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Polyethylene glycol recombinant human growth hormone in Chinese prepubertal slow-growing short children: doses reported in a multicenter real-world study

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单位: [1]Capital Med Univ, Dept Endocrine & Genet & Metab, Beijing Childrens Hosp, Natl Ctr Childrens Hlth, 56 Nanlishi Rd, Beijing 100045, Peoples R China [2]Hunan Childrens Hosp, Dept Child Hlth Care, Changsha 410007, Peoples R China [3]Zhengzhou Univ, Dept Endocrinol & Metab, Genet, Henan Childrens Hosp,Childrens Hosp, Changsha 450018, Peoples R China [4]Guangxi Med Univ, Dept Genet & Metab, Affiliated Hosp 2, Nanning 530005, Peoples R China [5]Shenzhen Childrens Hosp, Dept Endocrinol, Shenzhen 518038, Peoples R China [6]Hebei Childrens Hosp, Dept Endocrinol Genet & Metab, Shijiazhuang 050031, Hebei, Peoples R China [7]Sun Yat Sen Univ, Dept Pediat, Sun Yat Sen Mem Hosp, Guangzhou 510120, Peoples R China [8]Kunming Med Univ, Dept Pediat, Affiliated Hosp 1, Kunming 650032, Yunnan, Peoples R China [9]Soochow Univ, Depatment Endocrinol, Childrens Hosp, Suzhou 215025, Peoples R China [10]Fujian Med Univ, Dept Endocrinol Genet & Metab, Fuzhou Childrens Hosp, Fuzhou 350005, Peoples R China [11]Nanjing Med Univ, Dept Endocrinol, Childrens Hosp, Nanjing 210008, Peoples R China [12]Peking Univ Third Hosp, Dept Pediat, Beijing 100191, Peoples R China [13]First Peoples Hosp Yunnan Prov, Dept Pediat, Kunming 650032, Yunnan, Peoples R China [14]China Japan Friendship Hosp, Dept Pediat, Beijing 100029, Peoples R China [15]Wuxi Childrens Hosp, Dept Endocrinol, Wuxi 214023, Jiangsu, Peoples R China [16]Xi An Jiao Tong Univ, Dept Pediat, Affiliated Hosp 2, Coll Med, Xian 710004, Peoples R China [17]Huazhong Univ Sci & Technol, Dept Endocrinol & Metab, Wuhan Childrens Hosp, Tongji Med Coll, Wuhan 430015, Peoples R China [18]Tianjin Med Univ Gen Hosp, Dept Pediat, Tianjin 300052, Peoples R China [19]Huazhong Univ Sci & Technol, Dept Pediat, Union Hosp, Tongji Med Coll, Wuhan 430022, Peoples R China [20]Huazhong Univ Sci & Technol, Dept Pediat, Tongji Hosp, Tongji Med Coll, Wuhan 430030, Peoples R China
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关键词: Short stature Children Polyethylene glycol recombinant human growth hormone Multicenter Real-world study

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Background: To evaluate the effectiveness of individualized-dose polyethylene glycol recombinant human growth hormone (PEG-rhGH) for short stature. Methods: This real-world study enrolled children with short stature in 19 hospitals throughout China. They were treated with PEG-rhGH for 6 months. The starting dosage ranged from 0.10 to 0.20 mg/kg/week. The primary outcome was the change in height standard deviation score (Delta Ht SDS). Results: Five hundred and ten patients were included and grouped based on dosage as A (0.10-0.14 mg/kg/week), B (0.15-0.16 mg/kg/week), C (0.17-0.19 mg/kg/week), and D (0.20 mg/kg/week). The mean 6-month Delta Ht SDS for the total cohort was 0.49 +/- 0.27, and the means differed among the four dose groups (P = 0.002). The Delta Ht SDS was lower in group A than in groups B (LSM difference [95%CI], -0.09 [-0.17, -0.01]), C (LSM difference [95%CI], -0.10 [-0.18, -0.02]), and D (LSM difference [95%CI], -0.13 [-0.21, -0.05]) after adjusting baseline covariates. There were no significant differences among groups B, C, and D. When the baseline IGF-1 was <-2 SDS or > 0 SDS, the Delta Ht SDS was not different among the four groups (P=0.931 and P=0.400). In children with baseline IGF-1 SDS of-2 similar to 0 SDS, a higher dosage was associated with a better treatment effect (P= 0.003), and the Delta Ht SDS was lower in older children than in younger ones (P<0.001). Conclusions: PEG-rhGH could effectively increase height in prepubertal short children. When the baseline IGF-1 was <-2 SDS, 0.10 mg/kg/week could be a starting dose. In other IGF-1 statuses, 0.15-0.20 mg/kg/week might be preferred.

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出版当年[2021]版:
大类 | 4 区 医学
小类 | 4 区 内分泌学与代谢
最新[2025]版:
大类 | 3 区 医学
小类 | 3 区 内分泌学与代谢
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出版当年[2020]版:
Q4 ENDOCRINOLOGY & METABOLISM
最新[2023]版:
Q3 ENDOCRINOLOGY & METABOLISM

影响因子: 最新[2023版] 最新五年平均[2021-2025] 出版当年[2020版] 出版当年五年平均[2016-2020] 出版前一年[2019版] 出版后一年[2021版]

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第一作者单位: [1]Capital Med Univ, Dept Endocrine & Genet & Metab, Beijing Childrens Hosp, Natl Ctr Childrens Hlth, 56 Nanlishi Rd, Beijing 100045, Peoples R China
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