Long-term Pegylated GH for Children With GH Deficiency: A Large, Prospective, Real-world Study
Ling Hou, Ke Huang, Chunxiu Gong, Feihong Luo, Haiyan Wei, Liyang Liang, Hongwei Du, Jianping Zhang, Yan Zhong, Ruimin Chen, Xinran Chen, Jiayan Pan, Xianjiang Jin, Ting Zeng, Wei Liao, Deyun Liu, Dan Lan, Shunye Zhu, Zhiya Dong, Huamei Ma, Yu Yang, Feng Xiong, Ping Lu, Sheng-Quan Cheng, Xuefan Gu, Runming Jin, Yu Liu, Jinzhun Wu, Xu Xu, Linqi Chen, Qin Dong, Hui Pan, Zhe Su, Lijun Liu, Xiaoming Luo, Xiaoming Luo, Shining Ni, Zhihong Chen, Yuhua Hu, Chunlin Wang, Jing Liu, Li Liu, Biao Lü, Xinli Wang, Yunfeng Wang, Fan Yang, Manyan Zhang, Lizhi Cao, Geli Liu, Hui Yao, Yaqin Zhan, Mingjuan Dai, Guimei Li, Li Li, Yanjie Liu, Kan Wang, Yanfeng Xiao, Xingxing Zhang, Junhua Dong, Zaiyan Gu, Lirong Ying, Feng Huang, Yanling Liu, Zheng Liu, Ye Jin, Dongmei Zhao, Xu Hu, Zhi‐Hong Jiang, Kan Ye, Hong Zhu, Shaoke Chen, Xiaobo Chen, Naijun Wan, Zhuangjian Xu, Qingjin Yin, Hongxiao Zhang, Xiaodong Huang, Jianying Yin, Huifeng Zhang, Pin Li, Ping Yin, Junfen Fu, XiaoPing Luo, XiaoPing Luo
Abstract
CONTEXT: The evidence of long-term polyethylene glycol recombinant human GH (PEG-rhGH) in pediatric GH deficiency (GHD) is limited. OBJECTIVE: This study aimed to examine the effectiveness and safety of long-term PEG-rhGH in children with GHD in the real world, as well as to examine the effects of dose on patient outcomes. DESIGN: A prospective, observational, posttrial study (NCT03290235). SETTING, PARTICIPANTS AND INTERVENTION: Children with GHD were enrolled from 81 centers in China in 4 individual clinical trials and received weekly 0.2 mg/kg/wk (high-dose) or 0.1 to <0.2 mg/kg/wk (low-dose) PEG-rhGH for 30 months. MAIN OUTCOMES MEASURES: Height SD score (Ht SDS) at 12, 24, and 36 months. RESULTS: A total of 1170 children were enrolled in this posttrial study, with 642 patients in the high-dose subgroup and 528 in the low-dose subgroup. The Ht SDS improved significantly after treatment in the total population (P < 0.0001), with a mean change of 0.53 ± 0.30, 0.89 ± 0.48, 1.35 ± 0.63, 1.63 ± 0.75 at 6 months, 12 months, 24 months, and 36 months, respectively. In addition, the changes in Ht SDS from baseline were significantly improved in the high-dose subgroup compared with the low-dose subgroup at 6, 12, 24, and 36 months after treatment (all P < 0.05). A total of 12 (1.03%) patients developed serious adverse events. There was no serious adverse event related to the treatment, and no AEs leading to treatment discontinuation or death occurred. CONCLUSIONS: PEG-rhGH showed long-term effectiveness and safety in treating children with GHD. Both dose subgroups showed promising outcomes, whereas PEG-rhGH 0.2 mg/kg/wk might show additional benefit.