"Long-term Pegylated GH for Children With GH Deficiency: A Large, Prospective, Real-world Study"

作者全名："Hou, Ling; Huang, Ke; Gong, Chunxiu; Luo, Feihong; Wei, Haiyan; Liang, Liyang; Du, Hongwei; Zhang, Jianping; Zhong, Yan; Chen, Ruimin; Chen, Xinran; Pan, Jiayan; Jin, Xianjiang; Zeng, Ting; Liao, Wei; Liu, Deyun; Lan, Dan; Zhu, Shunye; Dong, Zhiya; Ma, Huamei; Yang, Yu; Xiong, Feng; Lu, Ping; Cheng, Shengquan; Gu, Xuefan; Jin, Runming; Liu, Yu; Wu, Jinzhun; Xu, Xu; Chen, Linqi; Dong, Qin; Pan, Hui; Su, Zhe; Liu, Lijun; Luo, Xiaoming; Ni, Shining; Chen, Zhihong; Hu, Yuhua; Wang, Chunlin; Liu, Jing; Liu, Li; Lu, Biao; Wang, Xinli; Wang, Yunfeng; Yang, Fan; Zhang, Manyan; Cao, Lizhi; Liu, GeLi; Yao, Hui; Zhan, Yaqin; Dai, Mingjuan; Li, Guimei; Li, Li; Liu, Yanjie; Wang, Kan; Xiao, Yanfeng; Zhang, Xingxing; Dong, Junhua; Gu, Zaiyan; Ying, Lirong; Huang, Feng; Liu, Yanling; Liu, Zheng; Ye, Jin; Zhao, Dongmei; Hu, Xu; Jiang, Zhihong; Ye, Kan; Zhu, Hong; Chen, Shaoke; Chen, Xiaobo; Wan, Naijun; Xu, Zhuangjian; Yin, Qingjin; Zhang, Hongxiao; Huang, Xiaodong; Yin, Jianying; Zhang, Huifeng; Li, Pin; Yin, Ping; Fu, Junfen; Luo, XiaoPing"

