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. 2023 Jul 14;108(8):2078-2086.
doi: 10.1210/clinem/dgad039.

Long-term Pegylated GH for Children With GH Deficiency: A Large, Prospective, Real-world Study

Ling Hou  1 Ke Huang  2 Chunxiu Gong  3 Feihong Luo  4 Haiyan Wei  5 Liyang Liang  6 Hongwei Du  7 Jianping Zhang  8 Yan Zhong  9 Ruimin Chen  10 Xinran Chen  11 Jiayan Pan  12 Xianjiang Jin  13 Ting Zeng  14 Wei Liao  15 Deyun Liu  16 Dan Lan  17 Shunye Zhu  18 Zhiya Dong  19 Huamei Ma  20 Yu Yang  21 Feng Xiong  22 Ping Lu  23 Shengquan Cheng  24 Xuefan Gu  25 Runming Jin  26 Yu Liu  27 Jinzhun Wu  28 Xu Xu  29 Linqi Chen  30 Qin Dong  31 Hui Pan  32 Zhe Su  33 Lijun Liu  34 Xiaoming Luo  35 Shining Ni  36 Zhihong Chen  37 Yuhua Hu  38 Chunlin Wang  39 Jing Liu  40 Li Liu  41 Biao Lu  42 Xinli Wang  43 Yunfeng Wang  44 Fan Yang  45 Manyan Zhang  46 Lizhi Cao  47 GeLi Liu  48 Hui Yao  49 Yaqin Zhan  50 Mingjuan Dai  51 Guimei Li  52 Li Li  53 Yanjie Liu  54 Kan Wang  55 Yanfeng Xiao  56 Xingxing Zhang  57 Junhua Dong  58 Zaiyan Gu  59 Lirong Ying  60 Feng Huang  61 Yanling Liu  62 Zheng Liu  63 Jin Ye  64 Dongmei Zhao  65 Xu Hu  66 Zhihong Jiang  67 Kan Ye  68 Hong Zhu  69 Shaoke Chen  70 Xiaobo Chen  71 Naijun Wan  72 Zhuangjian Xu  73 Qingjin Yin  74 Hongxiao Zhang  75 Xiaodong Huang  76 Jianying Yin  77 Huifeng Zhang  78 Pin Li  79 Ping Yin  80 Junfen Fu  2 XiaoPing Luo  1
Affiliations

Long-term Pegylated GH for Children With GH Deficiency: A Large, Prospective, Real-world Study

Ling Hou et al. J Clin Endocrinol Metab. .

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.

Keywords: Jintrolong; PEGylated recombinant human growth hormone; growth hormone deficiency; long-acting growth hormone.

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Conflict of interest statement

The corresponding author obtained the relevant information from all co-authors. The authors declare that there is no conflict of interest.

Figures

Figure 1.
Figure 1.
Flow chart of the post-trial study. PEG-rhGH, polyethylene glycol recombinant human GH.
Figure 2.
Figure 2.
The average dose of PEG-rhGH in the 2 dose subgroups. PEG-rhGH, polyethylene glycol recombinant human GH.
Figure 3.
Figure 3.
Changes in Ht SDS from baseline in the total population (A) and between the 2 dose subgroups (B). Data are presented as mean ± SEM (*P < 0.05; **P < 0.01; ***P < 0.01). Ht SDS, height SD score; SEM, standard error of mean.
Figure 4.
Figure 4.
Changes in HV from baseline in the total population (A) and between the 2 dose subgroups (B). Data are presented as mean ± SEM (*P < 0.05). HV, height velocity; SEM, standard error of mean.
Figure 5.
Figure 5.
Mean IGF1 SDS over time in the total population (A) and between the 2 dose subgroups (B). Data are presented as mean ± SEM (**P < 0.01). SDS, SD score; SEM, standard error of mean.

References

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