ECEESPE2025 Poster Presentations Reproductive and Developmental Endocrinology (93 abstracts)
1Beijing Childrens Hospital, Beijing, China; 2Henan Childrens Hospital Zhengzhou Childrens Hospital, Henan, China; 3The First Peoples Hospital of Wuhu, Wuhu, China; 4Jiangxi Provincial Childrens Hospital, Nanchang, China; 5Woman and Childrens Hospital of Ningbo University, Ningbo, China; 6The First Bethune Hospital of Jilin University, Jilin, China; 7Childrens Hospital of Nanjing Medical University, Nanjing, China; 8Shandong Provincial Hospital, Jinan, China; 9Tianjin Medical University General Hospital, Tianjin, China; 10Childrens Hospital of Fudan University, Shanghai, China; 11Childrens Hospital of Soochow University, Suzhou, China; 12The Second Hospital of Anhui Medical University, Hefei, China; 13Wuhan Childrens Hospital, Wuhan, China; 14Wuxi Childrens Hospital, Wuxi, China; 15Xuzhou Childrens Hospital, Xuzhou, China; 16Xinhua Hospital Affiliated to Shanghai Jiao Tong School of Medicine, Shanghai, China; 17Shanghai Childrens Medical Centre, Shanghai, China
JOINT917
Introduction: The Long-Acting Growth Hormone Database in China, a brunch of the international GloBE-Reg (https://globe-reg. net/) project, was launched in 2023. It aims to establish the first database focusing on long-acting growth hormone (LAGH) in China and even globally, providing clinical data on the long-term efficacy and safety of PEGylated recombinant human growth hormone (PEG-rhGH)in the clinical practice for children with short stature.
Method: The GloBE-Reg database of LAGH in China has three layers of datasets. The first dataset is the core dataset applicable to any short stature indication, another dataset that allows the selection of a specific therapy and diagnosis, and the third dataset includes a therapy and diagnosis-specific minimum dataset (MDS) which collects information on diagnosis, therapy, clinician reported outcomes, patient reported outcomes and adverse events.
Results: Up to January 9, 2025, 29 hospitals have completed ethical reviews and 25 have been initiated across 17 provinces and cities in China, successfully recruiting 616 patients(male: female, 368: 248). The median age at the start of treatment was 6.42 years (range 0.68-16.00), baseline height was 109.94 cm (range 60.00-152.00), height Standard Deviation Score(Ht SDS) was -2.64±0.73, and baseline weight was 19.48 kg (range 5.30-54.00), -1.4±0.80 SDS. Currently approved indications for PEG-rhGH in China include growth hormone deficiency (GHD), idiopathic short stature (ISS) and Turner syndrome (TS).A total of 569 cases (92.4%) have been recorded for indications, including GHD 373 cases (60.6%), ISS 147 cases (23.9%) and TS 49 cases (8.0%). There are 47 off-label cases, including 22 cases of (3.6%)small for gestational age (SGA), Noonan syndrome(NS) 8 cases(1.3%), Silver-Russell syndrome (SRS) 8 cases (1.3%) and 9 cases of other rare causes of short stature including mixed gonadal dysplasia, 46 XX ootestis and Wiedemann-Steiner syndrome and so on. The initial dose of PEG-rhGH for GHD/ISS/TS/SGA/NS was 0.18/0.18/0.18/0.18/0.17 mg/kg/w respectively, with a mean duration of 2.39 years (range 0.44-7.40). In the 616 cases, 244 were followed up for >6 months, and the Ht SDS increase was 0.54±0.28,199 were followed up for one year, and the Ht SDS increase was 0.88±0.44.
Conclusion: The GloBE-Reg database of LAGH in China has been active. The analysis of large amount of data will provide treatment recommendations and decision support for long-term safety and efficacy studies of PEG-rhGH.