长效聚乙二醇化重组人生长激素治疗生长激素缺乏症儿童的五年安全性及生长反应——来自CGLS数据库的数据
Five-year safety and growth response of long-acting PEGylated recombinant human growth hormone in children with growth hormone deficiency-data from CGLS database.
作者信息
Wu Wei, Wei Haiyan, Du Hongwei, Liang Liyang, Gong Chunxiu, Zhong Yan, Ma Liyan, Luo Feihong, Chen Ruimin, Huang Xiaoyan, Ye Kan, Cui Yunpu, Wang Chunlin, Zhu Min, Wang Kundi, Zhao Yufang, Zhang Huiwen, Xu Xu, Wu Haiying, Li Yuan, Cai Jin, Zhang Hongxiao, Ying Lirong, Chen Zhihong, Gu Wei, Liu Zheng, Li Yuwen, Zheng Rongxiu, Wan Naijun, Li Guimei, Luo Jingsi, Ma Yaping, Chen Yuqing, Li Nan, Wang Tingting, Luo Xiaoping
机构信息
Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, China.
Department of Endocrinology, Genetics and Metabolism, Henan Children's Hospital, Children's Hospital Affiliated to Zhengzhou University, Zhenzhou, 450000, China.
出版信息
Eur J Pediatr. 2025 Jun 21;184(7):434. doi: 10.1007/s00431-025-06268-5.
UNLABELLED
Growth hormone deficiency (GHD) is an endocrine disorder characterized by insufficient production of growth hormone (GH). PEGylated recombinant human growth hormone (PEG-rhGH; Jintrolong®, GeneScience Pharmaceuticals Co., Ltd.) is the only long-acting GH approved in China for treating paediatric GHD (PGHD). Long-term Efficacy and Safety Evaluation of Growth Hormone in Children in China (CGLS) is a large, surveillance registry database of participants with short stature treated with PEG-rhGH or rhGH in a real-world setting. In this study, we evaluated the safety profile and five-year growth response of PEG-rhGH based on the data from the CGLS database in participants with PGHD in China. In this real-world registry-based observational study, a total of 1,207 participants were included in the safety analysis set. Of these, 339 participants who had received PEG-rhGH continuously for five years were also included in the efficacy analysis. Key outcomes assessed comprised adverse events (AEs), serious AEs (SAEs), and height gain. The safety assessment indicated that 563 participants exhibited 1328 AEs with an incidence rate of 46.6%. Furthermore, SAEs occurred in 1.0% of participants (n = 12), with none of them associated with PEG-rhGH treatment. A significant increase in mean change in height-SD score (∆Ht SDS) was observed during the treatment period, with a mean ∆Ht SDS of 2.1 ± 0.9 in five years. Subgroup analysis showed that the younger participants exhibited a more favourable response to therapy.
CONCLUSION
CGLS data showed that five-year PEG-rhGH treatment in children with PGHD was associated with a favourable safety profile and sustained height gain.
WHAT IS KNOWN
• Several long-acting growth hormones (LAGH) have been approved for use in PGHD. PEGylated recombinant growth hormone (PEG-rhGH) is the only LAGH marketed in China, with its three-year efficacy and safety have been reported.
WHAT IS NEW
• Data from the CGLS database confirms that PEG-rhGh has an acceptable safety profile over five years, with significant improvement in height. Importantly, the data indicate that initiating the treatment earlier yields better outcomes.
未标注
生长激素缺乏症（GHD）是一种内分泌疾病，其特征是生长激素（GH）分泌不足。聚乙二醇化重组人生长激素（PEG-rhGH；金赛增®，金赛药业股份有限公司）是中国唯一获批用于治疗儿童生长激素缺乏症（PGHD）的长效生长激素。中国儿童生长激素长期疗效和安全性评估（CGLS）是一个大型监测注册数据库，记录了在现实环境中接受PEG-rhGH或rhGH治疗的身材矮小参与者的情况。在本研究中，我们基于CGLS数据库中中国PGHD参与者的数据，评估了PEG-rhGH的安全性和五年生长反应。在这项基于现实注册的观察性研究中，共有1207名参与者纳入安全性分析集。其中，339名连续接受PEG-rhGH治疗五年的参与者也纳入疗效分析。评估的主要结果包括不良事件（AE）、严重不良事件（SAE）和身高增长。安全性评估表明，563名参与者出现了1328起不良事件，发生率为46.6%。此外，1.0%的参与者（n = 12）发生了严重不良事件，且均与PEG-rhGH治疗无关。在治疗期间观察到身高标准差评分（∆Ht SDS）的平均变化显著增加，五年内平均∆Ht SDS为2.1±0.9。亚组分析表明，年龄较小的参与者对治疗的反应更有利。
结论
CGLS数据显示，PGHD儿童接受五年PEG-rhGH治疗具有良好的安全性和持续的身高增长。
已知信息
• 几种长效生长激素（LAGH）已获批用于PGHD。聚乙二醇化重组生长激素（PEG-rhGH）是中国市场上唯一的长效生长激素，其三年疗效和安全性已有报道。
新发现
• CGLS数据库的数据证实，PEG-rhGh在五年内具有可接受的安全性，身高有显著改善。重要的是，数据表明早期开始治疗效果更好。
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