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Long-term pegylated growth hormone for children with growth hormone deficiency: a large, prospective, real-world study

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机构: [1]Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, China [2]Department of Endocrinology, National Clinical Research Center for Child Health, The Children's Hospital, Zhejiang University School of Medicine, Hangzhou 310052, China [3]Department of Endocrine and Genetics and Metabolism, Beijing Children’s Hospital, Capital Medical University, National Centre for Children’s Health, Beijing 100045, China [4]Department of Pediatric Endocrinology and Inherited Metabolic Diseases, Children's Hospital of Fudan University, Shanghai 201102, China [5]Department of Endocrinology and Metabolism, Genetics, Henan Children's Hospital (Children's Hospital Affiliated to Zhengzhou University), Zhengzhou 450018, China [6]Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou 510120, China [7]Department of Paediatrics, First Hospital of Jilin University, Changchun 130021, China [8]Department of Pediatrics, Ningbo Women & Children′s Hospital, Ningbo 315012, China [9]Department of Child Health Care, Hunan Children's Hospital, Changsha 410007, China [10]Department of Endocrinology, Genetics and Metabolism, Fuzhou Children's Hospital of Fujian Medical University, Fuzhou 350005, China [11]Department of Pediatric Endocrine Genetics and Metabolism, Chengdu Women's and Children's Center Hospital, Chengdu 610074, China [12]Department of Pediatrics, Wuhu First People’s Hospital, Wuhu 241000, China [13]Department of Genetics and Endocrinology, The Second Affiliated Hospital &Yuying Children's Hospital of Wenzhou Medical University, Wenzhou 325000,China [14]Department of Child Health Care, Liuzhou Maternilty and Child Heulthcare Hospital, Liuzhou, Guangxi 545001, China [15]Department of Pediatrics, First Affiliated Hospital of Army Medical University (Thrid Military Medical University), Chongqing 400038, China [16]Department of Pediatrics, The Second Affiliated Hospital of Anhui Medical University, Hefei 230601, China [17]Department of Pediatrics, the First Affiliated Hospital of Guangxi Medical University,Nanning 530021, China [18]Department of Pediatrics, The Third Affiliated Hospital of Sun Yat-Sen University,Guangzhou 510630, China [19]Department of Pediatrics, Ruijin Hospital, Shanghai Jiao-Tong University, School of Medicine, Shanghai 200025, China [20]Department of Pediatrics, The First Affiliated Hospital of Sun Yat-Sen University, Guangzhou 510080, China [21]Department of Endocrinology and Genetics, Jiangxi Provincial Children′s Hospital, Affiliated Children′s Hospital of Nanchang University, Nanchang 330006, China [22]Department of Endocrinology, Children's Hospital of Chongqing Medical University,Chongqing 400016, China [23]Department of Pediatrics,The First Affiliated Hospital of Kunming Medical University, Kunming 650032, China [24]Department of Pediatrics, First Affiliated Hospital of Air Force Medical University, Xi'an 710032, China [25]Department of Pediatric Endocrinology and Genetic Metabolism, Xinhua Hospital, Shanghai Institute for Pediatric Research, School of Medicine, Shanghai Jiao Tong University, Shanghai 200092, China [26]Department of Pediatrics, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430022, China [27]Department of Endocrine and Genetic Metabolism, Maternal and Child Health-Care Hospital in Guiyang, Guiyang 550003, China [28]Department of Pediatrics, the First Affiliated Hospital of Xiamen University, Xiamen 361003, China [29]Department of Endocrinology, Wuxi Children′s Hospital, Wuxi 214023, China [30]Depatment of Endocrinology, Children’s Hospital of Soochow University, Suzhou 215025, China [31]Department of Pediatrics, Zhejiang Hospital of Traditional Chinese Medicine, Hangzhou 310000, China [32]Key Laboratory of Endocrinology of National Health Commission, Department of Endocrinology, State Key Laboratory of Complex Severe and Rare Diseases ,Peking Union Medical College Hospital, Chinese Academy of Medical Science and Peking Union Medical College, Beijing 100730, China [33]Department of Endocrinology, Shenzhen Children's Hospital, No. 7019, Yitian Road, Shenzhen 518038, China [34]Department of Endocrinology, Genetics and Metabolism, Hebei Children's Hospital, Shijiazhuang 050031, China [35]Department of Pediatrics, Zhejiang Provincial People’s Hospital, People’s Hospital of Hangzhou Medical College, Hangzhou 310014, China [36]Department of Endocrinology, Children's Hospital of Nanjing Medical University, Nanjing 210008, China [37]Department of Pediatric Endocrinology, Metabolism & Gastroenterology, The Affiliated Hospital of Qingdao University, Qingdao 266003, China [38]Department of Pediatrics,The First Affiliated Hospital, Nanjing Medical University, Nanjing 210029, China [39]Department of Pediatrics, The First Affiliated Hospital, College of Medicine, Zhejiang University, Hangzhou 310003, China [40]Department of Pediatrics, Changchun Children's Hospital, Changchun, Jilin 130000, China [41]Department of Genetics and Endocrinology, Guangzhou Women and Children′s Medical Center, Guangzhou 510623, China [42]Department of Pediatrics, General Hospital of Ningxia Medical University, Yinchuan 750004, China [43]Department of Pediatric, Peking University Third Hospital, No.49, Huayuanbei Road, Haidian District, Beijing 100191, China [44]Department of Pediatrics, China-Japan Friendship Hospital, Beijing 100029, China [45]Department of Pediatrics, West China Second Hospital, Sichuan University, Chengdu 610041, China [46]Department of Pediatrics, Shaoxing Second Hospital, Shaoxing 312000, China [47]Department of Pediatrics, Xiangya Hospital, Central South University, Changsha 410008, China [48]Department of Pediatrics, Tianjin Medical University General Hospital, Tianjin 300052, China [49]Department of Endocrinology and Metabolism, Wuhan Children's Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430015, China [50]Department of Child Health, Maternal and Child Health Care Hospital of Hainan Province,Haikou 570206, China [51]Department of Pediatrics, Hangzhou First People's Hospital, Hangzhou 310022, China [52]Department of Pediatrics, Shandong Provincial Hospital Affiliated to Shandong University,Jinan 250021, China [53]Department of Pediatrics, The 1st People's Hospital of Yunnan Province, Kunming 650032, China [54]Department of Pediatrics, Inner Mongolia People's Hospital, Hohhot Inner Mongolia 010017, China [55]Department of Pediatrics, Jinhua Hospital, Zhejiang University School of Medicine, Jinhua 321000, China [56]Department of Pediatrics, The 2nd Affiliated Hospital of Medical College of Xi'an Jiaotong University, Xi'an 710004, China [57]Department of Pediatrics, the Second Xiangya Hospital, Central South University, Changsha 410011, China [58]Department of Pediatrics, Qilu Hospital of Shandong University, Jinan 250012, China [59]Department of Pediatrics, Jiaxing First Hospital, Jiaxing 314000, China [60]Department of Pediatrics, Cixi People's Hospital, Cixi 315300, China [61]Department of Pediatrics, Affiliated Hospital of Nantong University, Nantong 226000, China [62]Department of Pediatrics, the Second Affiliated Hospital of Nanchang University, Nanchang 330006, China [63]Department of Pediatrics, Tai'an Maternal and Child Health Care Hospital, Tai'an, Shandong 271000, China [64]Department of Pediatrics, Jiangsu Province Hospital of Chinese Medicine, Nanjing 210029,China [65]Pediatric Research Institute, Qilu Children’s Hospital of Shandong University, Jinan, Shandong 250022, China [66]Department of Pediatrics, Lu'an People's Hospital, Lu'an 237000, China [67]Department of Pediatric, The First Affiliated Hospital of He'nan University of Science and Technology, Luoyang 471003, China [68]Department of Child Health , The Affiliated Suzhou Hospital of Nanjing Medical University, Suzhou 215002, China [69]Department of Pediatrics, The First People's Hospital of Changzhou, Changzhou 213000, China [70]The Second Affiliated Hospital of Guangxi Medical University, Nanning 530005, China [71]Department of Endocrinology, Children’s Hospital, Capital Institute of Pediatrics, Beijing 100020, China [72]Department of Pediatrics, Jishuitan Hospital, Beijing 100035, China [73]Department of Pediatrics, Affiliated Hospital of Jiangnan University, Wuxi 214122, China [74]Ward 1, Department of Internal Medicine, Chengdu Children's Specialized Hospital, Chengdu 610015, China [75]Department of Pediatric, Second Hospital of Lanzhou University, Lanzhou 730030, China [76]Department of Endocrinology and Genetics, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai 200127, China [77]Department of Pediatrics, Hebei General Hospital, Shijiazhuang 050051, China [78]Department of Pediatrics, the Second Hospital of Hebei Medical University, Shijiazhuang 050000, China [79]Department of Endocrinology, Shanghai Children's Hospital, Shanghai Jiao Tong University, Shanghai 200333, China [80]School of Public Health, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, China
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关键词: Jintrolong PEGylated recombinant human growth hormone long-acting growth hormone Growth hormone deficiency

