Short-term efficacy and safety of a lower dose of polyethylene glycol recombinant human growth hormone in children with growth hormone deficiency: A randomized, dose-comparison study

Zhouhong Jiang; Xuefeng Chen; Guanping Dong; Yin Lou; Jianping Zhang; Xinran Cheng; Jiayan Pan; Wei Liao; Jinzhun Wu; Xiaodong Huang; Xianjiang Jin; Deyun Liu; Ting Zeng; Shunye Zhu; Qin Dong; Xiaoming Luo; Dan Lan; Lizhi Cao; Xingxing Zhang; Jing Liu; Mingjuan Dai; Manyan Zhang; Li Liu; Junhua Dong; Dongmei Zhao; Shaoqing Ni; Junfen Fu

doi:10.3389/fphar.2022.955809

Short-term efficacy and safety of a lower dose of polyethylene glycol recombinant human growth hormone in children with growth hormone deficiency: A randomized, dose-comparison study

Front Pharmacol. 2022 Aug 11:13:955809. doi: 10.3389/fphar.2022.955809. eCollection 2022.

Authors

Zhouhong Jiang¹, Xuefeng Chen², Guanping Dong², Yin Lou¹, Jianping Zhang³, Xinran Cheng⁴, Jiayan Pan⁵, Wei Liao⁶, Jinzhun Wu⁷, Xiaodong Huang⁸, Xianjiang Jin⁹, Deyun Liu¹⁰, Ting Zeng¹¹, Shunye Zhu¹², Qin Dong¹³, Xiaoming Luo¹⁴, Dan Lan¹⁵, Lizhi Cao¹⁶, Xingxing Zhang¹⁷, Jing Liu¹⁸, Mingjuan Dai¹⁹, Manyan Zhang²⁰, Li Liu²¹, Junhua Dong²², Dongmei Zhao²³, Shaoqing Ni^{24

25}, Junfen Fu^{2

26}

Affiliations

¹ Department of Pharmacy, The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China.
² Department of Endocrinology, The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China.
³ Department of Pediatrics, Ningbo Women's and Children's Hospital, Ningbo, China.
⁴ Department of Pediatric Endocrine Genetics and Metabolism, Chengdu Women's and Children's Center Hospital, Chengdu, China.
⁵ Department of Pediatrics, Wuhu First People's Hospital, Wuhu, China.
⁶ Department of Pediatrics, First Affiliated Hospital of Army Medical University (Third Military Medical University), Chongqing, China.
⁷ Department of Pediatrics, The First Affiliated Hospital of Xiamen University, Xiamen, China.
⁸ Department of Endocrinology and Genetics, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, China.
⁹ Department of Genetics and Endocrinology, The Second Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University, Wenzhou, China.
¹⁰ Department of Pediatrics, The Second Affiliated Hospital of Anhui Medical University, Hefei, China.
¹¹ Department of Child Health Care, Liuzhou Maternity and Child Healthcare Hospital, Liuzhou, China.
¹² Department of Pediatrics, The Third Affiliated Hospital of Sun Yat-Sen University, Guangzhou, China.
¹³ Department of Pediatrics, Zhejiang Provincial Hospital of Chinese Medicine, Hangzhou, China.
¹⁴ Department of Pediatrics, Zhejiang Provincial People's Hospital, People's Hospital of Hangzhou Medical College, Hangzhou, China.
¹⁵ Department of Pediatrics, The First Affiliated Hospital of Guangxi Medical University, Nanning, China.
¹⁶ Department of Pediatrics, Xiangya Hospital, Central South University, Changsha, China.
¹⁷ Department of Pediatrics, The Second Xiangya Hospital, Central South University, Changsha, China.
¹⁸ Department of Pediatrics, Changchun Children's Hospital, Changchun, China.
¹⁹ Department of Pediatrics, Hangzhou First People's Hospital, Hangzhou, China.
²⁰ Department of Pediatrics, Shaoxing Second Hospital, Shaoxing, China.
²¹ Department of Genetics and Endocrinology, Guangzhou Women and Children's Medical Center, Guangzhou, China.
²² Department of Pediatrics, Qilu Hospital of Shandong University, Jinan, China.
²³ Pediatric Research Institute, Qilu Children's Hospital of Shandong University, Jinan, China.
²⁴ National Clinical Trial Institute, The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China.
²⁵ Research Center for Clinical Pharmacy, Zhejiang University, Hangzhou, China.
²⁶ The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China.

Abstract

Objective: Polyethylene glycol recombinant human growth hormone (PEG-rhGH, Jintrolong^®) is the first long-acting rhGH preparation that is approved to treat children with growth hormone deficiency (GHD) in China. Clinical experience with dose selections of PEG-rhGH is scarce. The present study compared the efficacy and safety of a lower dose to increase dosing regimens of PEG-rhGH treatment. Methods: A multicenter, randomized, open-label, dose-comparison clinical study was conducted to compare the improvements in the height standard deviation score (Ht SDS), height velocity (HV), insulin-like growth factor-1 (IGF-1) SDS, and safety profiles of children with GHD who are treated with 0.2 mg/kg/week of PEG-rhGH dose or 0.14 mg/kg/week for 26 weeks. Results: Ht SDS, HV, and IGF-1 SDS increased significantly after PEG-rhGH treatment in the two dose groups (p < 0.05). The improvements of Ht SDS, HV, and IGF-1 SDS were more significant in the high-dose group than in the low-dose group (p < 0.05). Ht SDS improvement in low-dose group was not non-inferiority to that in the high-dose group (p = 0.2987). The incidences of adverse events were comparable between the two groups. Conclusion: The improvements of Ht SDS, HV, and IGF-1 SDS were more significant in the high-dose group than in the low-dose group (p < 0.05). PEG-rhGH at the dose of 0.14 mg/kg/week was effective and safe for children with GHD. Clinical Trial Registration: clinicaltrials.gov, identifier NCT02908958.

Keywords: GHD; IGF-1; PEG-rhGH; children; dose.

Associated data

ClinicalTrials.gov/NCT02908958