附属第一医院儿科,广东,广州,510080
网络首发:2020-11-20,
纸质出版:2020
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何肖华, 马华梅, 李燕虹, 等. 长效生长激素治疗青春期前生长激素缺乏症的疗效分析[J]. 中山大学学报(医学科学版), 2020,41(6).
HE Xiao-hua, MA Hua-mei, LI Yan-hong, et al. Efficacy Analysis of Long-Acting Growth Hormone Treatment on Prepubertal Children with Growth Hormone Deficiency[J]. Journal of Sun Yat-sen University (Medical Sciences), 2020, 41(6).
何肖华, 马华梅, 李燕虹, 等. 长效生长激素治疗青春期前生长激素缺乏症的疗效分析[J]. 中山大学学报(医学科学版), 2020,41(6). DOI:
HE Xiao-hua, MA Hua-mei, LI Yan-hong, et al. Efficacy Analysis of Long-Acting Growth Hormone Treatment on Prepubertal Children with Growth Hormone Deficiency[J]. Journal of Sun Yat-sen University (Medical Sciences), 2020, 41(6). DOI:
【目的】对比不同剂量聚乙二醇重组人生长激素(PEG-rhGH)注射液治疗青春期前生长激素缺乏症(GHD)儿童促生长的疗效、安全性和不良事件,为临床用药提供参考。【方法】2015 年6 月至2016 年10 月在中山大学附属第一医院儿童生长发育中心确诊青春期前GHD 儿童 37 例,按PEG-rhGH 剂量随机分为两组:低剂量组(0.12 mg/kg,每周皮下注射 1 次)19 例和高剂量组(0.20 mg/kg,每周皮下注射 1 次)18 例,疗程 26 周。治疗过程监测身高、体质量、身高增长速度(HV)、身高标准差分值(HtSDS)、体质量指数(BMI)、骨龄(BA)和血清IGF-1 水平,收集不良反应信息。【结果】两组儿童治疗后的HV、HtSDS 及IGF-1 均较治疗前显著提高(P < 0.001),高剂量组 HV、HtSDS 增加值和血清 IGF-1 水平均高于低剂量组,但差异无显著性(P 值分别为 0.055、0.052 和 0.054); HtSDS 和骨龄增长两组差异无统计学意义(P > 0.05)。治疗过程中出现不同程度局部脂肪萎缩6 例(16.2%)(高剂量组3 例16.7%,低剂量组3 例15.8%),与家长相对固定注射部位相关,经指导变换注射部位后3~6 月脂肪萎缩好转。低皮 质醇血症6 例(16.2%)(高剂量组4 例22.2%,低剂量组2 例10.5%);亚临床甲状腺功能减退2 例(高剂量组1 例, 低剂量组1 例,为高TSH,T3、T4 正常)。两组的不良反应发生率无统计学差异(P > 0.05),未见明显严重不良反应。【结论】PEG-rhGH 能有效促进GHD 患儿的线性生长,高剂量组疗效似优于低剂量组。两组不良事件差异无统计学意义。聚乙二醇重组人生长激素治疗过程中有相当比例发生一定程度的局部脂肪萎缩,临床应用中必须注意注射部位轮换,可防范和降低不良反应的发生,提高治疗依从性。
【Objective】To compare the efficacy ,safety and adverse events of polyethylene glycol recombinant human growth hormone(PEG- rhGH)treatment at different dosage on prepubertal growth hormone deficiency (GHD) children,guiding clinical medication.【Methods】A total of 37 cases of prepubertal growth hormone deficiency children diagnosed in the Children Growth and Development Center of the First Affiliated Hospital of Sun Yat-sen University from June 2015 to October 2016 were collected,which were randomly divided into two groups,19 cases in the low-dose group(0.12 mg/kg per week)and 18 cases in the high-dose group(0.20 mg/kg per week). Both groups received PEG-rhGH injection subcutaneously once a week for 26 weeks. Height,weight,height velocity(HV),height standard deviation scores(HtSDS),bone age(BA),BMI(body mass index)and serum IGF-1 level were monitored and adverse events information were collected during the treatment.【Results】The HV,HtSDS incremen(t ΔHtSDS),and serum IGF-1 level of both groups were significantly higher after treatment(P < 0.001). The HV,ΔHtSDS and serum IGF- 1 level of the high-dose group was higher than that of the low-dose group,but there was no significant difference(P value 0.055,0.052 and 0.054,respectively)。 There was no significant difference in HtSDS and bone age increment between the two groups(P > 0.05). During the treatment,6 cases(16.2%)of local lipoatrophy at different degree occurred[3 cases(16.7%)in the high-dose group,3 cases(15.8%)in the low-dose group],related to the fixed injection site,which improved in 3- 6 months after changing the injection site with guidance. 6 cases(16.2%)of hypocortisolemia[4 cases(22.2%)in the high-dose group,2 cases(10.5%)in the low-dose group]and 2 cases(5.4%)of subclinical hypothyroidism[1 case(5.3%)in the high-dose group,1 case(5.6%)in the low-dose group]occurred. There was no significant difference in the incidence of adverse events between the two groups (P > 0.05). There was no serious adverse event occurred.【Conclusions】PEG-rhGH can promote the linear growth of children with GHD effectively and the growth rate of the high- dose treatment group seems to be better than that of the low-dose group. There was no significant difference in ad⁃ verse events between the two groups. A certain degree of local lipoatrophy occurs during polyethylene glycol recombinant human growth hormone treatment. Attention must be paid to the replacement of the injection site to prevent and reduce the occurrence of adverse reactions and improve treatment compliance.
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