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

doi:10.1007/s00431-025-06268-5

Observational Study

. 2025 Jun 21;184(7):434.

doi: 10.1007/s00431-025-06268-5.

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

Wei Wu¹, Haiyan Wei², Hongwei Du³, Liyang Liang⁴, Chunxiu Gong⁵, Yan Zhong⁶, Liyan Ma⁷, Feihong Luo⁸, Ruimin Chen⁹, Xiaoyan Huang¹⁰, Kan Ye¹¹, Yunpu Cui¹², Chunlin Wang¹³, Min Zhu¹⁴, Kundi Wang¹⁵, Yufang Zhao¹⁶, Huiwen Zhang¹⁷, Xu Xu¹⁸, Haiying Wu¹⁹, Yuan Li²⁰, Jin Cai²¹, Hongxiao Zhang²², Lirong Ying²³, Zhihong Chen²⁴, Wei Gu²⁵, Zheng Liu²⁶, Yuwen Li²⁷, Rongxiu Zheng²⁸, Naijun Wan²⁹, Guimei Li³⁰, Jingsi Luo³¹, Yaping Ma³², Yuqing Chen³³, Nan Li^{34

35}, Tingting Wang³⁶, Xiaoping Luo³⁷

Affiliations

¹ Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, China.
² Department of Endocrinology, Genetics and Metabolism, Henan Children's Hospital, Children's Hospital Affiliated to Zhengzhou University, Zhenzhou, 450000, China.
³ Department of Pediatrics, First Hospital of Jilin University, Changchun, 130021, China.
⁴ Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, 510120, China.
⁵ Department of Endocrine and Genetics and Metabolism, Beijing Children's Hospital, Capital Medical University, National Centre for Children's Health, Beijing, 100045, China.
⁶ Department of Child Health Care, Hunan Children's Hospital, Changsha, 410007, China.
⁷ Department of Pediatrics, The General Hospital of Ningxia Medical University, Ningxia, 750004, China.
⁸ Department of Pediatric Endocrinology and Inherited Metabolic Diseases, Children's Hospital of Fudan University, Shanghai, 201102, China.
⁹ Department of Pediatric Endocrinology, Genetics and Metabolism, Fuzhou Children's Hospital, Fuzhou First General Hospital Affiliated With Fujian Medical University, Fujian Medical University, Fuzhou, 350005, China.
¹⁰ Department of Genetics, Metabolism and Endocrinology, Hainan Women and Children's Medical Center, Haikou, 570206, China.
¹¹ Department of Child Health, The Affiliated Suzhou Hospital of Nanjing Medical University, Suzhou, 215002, China.
¹² Department of Pediatrics, Peking University Third Hospital, Beijing, 100191, China.
¹³ Department of Pediatrics, The First Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, 310003, China.
¹⁴ Department of Endocrinology, Genetics and Metabolism, Children'S Hospital of Chongqing Medical University, Chongqing, 400014, China.
¹⁵ Department of Pediatrics, China-Japan Friendship Hospital, Beijing, 100029, China.
¹⁶ Department of Pediatrics, Jinhua Hospital, Zhejiang University School of Medicine, Jinhua, 321000, China.
¹⁷ Department of Pediatric Endocrinology and Genetic Metabolism, School of Medicine, Xinhua Hospital, Shanghai Institute for Pediatric Research, Shanghai Jiao Tong University, Shanghai, 200092, China.
¹⁸ Department of Endocrinology, Wuxi Children'S Hospital, Affiliated Children's Hospital of Jiangnan University, Wuxi, 214023, China.
¹⁹ Depatment of Endocrinology, Genetics and Metabolism, Children's Hospital of Soochow University, Suzhou, 215025, China.
²⁰ Department of Pediatrics, The First People's Hospital of Yunnan Province, Kunming, 650032, China.
²¹ Department of Pediatrics, Affiliated Hospital of Nantong University, Nantong, 226001, China.
²² Department of Pediatrics, Second Hospital of Lanzhou University, Lanzhou, 730030, China.
²³ Department of Pediatrics, Cixi People's Hospital, Cixi, 315300, China.
²⁴ Department of Pediatric Endocrinology, Metabolism & Gastroenterology, The Affiliated Hospital of Qingdao University, Qingdao, 266003, China.
²⁵ Department of Endocrinology, Genetics and Metabolism, Children's Hospital of Nanjing Medical University, Nanjing, 210008, China.
²⁶ Department of Pediatrics, Tai'an Maternal and Child Health Care Hospital, Tai'an, 271000, China.
²⁷ Department of Pediatrics, The First Affiliated Hospital of Nanjing Medical University, Nanjing, 210029, China.
²⁸ Department of Pediatrics, Tianjin Medical University General Hospital, Tianjin, 300052, China.
²⁹ Department of Pediatrics, Beijing Jishuitan Hospital, Capital Medical University, Beijing, China.
³⁰ Department of Pediatrics, Shandong Provincial Hospital Affiliated to Shandong University, Jinan, 250021, China.
³¹ Genetic and Metabolic Center, Maternaland , Child Health Hospital of Guangxi Zhuang Autonomous Region, Nanning, China.
³² Department of Pediatrics, Affiliated Hospital of Jiangnan University, Wuxi, 214122, China.
³³ Department of Endocrinology and Metabolism, Anhui Children's Hospital, Hefei, 230022, China.
³⁴ Research Center of Clinical Epidemiology, Peking University Third Hospital, Beijing, 100191, China.
³⁵ Key Laboratory of Epidemiology of Major Diseases (Peking University), Ministry of Education, Beijing, China.
³⁶ Medical Affairs Department, GeneScience Pharmaceuticals Co., Ltd, Changchun, 130012, China.
³⁷ Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, China. xpluo@tjh.tjmu.edu.cn.