摘要:
The evidence of long-term polyethylene glycol recombinant human growth hormone (PEG-rhGH) in pediatric growth hormone deficiency (GHD) is limited.This study aimed to examine the effectiveness and safety of long-term PEG-rhGH in children with GHD in real world, as well as to examine the effects of dose on patient outcomes.A prospective, observational, post-trial study (NCT03290235).Children with GHD were enrolled from 81 centers in China in four individual clinical trials, and received weekly 0.2 mg/kg/week (high-dose) or 0.1-<0.2 mg/kg/week (low-dose) PEG-rhGH for 30 months.Height standard deviation score (Ht SDS) at 12, 24, and 36 months.A total of 1170 children were enrolled in this post-trial study, with 642 patients in the high-dose subgroup and 528 in the low-dose subgroup, respectively. 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. Besides, the changes in Ht SDS from baseline was significantly improved in the high-dose subgroup than in 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 AEs. There was no serious AE related to the treatment, and no AEs leading to treatment discontinuation or death occurred.PEG-rhGH showed long-term effectiveness and safety in treating children with GHD. Both dose subgroups showed promising outcomes, while PEG-rhGH 0.2 mg/kg/week might show additional benefit.© The Author(s) 2023. Published by Oxford University Press on behalf of the Endocrine Society.

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出版当年[2023]版:
大类 | 2 区 医学
小类 | 2 区 内分泌学与代谢
最新[2023]版:
大类 | 2 区 医学
小类 | 2 区 内分泌学与代谢
第一作者:
第一作者机构: [1]Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, China
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通讯作者:
通讯机构: [1]Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, China [2]Department of Endocrinology, National Clinical Research Center for Child Health, The Children's Hospital, Zhejiang University School of Medicine, Hangzhou 310052, China [*1]Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, China [*2]Department of Endocrinology, National Clinical Research Center for Child Health, The Children's Hospital, Zhejiang University School of Medicine, Hangzhou, 310052, China.
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