ECEESPE2025 Poster Presentations Growth Axis and Syndromes (91 abstracts)
Long-acting pegylated growth hormone in prepubertal children with turner syndrome: a 3-year multicenter study in China
chen Jiajia 1 , Yongfen Lv 2 , Hongwei Du 3 , Yaping Ma 4 , Lianshu Han 5 , Feihong Luo 6 , Guimei Li 7 , Linqi Chen 8 & Chunxiu Gong 9
1Beijing Children’s Hospital, Capital Medical University, National Centre for Children’s Health, Department of Endocrine and Genetics and Metabolism, Beijing, China; 2Children’s Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai Children’s Hospital, Shanghai, China; 3The First Hospital of Jilin University, Changchun, China; 4Affiliated Hospital of Jiangnan University, Wuxi, China; 5Xinhua Hospital, Shanghai Institute of Pediatric Research, Shanghai Jiao Tong University School of Medicine, Shanghai, China; 6Children’s hospital of Fudan University, Shanghai, China; 7Shandong Provincial Hospital Affiliated to Shandong First Medical University, Jinan, China; 8Children’s Hospital of Soochow University, Suzhou, China; 9Beijing Children’s Hospital, Capital Medical University, National Centre for Children’s Health, Beijing, China
JOINT3638
Background: Short stature is a major feature of Turner syndrome (TS) and requires long-term growth hormone (GH) treatment. We report the results of the 3-year trial evaluating the efficacy and safety of pegylated recombinant human growth hormone (PEG-rhGH) in the treatment of children with TS.
Methods: Eleven hospitals in China conducted this 3-year multicenter, open-label, randomized, negative-controlled, phase 2 and 3 clinical trial. First-year patients were randomized to 0. 1 mg/kg/week (LD), 0. 2 mg/kg/week (HD), and an untreated control group. In the second year, the control group was directly given 0. 2 mg/kg/week of PEG-rhGH, and the other children were adjusted according to the annual growth rate of each visit and IGF-1 SDS, and the maximum single dose should not exceed 0. 4 mg/kg/week. The study reported changes in the height standard deviation score (Δ HT-SDS), while observing drug-related side effects over time. The trial has been registered with ClinicalTrials. gov, number NCT03189160.
Findings: A total of 180 patients aged 2. 9 to 16. 2 years were randomly assigned to each study group. 144 patients completed 3-year-treatment. The average ΔHT-SDS from baseline after 3-year-treatment was 0. 92, 1. 32, 0. 77, respectively in the LD, HD and control group. There were statistically significant differences between any two of the three groups (P <0. 0001 for all). There were statistically significant differences in change in IGF-1SDS and IGF-1/IGFBP-3 from baseline after 3-year-treatment between any two of the three groups (P < 0. 0001). Injection site atrophy occurred in 3 cases (1. 67%) and injection site sclerosis occurred in 1 case (0. 56%). No serious treatment-related adverse events There were no adverse events that resulted in discontinuation, dose reduction, or death. Laboratory test indicators at each visit after treatment did not show any special safety issues.
Conclusion: The study is the first a clinical trial for registration of long-acting rhGH for the treatment of short-statured children with TS. PEG-rhGH injection 0. 2 mg/kg/w can significantly improve HT-SDS of Turner syndrome, and had a good safety and tolerability.
Volume 110
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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