Five-year safety and growth response of long-acting PEGylated recombinant human growth hormone in children with growth hormone deficiency-data from CGLS database

被引：0

作者：

Wu, Wei ^{[1
]}

Wei, Haiyan ^{[2
]}

Du, Hongwei ^{[3
]}

Liang, Liyang ^{[4
]}

Gong, Chunxiu ^{[5
]}

Zhong, Yan ^{[6
]}

Ma, Liyan ^{[7
]}

Luo, Feihong ^{[8
]}

Chen, Ruimin ^{[9
]}

Huang, Xiaoyan ^{[10
]}

Ye, Kan ^{[11
]}

Cui, Yunpu ^{[12
]}

Wang, Chunlin ^{[13
]}

Zhu, Min ^{[14
]}

Wang, Kundi ^{[15
]}

Zhao, Yufang ^{[16
]}

Zhang, Huiwen ^{[17
]}

Xu, Xu ^{[18
]}

Wu, Haiying ^{[19
]}

Li, Yuan ^{[20
]}

Cai, Jin ^{[21
]}

Zhang, Hongxiao ^{[22
]}

Ying, Lirong ^{[23
]}

Chen, Zhihong ^{[24
]}

Gu, Wei ^{[25
]}

Liu, Zheng ^{[26
]}

Li, Yuwen ^{[27
]}

Zheng, Rongxiu ^{[28
]}

Wan, Naijun ^{[29
]}

Li, Guimei ^{[30
]}

Luo, Jingsi ^{[31
]}

Ma, Yaping ^{[32
]}

Chen, Yuqing ^{[33
]}

Li, Nan ^{[34
,35
]}

Wang, Tingting ^{[36
]}

Luo, Xiaoping ^{[1
]}

机构：

[1] Huazhong Univ Sci & Technol, Tongji Hosp, Tongji Med Coll, Dept Pediat, Wuhan 430030, Peoples R China

[2] Zhengzhou Univ, Henan Childrens Hosp, Dept Endocrinol Genet & Metab, Childrens Hosp, Zhenzhou 450000, Peoples R China

[3] First Hosp Jilin Univ, Dept Pediat, Changchun 130021, Peoples R China

[4] Sun Yat Sen Univ, Sun Yat Sen Mem Hosp, Dept Pediat, Guangzhou 510120, Peoples R China

[5] Capital Med Univ, Beijing Childrens Hosp, Natl Ctr Childrens Hlth, Dept Endocrine & Genet & Metab, Beijing 100045, Peoples R China

[6] Hunan Childrens Hosp, Dept Child Hlth Care, Changsha 410007, Peoples R China

[7] Ningxia Med Univ, Dept Pediat, Gen Hosp, Yinchuan 750004, Ningxia, Peoples R China

[8] Fudan Univ, Childrens Hosp, Dept Pediat Endocrinol & Inherited Metab Dis, Shanghai 201102, Peoples R China

[9] Fujian Med Univ, Fuzhou Childrens Hosp, Fuzhou Gen Hosp 1, Dept Pediat Endocrinol Genet & Metab, Fuzhou 350005, Peoples R China

[10] Hainan Women & Childrens Med Ctr, Dept Genet Metab & Endocrinol, Haikou 570206, Peoples R China

[11] Nanjing Med Univ, Affiliated Suzhou Hosp, Dept Obstet, Suzhou 215002, Peoples R China

[12] Peking Univ Third Hosp, Dept Pediat, Beijing 100191, Peoples R China

[13] Zhejiang Univ, Sch Med, Dept Neurol, Affiliated Hosp 1, Hangzhou 310003, Zhejiang, Peoples R China

[14] Chongqing Med Univ, Childrens Hosp, Dept Endocrinol & Genet Metab, Chongqing 400014, Peoples R China

[15] China Japan Friendship Hosp, Dept Pediat, Beijing 100029, Peoples R China

[16] Zhejiang Univ, Jinhua Hosp, Sch Med, Dept Pediat, Jinhua 321000, Peoples R China

[17] Shanghai Jiao Tong Univ, Shanghai Inst Pediat Res, Xinhua Hosp, Dept Pediat Endocrinol & Genet Metab,Sch Med, Shanghai 200092, Peoples R China

[18] Jiangnan Univ, Dept Endocrinol, Wuxi Childrens Hosp, Affiliated Childrens Hosp, Wuxi 214023, Peoples R China

[19] Soochow Univ, Childrens Hosp, Dept Endocrinol Genet & Metab, Suzhou 215025, Peoples R China

[20] First Peoples Hosp Yunnan Prov, Dept Ophthalmol, Kunming 650032, Peoples R China

[21] Nantong Univ, Dept Pediat, Affiliated Hosp, Nantong 226001, Peoples R China

[22] Lanzhou Univ, Dept Neurosurg, Hosp 2, Lanzhou 730030, Peoples R China

[23] Cixi Peoples Hosp, Dept Pediat, Cixi 315300, Peoples R China

[24] Qingdao Univ, Dept Pediat Endocrinol Metab & Gastroenterol, Affiliated Hosp, Qingdao 266003, Peoples R China

[25] Nanjing Med Univ, Dept Endocrinol Genet & Metab, Childrens Hosp, Nanjing 210008, Peoples R China

[26] Taian Maternal & Child Hlth Care Hosp, Dept Pediat, Tai An 271000, Peoples R China

[27] Nanjing Med Univ, Dept Pediat, Affiliated Hosp 1, Nanjing 210029, Peoples R China

[28] Tianjin Med Univ, Gen Hosp, Dept Pediat, Tianjin 300052, Peoples R China

[29] Capital Med Univ, Beijing Jishuitan Hosp, Dept Pediat, Beijing, Peoples R China

[30] Shandong Univ, Shandong Prov Hosp, Dept Pediat, Jinan 250021, Shandong, Peoples R China

[31] Child Hlth Hosp Guangxi Zhuang Autonomous Reg, Genet & Metab Ctr, Maternaland, Nanning, Peoples R China

[32] Jiangnan Univ, Dept Pediat, Affiliated Hosp, Wuxi 214122, Peoples R China

[33] Anhui Childrens Hosp, Dept Endocrinol & Metab, Hefei 230022, Peoples R China

[34] Peking Univ Third Hosp, Res Ctr Clin Epidemiol, Beijing 100191, Peoples R China

[35] Peking Univ, Key Lab Epidemiol Major Dis, Minist Educ, Beijing, Peoples R China

[36] GeneSci Pharmaceut Co Ltd, Med Affairs Dept, Changchun 130012, Peoples R China

来源：

EUROPEAN JOURNAL OF PEDIATRICS | 2025年 / 184卷 / 07期

关键词：

Growth hormone; Safety; Growth response; PEG-rhGH; CGLS database; THERAPY; RESPONSIVENESS; HEIGHT;

D O I：

10.1007/s00431-025-06268-5

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

Growth hormone deficiency (GHD) is an endocrine disorder characterized by insufficient production of growth hormone (GH). PEGylated recombinant human growth hormone (PEG-rhGH; Jintrolong (R), 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 (triangle Ht SDS) was observed during the treatment period, with a mean triangle 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:center dot 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:center dot 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.

引用

页数：10

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