作者地址："[Hou, Ling; Luo, XiaoPing] Huazhong Univ Sci & Technol, Tongji Hosp, Tongji Med Coll, Dept Pediat, Wuhan 430030, Peoples R China; [Huang, Ke; Fu, Junfen] Zhejiang Univ, Childrens Hosp, Natl Clin Res Ctr Child Hlth, Dept Endocrinol,Sch Med, Hangzhou 310052, Peoples R China; [Gong, Chunxiu] Capital Med Univ, Beijing Childrens Hosp, Natl Ctr Childrens Hlth, Dept Endocrine & Genet & Metab, Beijing 100045, Peoples R China; [Luo, Feihong] Fudan Univ, Dept Pediat Endocrinol & Inherited Metab Dis, Childrens Hosp, Shanghai 201102, Peoples R China; [Wei, Haiyan] Zhengzhou Univ, Henan Childrens Hosp, Dept Endocrinol & Metab Genet, Childrens Hosp, Zhengzhou 450018, Peoples R China; [Liang, Liyang] Sun Yat sen Univ, Sun Yat sen Mem Hosp, Dept Pediat, Guangzhou 510120, Peoples R China; [Du, Hongwei] First Hosp Jilin Univ, Dept Paediat, Changchun 130021, Peoples R China; [Zhang, Jianping] Ningbo Women & Childrens Hosp, Dept Pediat, Ningbo 315012, Peoples R China; [Zhong, Yan] Hunan Childrens Hosp, Dept Child Hlth Care, Changsha 410007, Peoples R China; [Chen, Ruimin] Huazhong Univ Sci & Technol, Tongji Med Coll, Sch Publ Hlth, Fuzhou 430030, Peoples R China; [Chen, Xinran] Chengdu Womens & Childrens Ctr Hosp, Dept Pediat Endocrine Genet & Metab, Chengdu 610074, Peoples R China; [Pan, Jiayan] Wuhu First Peoples Hosp, Dept Pediat, Wuhu 241000, Peoples R China; [Jin, Xianjiang] Wenzhou Med Univ, Affiliated Hosp 2, Dept Genet & Endocrinol, Wenzhou 325000, Peoples R China; [Jin, Xianjiang] Yuying Childrens Hosp Wenzhou Med Univ, Wenzhou 325000, Peoples R China; [Zeng, Ting] Liuzhou Maternilty & Child Heulthcare Hosp, Dept Child Hlth Care, Liuzhou 545001, Guangxi, Peoples R China; [Liao, Wei] Army Med Univ Thrid Mil Med Univ, Dept Pediat, Affiliated Hosp 1, Chongqing 400038, Peoples R China; [Liu, Deyun] Anhui Med Univ, Dept Pediat, Affiliated Hosp 2, Hefei 230601, Peoples R China; [Lan, Dan] Guangxi Med Univ, Dept Pediat, Affiliated Hosp 1, Nanning 530021, Peoples R China; [Zhu, Shunye] Sun Yat Sen Univ, Affiliated Hosp 3, Dept Pediat, Guangzhou 510630, Peoples R China; [Dong, Zhiya] Shanghai Jiao Tong Univ, Ruijin Hosp, Sch Med, Dept Pediat, Shanghai 200025, Peoples R China; [Ma, Huamei] Sun Yat Sen Univ, Affiliated Hosp 1, Dept Pediat, Guangzhou 510080, Peoples R China; [Yang, Yu] Nanchang Univ, Jiangxi Prov Childrens Hosp, Dept Endocrinol & Genet, Affiliated Childrens Hosp, Nanchang 330006, Peoples R China; [Xiong, Feng] Chongqing Med Univ, Dept Endocrinol, Childrens Hosp, Chongqing 400016, Peoples R China; [Lu, Ping] Kunming Med Univ, Dept Pediat, Affiliated Hosp 1, Kunming 650032, Peoples R China; [Cheng, Shengquan] Air Force Med Univ, Dept Pediat, Affiliated Hosp 1, Xian 710032, Peoples R China; [Gu, Xuefan] Shanghai Jiao Tong Univ, Xinhua Hosp, Shanghai Inst Pediat Res, Sch Med,Dept Pediat Endocrinol & Genet Metab, Shanghai 200092, Peoples R China; [Jin, Runming] Huazhong Univ Sci & Technol, Union Hosp, Tongji Med Coll, Dept Pediat, Wuhan 430022, Peoples R China; [Liu, Yu] Maternal & Child Hlth Care Hosp Guiyang, Dept Endocrine & Genet Metab, Guiyang 550003, Peoples R China; [Wu, Jinzhun] Xiamen Univ, Dept Pediat, Affiliated Hosp 1, Xiamen 361003, Peoples R China; [Xu, Xu] Wuxi Childrens Hosp, Dept Endocrinol, Wuxi 214023, Peoples R China; [Chen, Linqi] Soochow Univ, Depatment Endocrinol, Childrens Hosp, Suzhou 215025, Peoples R China; [Dong, Qin] Zhejiang Hosp Tradit Chinese Med, Dept Pediat, Hangzhou 310000, Peoples R China; [Pan, Hui] Chinese Acad Med Sci & Peking Union Med Coll, Peking Union Med Coll Hosp, Dept Endocrinol, Key Lab Endocrinol,Natl Hlth Commiss,State Key Lab, Beijing 100730, Peoples R China; [Su, Zhe] Shenzhen Childrens Hosp, Dept Endocrinol, Shenzhen 518038, Peoples R China; [Liu, Lijun] Hebei Childrens Hosp, Dept Endocrinol Genet & Metab, Shijiazhuang 050031, Peoples R China; [Luo, Xiaoming] Peoples Hosp Hangzhou Med Coll, Zhejiang Prov Peoples Hosp, Dept Pediat, Hangzhou 310014, Peoples R China; [Ni, Shining] Nanjing Med Univ, Dept Endocrinol, Childrens Hosp, Nanjing 210008, Peoples R China; [Chen, Zhihong] Qingdao Univ, Dept Pediat