Liang Yan, Wei Haiyan, Yang Fan, Zhang Hua, Chen Linqi, Yao Hui, Luo Xiaoming, Cheng Xinran, Yang Yu, Lian Qun, Du Hongwei, Li Tang, Li Pin, Zhang Gaixiu, Song Fuying, Liang Liyang, Liu Deyun, Zhu Shunye, Gong Haihong, Gong Chunxiu, Cheng Xiangao, Xu Zhuangjian, Ma Yaping, Su Zhe, Zhou Weidong, He Ruoyi, Yin Yalin, Sun Li, Luo Xiaoping
Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, China.
Department of Endocrinology, Genetics and Metabolism, Children's Hospital Affiliated to Zhengzhou University, Henan Children's Hospital, Zhengzhou Children's Hospital, Zhengzhou 450018, China.
J Clin Endocrinol Metab. 2025 Jul 15;110(8):e2605-e2613. doi: 10.1210/clinem/dgae816.
Pegpesen is a novel Y-shape pegylated recombinant human growth hormone (rhGH) for once-weekly treatment of children with growth hormone deficiency (GHD).
This work aimed to evaluate the efficacy and safety of Pegpesen in children with GHD vs daily rhGH.
A multicenter, randomized, controlled phase 3 clinical trial was conducted at 23 centers in China with a duration of 52 weeks' treatment. There were 391 pediatric participants diagnosed with GHD. Participants were randomly assigned 2:1 to a weekly Pegpesen group (140 μg/kg/week) or a daily rhGH group (245 μg/kg/week) for 52 weeks. The primary end point was the growth velocity (GV) at 52 weeks, and the secondary end points mainly involved changes from baseline in height SD scores for chronological age and bone age (ΔHt SDS CA and ΔHt SDS BA).
At 52 weeks, the least squares mean (LS means) of GV was 9.910 cm/year in the Pegpesen group and 10.037 cm/year in the daily rhGH group. The LS means difference between groups was -0.127 (95% CI, -0.4868 to 0.2332), confirming that weekly Pegpesen is noninferior to daily rhGH. The LS means of ΔHt SDS CA, ΔHt SDS BA, were similar across both groups (all P > .05). Safety profiles and adherence were comparable.
Pegpesen was noninferior to daily rhGH, with similar safety, lower dosage requirements, thus presenting a new therapeutic option for children with GHD.
培戈培塞是一种新型的Y型聚乙二醇化重组人生长激素（rhGH），用于每周一次治疗生长激素缺乏症（GHD）儿童。
本研究旨在评估培戈培塞与每日注射rhGH相比，在GHD儿童中的疗效和安全性。
在中国23个中心进行了一项多中心、随机、对照的3期临床试验，治疗期为52周。共有391名被诊断为GHD的儿科参与者。参与者按2:1随机分配至每周一次培戈培塞组（140μg/kg/周）或每日注射rhGH组（245μg/kg/周），治疗52周。主要终点是52周时的生长速度（GV），次要终点主要包括按实际年龄和骨龄计算的身高标准差评分相对于基线的变化（ΔHt SDS CA和ΔHt SDS BA）。
在52周时，培戈培塞组GV的最小二乘均值（LS均值）为9.910cm/年，每日注射rhGH组为10.037cm/年。两组间的LS均值差异为-0.127（95%CI，-0.4868至0.2332），证实每周一次的培戈培塞不劣于每日注射rhGH。两组间ΔHt SDS CA和ΔHt SDS BA的LS均值相似（所有P>.05）。安全性和依从性相当。
培戈培塞不劣于每日注射rhGH，安全性相似，剂量需求更低，从而为GHD儿童提供了一种新的治疗选择。