PMID: 40542826
PMCID: PMC12182472
DOI: 10.1007/s00431-025-06268-5

Observational Study

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

Wei Wu et al. Eur J Pediatr. 2025.

. 2025 Jun 21;184(7):434.

doi: 10.1007/s00431-025-06268-5.

Authors

Affiliations

¹ Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, China.
² Department of Endocrinology, Genetics and Metabolism, Henan Children's Hospital, Children's Hospital Affiliated to Zhengzhou University, Zhenzhou, 450000, China.
³ Department of Pediatrics, First Hospital of Jilin University, Changchun, 130021, China.
⁴ Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, 510120, China.
⁵ Department of Endocrine and Genetics and Metabolism, Beijing Children's Hospital, Capital Medical University, National Centre for Children's Health, Beijing, 100045, China.
⁶ Department of Child Health Care, Hunan Children's Hospital, Changsha, 410007, China.
⁷ Department of Pediatrics, The General Hospital of Ningxia Medical University, Ningxia, 750004, China.
⁸ Department of Pediatric Endocrinology and Inherited Metabolic Diseases, Children's Hospital of Fudan University, Shanghai, 201102, China.
⁹ Department of Pediatric Endocrinology, Genetics and Metabolism, Fuzhou Children's Hospital, Fuzhou First General Hospital Affiliated With Fujian Medical University, Fujian Medical University, Fuzhou, 350005, China.
¹⁰ Department of Genetics, Metabolism and Endocrinology, Hainan Women and Children's Medical Center, Haikou, 570206, China.
¹¹ Department of Child Health, The Affiliated Suzhou Hospital of Nanjing Medical University, Suzhou, 215002, China.
¹² Department of Pediatrics, Peking University Third Hospital, Beijing, 100191, China.
¹³ Department of Pediatrics, The First Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, 310003, China.
¹⁴ Department of Endocrinology, Genetics and Metabolism, Children'S Hospital of Chongqing Medical University, Chongqing, 400014, China.
¹⁵ Department of Pediatrics, China-Japan Friendship Hospital, Beijing, 100029, China.
¹⁶ Department of Pediatrics, Jinhua Hospital, Zhejiang University School of Medicine, Jinhua, 321000, China.
¹⁷ Department of Pediatric Endocrinology and Genetic Metabolism, School of Medicine, Xinhua Hospital, Shanghai Institute for Pediatric Research, Shanghai Jiao Tong University, Shanghai, 200092, China.
¹⁸ Department of Endocrinology, Wuxi Children'S Hospital, Affiliated Children's Hospital of Jiangnan University, Wuxi, 214023, China.
¹⁹ Depatment of Endocrinology, Genetics and Metabolism, Children's Hospital of Soochow University, Suzhou, 215025, China.
²⁰ Department of Pediatrics, The First People's Hospital of Yunnan Province, Kunming, 650032, China.