Endocrinol Metab & Gastroenterol, Affiliated Hosp, Qingdao 266003, Peoples R China; [Hu, Yuhua] Nanjing Med Univ, Affiliated Hosp 1, Dept Pediat, Nanjing 210029, Peoples R China; [Wang, Chunlin] Zhejiang Univ, Affiliated Hosp 1, Coll Med, Dept Pediat, Hangzhou 310003, Peoples R China; [Liu, Jing] Changchun Childrens Hosp, Dept Pediat, Changchun 130000, Jilin, Peoples R China; [Liu, Li] Guangzhou Women & Childrens Med Ctr, Dept Genet & Endocrinol, Guangzhou 510623, Peoples R China; [Lu, Biao] Ningxia Med Univ, Dept Pediat, Gen Hosp, Yinchuan 750004, Peoples R China; [Wang, Xinli] Peking Univ Third Hosp, Dept Pediat, Beijing 100191, Peoples R China; [Wang, Yunfeng] China Japan Friendship Hosp, Dept Pediat, Beijing 100029, Peoples R China; [Yang, Fan] Sichuan Univ, West China Hosp 2, Dept Pediat, Chengdu 610041, Peoples R China; [Zhang, Manyan] Shaoxing Second Hosp, Dept Pediat, Shaoxing 312000, Peoples R China; [Cao, Lizhi] Cent South Univ, Xiangya Hosp, Dept Pediat, Changsha 410008, Peoples R China; [Liu, GeLi] Tianjin Med Univ, Dept Pediat, Gen Hosp, Tianjin 300052, Peoples R China; [Yao, Hui] Huazhong Univ Sci & Technol, Wuhan Childrens Hosp, Tongji Med Coll, Dept Endocrinol & Metab, Wuhan 430015, Peoples R China; [Zhan, Yaqin] Zhejiang Univ, Jinhua Hosp, Dept Pediat, Sch Med, Jinhua 321000, Peoples R China; [Dai, Mingjuan] Maternal & Child Hlth Care Hosp Hainan Prov, Dept Child Hlth, Haikou 570206, Peoples R China; [Li, Guimei] Hangzhou First Peoples Hosp, Dept Pediat, Hangzhou 310022, Peoples R China; [Li, Li] Shandong Univ, Dept Pediat, Shandong Prov Hosp, Jinan 250021, Peoples R China; [Liu, Yanjie] 1st Peoples Hosp Yunnan Prov, Dept Pediat, Kunming 650032, Peoples R China; [Wang, Kan] Inner Mongolia Peoples Hosp, Dept Pediat, Hohhot 010017, Inner Mongolia, Peoples R China; [Xiao, Yanfeng] Xi An Jiao Tong Univ, Dept Pediat, Affiliated Hosp 2, Med Coll, Xian 710004, Peoples R China; [Zhang, Xingxing] Cent South Univ, Xiangya Hosp 2, Dept Pediat, Changsha 410011, Peoples R China; [Dong, Junhua] Shandong Univ, Dept Pediat, Qilu Hosp, Jinan 250012, Peoples R China; [Gu, Zaiyan] Jiaxing First Hosp, Dept Pediat, Jiaxing 314000, Peoples R China; [Ying, Lirong] Cixi Peoples Hosp, Dept Pediat, Cixi 315300, Peoples R China; [Huang, Feng] Nantong Univ, Dept Pediat, Affiliated Hosp, Nantong 226000, Peoples R China; [Liu, Yanling] Nanchang Univ, Dept Pediat, Affiliated Hosp 2, Nanchang 330006, Peoples R China; [Liu, Zheng] Taian Maternal & Child Hlth Care Hosp, Dept Pediat, Tai An 271000, Shandong, Peoples R China; [Ye, Jin] Jiangsu Prov Hosp Chinese Med, Dept Pediat, Nanjing 210029, Peoples R China; [Zhao, Dongmei] Shandong Univ, Pediat Res Inst, Qilu Childrens Hosp, Jinan 250022, Shandong, Peoples R China; [Hu, Xu] Luan Peoples Hosp, Dept Pediat, Luan 237000, Peoples R China; [Jiang, Zhihong] Henan Univ Sci & Technol, Dept Pediat, Affiliated Hosp 1, Luoyang 471003, Peoples R China; [Ye, Kan] Nanjing Med Univ, Dept Child Hlth, Affiliated Suzhou Hosp, Suzhou 215002, Peoples R China; [Zhu, Hong] First Peoples Hosp Changzhou, Dept Pediat, Changzhou 213000, Peoples R China; [Chen, Shaoke] Guangxi Med Univ, Dept Pediat, Affiliated Hosp 2, Nanning 530005, Peoples R China; [Chen, Xiaobo] Childrens Hosp, Capital Inst Pediat, Dept Endocrinol, Beijing 100020, Peoples R China; [Wan, Naijun] Jishuitan Hosp, Dept Pediat, Beijing 100035, Peoples R China; [Xu, Zhuangjian] Jiangnan Univ, Dept Pediat, Affiliated Hosp, Wuxi 214122, Peoples R China; [Yin, Qingjin] Chengdu Childrens Specialized Hosp, Dept Internal Med, Ward 1, Chengdu 610015, Peoples R China; [Zhang, Hongxiao] Second Hosp Lanzhou Univ, Dept Pediat, Lanzhou 730030, Peoples R China; [Huang, Xiaodong] Shanghai Jiao Tong Univ, Shanghai Childrens Med Ctr, Dept Endocrinol & Genet, Sch Med, Shanghai 200127, Peoples R China; [Yin, Jianying] Hebei Gen Hosp, Dept Pediat, Shijiazhuang 050051, Peoples R China; [Zhang, Huifeng] Second Hosp Hebei Med Univ, Dept Pediat, Shijiazhuang 050000, Peoples R China; [Li, Pin] Shanghai Jiao Tong Univ, Shanghai Childrens Hosp, Dept Endocrinol, Shanghai 200333, Peoples R China"

