能、空腹血糖、空腹胰岛素及其他副作用。此外，我们计算胰岛素抵抗的稳态模型评估值。
治疗1年后，两组患者的GV、HtSDS、IGF-1、BA和IGFBP-3较治疗前水平均显著改善（<0.05）。而且，比较两组患者的GV、HtSDS、IGF-1、BA和IGFBP-3，治疗前后均无统计学显著差异（>0.05）。在0 - 1.0年和1.0 - 2.0年的治疗间隔期内，两组患者的GV均放缓，HtSDS改善程度下降（<0.05）。
证实PEG-rhGH治疗GHD患者有效，高剂量组和低剂量组疗效相似，主要副作用无显著差异。