²¹ Department of Pediatrics, Affiliated Hospital of Nantong University, Nantong, 226001, China.
²² Department of Pediatrics, Second Hospital of Lanzhou University, Lanzhou, 730030, China.
²³ Department of Pediatrics, Cixi People's Hospital, Cixi, 315300, China.
²⁴ Department of Pediatric Endocrinology, Metabolism & Gastroenterology, The Affiliated Hospital of Qingdao University, Qingdao, 266003, China.
²⁵ Department of Endocrinology, Genetics and Metabolism, Children's Hospital of Nanjing Medical University, Nanjing, 210008, China.
²⁶ Department of Pediatrics, Tai'an Maternal and Child Health Care Hospital, Tai'an, 271000, China.
²⁷ Department of Pediatrics, The First Affiliated Hospital of Nanjing Medical University, Nanjing, 210029, China.
²⁸ Department of Pediatrics, Tianjin Medical University General Hospital, Tianjin, 300052, China.
²⁹ Department of Pediatrics, Beijing Jishuitan Hospital, Capital Medical University, Beijing, China.
³⁰ Department of Pediatrics, Shandong Provincial Hospital Affiliated to Shandong University, Jinan, 250021, China.
³¹ Genetic and Metabolic Center, Maternaland , Child Health Hospital of Guangxi Zhuang Autonomous Region, Nanning, China.
³² Department of Pediatrics, Affiliated Hospital of Jiangnan University, Wuxi, 214122, China.
³³ Department of Endocrinology and Metabolism, Anhui Children's Hospital, Hefei, 230022, China.
³⁴ Research Center of Clinical Epidemiology, Peking University Third Hospital, Beijing, 100191, China.
³⁵ Key Laboratory of Epidemiology of Major Diseases (Peking University), Ministry of Education, Beijing, China.
³⁶ Medical Affairs Department, GeneScience Pharmaceuticals Co., Ltd, Changchun, 130012, China.
³⁷ Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, China. xpluo@tjh.tjmu.edu.cn.

PMID: 40542826
PMCID: PMC12182472
DOI: 10.1007/s00431-025-06268-5

Abstract

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.

Keywords: CGLS database; Growth hormone; Growth response; PEG-rhGH; Safety.

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Conflict of interest statement

Declarations. Conflict of interest: The authors declare no competing interests.

Figures

**Fig. 1**
Frequency of AEs by system organ class. AE, adverse events; SOC, system organ class

**Fig. 2**
Growth response outcomes in participants with PGHD treated with PEG-rhGH. (A) Ht SDS (B) Height gain (∆Ht SDS) (C) height velocity (cm/year) (D) subgroup analysis of Height gain (∆Ht SDS) (E) Height gain/accumulated dose (F) IGF-1 SDS. GHD, growth hormone deficiency; Ht, height; Ht SDS, Ht standard deviation score; IGF-1, insulin-like growth factor-1; PEG-rhGH, PEGylated recombinant human growth hormone; SDS, standard deviation score