通信作者："Luo, XP (通讯作者)，Huazhong Univ Sci & Technol, Tongji Hosp, Tongji Med Coll, Dept Pediat, Wuhan 430030, Peoples R China.; Fu, JF (通讯作者)，Zhejiang Univ, Childrens Hosp, Natl Clin Res Ctr Child Hlth, Dept Endocrinol,Sch Med, Hangzhou 310052, Peoples R China."

来源：JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

ESI学科分类：CLINICAL MEDICINE

WOS号：WOS:000941064000001

JCR分区：Q1

影响因子：5

年份：2023

卷号：　

期号：　

开始页：　

结束页：　

文献类型：Article; Early Access

关键词：Jintrolong; PEGylated recombinant human growth hormone; long-acting growth hormone; growth hormone deficiency

摘要："Context The evidence of long-term polyethylene glycol recombinant human GH (PEG-rhGH) in pediatric GH deficiency (GHD) is limited. Objective This study aimed to examine the effectiveness and safety of long-term PEG-rhGH in children with GHD in the real world, as well as to examine the effects of dose on patient outcomes. Design A prospective, observational, posttrial study (NCT03290235). Setting, participants and intervention Children with GHD were enrolled from 81 centers in China in 4 individual clinical trials and received weekly 0.2 mg/kg/wk (high-dose) or 0.1 to <0.2 mg/kg/wk (low-dose) PEG-rhGH for 30 months. Main outcomes measures Height SD score (Ht SDS) at 12, 24, and 36 months. Results A total of 1170 children were enrolled in this posttrial study, with 642 patients in the high-dose subgroup and 528 in the low-dose subgroup. 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. In addition, the changes in Ht SDS from baseline were significantly improved in the high-dose subgroup compared with 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 adverse events. There was no serious adverse event related to the treatment, and no AEs leading to treatment discontinuation or death occurred. Conclusions PEG-rhGH showed long-term effectiveness and safety in treating children with GHD. Both dose subgroups showed promising outcomes, whereas PEG-rhGH 0.2 mg/kg/wk might show additional benefit."

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