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作者

Jiajia Chen Yongfen Lv Hongwei Du Yaping Ma Lianshu Han Feihong Luo Guimei Li Linqi Chen Bingyan Cao Miao Qin Chang Su Liya Wei Qiao Wang Pin Li Zhuangjian Xu Rongrong Xie Xiuli Chen Haiying Wu Jing Xia Dongqing Pang Chunxiu Gong

作者单位

¹Department of Endocrine and Genetics and Metabolism, Beijing Children’s Hospital, Capital Medical University, National Centre for Children’s Health , Beijing , China ⁶Department of Pediatric Endocrinology and Inherited Metabolic Diseases, Children's hospital of Fudan University , Shanghai , China ⁵Department of Pediatric Endocrinology and Genetic Metabolism, Xinhua Hospital, Shanghai Institute of Pediatric Research, Shanghai Jiao Tong University School of Medicine , Shanghai , China ⁹Changchun GeneScience Pharmaceutical Co., Ltd. , Beijing , China ⁸Department of Endocrinology and Metabolism, Children’s Hospital of Soochow University , Suzhou , China ⁷Department of Pediatric Endocrinology, Shandong Provincial Hospital Affiliated to Shandong First Medical University , Jinan , China ²Department of Medical Genetics and Endocrinology, Children’s Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai Children’s Hospital , Shanghai , China ⁴Department of Pediatrics, Affiliated Hospital of Jiangnan University , Wuxi , China ³Department of Pediatrics, The First Hospital of Jilin University , Changchun , China

刊名

Journal of the Endocrine Society

年份

2025

卷号

Vol.9 Suppl 1

摘要

Objective: To evaluate the efficacy and safety of weekly PEGylated recombinant human growth hormone over 3-year in prepubertal children with Turner Syndrome . Methods: This multicenter, randomized, negative-controlled phase 2 study included a 52-week main phase followed by an ongoing open-label extension. Prepubertal patients with genetically confirmed TS, bone age <12 years, height≤-2.5 SD for age, and no prior GH therapy were randomized 1:1:1 across 8 centers to receive weekly subcutaneou...更多

Objective: To evaluate the efficacy and safety of weekly PEGylated recombinant human growth hormone over 3-year in prepubertal children with Turner Syndrome . Methods: This multicenter, randomized, negative-controlled phase 2 study included a 52-week main phase followed by an ongoing open-label extension. Prepubertal patients with genetically confirmed TS, bone age <12 years, height≤-2.5 SD for age, and no prior GH therapy were randomized 1:1:1 across 8 centers to receive weekly subcutaneous Jintrolong at 0.1 mg/kg, 0.2 mg/kg, or no treatment with monitoring only. Patients completing the main phase entered the open-label extension, during which all received Jintrolong . Doses were adjusted every 13 weeks based on annualized height velocity , IGF-1 SDS, and clinical guidelines. Treatment continued until near-adult height, with follow-up every 26 weeks thereafter until final height was attained. The primary endpoint was change in height SDS calculated using the LMS method from baseline at Week 52 and every 52 weeks during extension. Other growth-related parameters and safety were also evaluated. Results: A total of 177 patients were included in the main phase analysis, and 170 in the extension phase. The mean age at baseline was 7.87 years, and mean HT-SDS was -3.33 . At Week 52, mean changes in HT-SDS from baseline were -0.07 in the control group, 0.28 in the 0.1 mg/kg/week group, and 0.60 in the 0.2 mg/kg/week group. Both treatment groups showed significantly improvement compared with control , and the 0.2 mg/kg/week group improved significantly than the 0.1 mg/kg/week group . After two years of extension , mean increases in HT-SDS from baseline were 0.81 and 1.15 in the 0.1 and 0.2 mg/kg/week groups, respectively, indicating sustained benefit, particularly in patients initially received the higher dose. AHV in both Jintrolong groups peaked at Week 52 and gradually declined over time but remained above baseline at Week 104 and Week 156. During the extension, 84.1% of patients underwent dose escalation. The incidence of adverse events and treatment adherence were comparable across groups during the main phase. Most AEs during long-term treatment were mild to moderate and consistent with the known GH safety profile. Conclusion: Weekly Jintrolong at 0.1 to 0.4 mg/kg/week provided sustained and clinically meaningful growth improvements over 3 years in girls with TS. Most patients required dose escalation after the first year to achieve optimal growth velocity. This dosing range was appropriate for most patients and associated with a favorable long-term safety profile. Jintrolong offers a convenient long-acting formulation for managing short stature associated with TS.收起

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