See this image and copyright information in PMC

References

1. Hage C, Gan H-W, Ibba A et al (2021) Advances in differential diagnosis and management of growth hormone deficiency in children. Nat Rev Endocrinol 17:608–624. 10.1038/s41574-021-00539-5 - PubMed
1. Chinoy A, Murray PG (2016) Diagnosis of growth hormone deficiency in the paediatric and transitional age. Best Pract Res Clin Endocrinol Metab 30:737–747. 10.1016/j.beem.2016.11.002 - PubMed
1. Bao XL, Shi YF, Du YC et al (1992) Prevalence of growth hormone deficiency of children in Beijing. Chin Med J (Engl) 105:401–405 - PubMed
1. Ranke MB, Lindberg A, Albertsson-Wikland K et al (2005) Increased Response, But Lower Responsiveness, to Growth Hormone (GH) in Very Young Children (Aged 0–3 Years) with Idiopathic GH Deficiency: Analysis of Data from KIGS. J Clin Endocrinol Metab 90:1966–1971. 10.1210/jc.2004-1051 - PubMed
1. Sävendahl L, Polak M, Backeljauw P et al (2021) Long-Term Safety of Growth Hormone Treatment in Childhood: Two Large Observational Studies: NordiNet IOS and ANSWER. J Clin Endocrinol Metab 106:1728–1741. 10.1210/clinem/dgab080 - PMC - PubMed

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[1] Hage C, Gan H-W, Ibba A et al (2021) Advances in differential diagnosis and management of growth hormone deficiency in children. Nat Rev Endocrinol 17:608–624. 10.1038/s41574-021-00539-5 - PubMed

[2] Hage C, Gan H-W, Ibba A et al (2021) Advances in differential diagnosis and management of growth hormone deficiency in children. Nat Rev Endocrinol 17:608–624. 10.1038/s41574-021-00539-5 - PubMed

[3] Chinoy A, Murray PG (2016) Diagnosis of growth hormone deficiency in the paediatric and transitional age. Best Pract Res Clin Endocrinol Metab 30:737–747. 10.1016/j.beem.2016.11.002 - PubMed

[4] Chinoy A, Murray PG (2016) Diagnosis of growth hormone deficiency in the paediatric and transitional age. Best Pract Res Clin Endocrinol Metab 30:737–747. 10.1016/j.beem.2016.11.002 - PubMed

[5] Bao XL, Shi YF, Du YC et al (1992) Prevalence of growth hormone deficiency of children in Beijing. Chin Med J (Engl) 105:401–405 - PubMed

[6] Bao XL, Shi YF, Du YC et al (1992) Prevalence of growth hormone deficiency of children in Beijing. Chin Med J (Engl) 105:401–405 - PubMed

[7] Ranke MB, Lindberg A, Albertsson-Wikland K et al (2005) Increased Response, But Lower Responsiveness, to Growth Hormone (GH) in Very Young Children (Aged 0–3 Years) with Idiopathic GH Deficiency: Analysis of Data from KIGS. J Clin Endocrinol Metab 90:1966–1971. 10.1210/jc.2004-1051 - PubMed

[8] Ranke MB, Lindberg A, Albertsson-Wikland K et al (2005) Increased Response, But Lower Responsiveness, to Growth Hormone (GH) in Very Young Children (Aged 0–3 Years) with Idiopathic GH Deficiency: Analysis of Data from KIGS. J Clin Endocrinol Metab 90:1966–1971. 10.1210/jc.2004-1051 - PubMed

[9] Sävendahl L, Polak M, Backeljauw P et al (2021) Long-Term Safety of Growth Hormone Treatment in Childhood: Two Large Observational Studies: NordiNet IOS and ANSWER. J Clin Endocrinol Metab 106:1728–1741. 10.1210/clinem/dgab080 - PMC - PubMed

[10] Sävendahl L, Polak M, Backeljauw P et al (2021) Long-Term Safety of Growth Hormone Treatment in Childhood: Two Large Observational Studies: NordiNet IOS and ANSWER. J Clin Endocrinol Metab 106:1728–1741. 10.1210/clinem/dgab080 - PMC - PubMed

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

Affiliations

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

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