长效生长激素治疗青春期前生长激素缺乏症的疗效分析  

Efficacy Analysis of Long-Acting Growth Hormone Treatment on Prepubertal Children with Growth Hormone Deficiency

作　　者：何肖华[1] 马华梅[1] 李燕虹[1] 李思勤[1] HE Xiao-hua;MA Hua-mei;LI Yan-hong;LI Si-qin(Department of Pediatrics,The First Affiliated Hospital of Sun Yat-sen University,Guangzhou 510080,China)

机构地区：[1]中山大学附属第一医院儿科,广东广州510080

出　　处：《中山大学学报（医学科学版）》2020年第6期981-990,共10页Journal of Sun Yat-Sen University:Medical Sciences

基　　金：广东省科技计划项目(2010B031600230)。

摘　　要：【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)、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 children with prepubertal growth hormone deficiency(GHD)children,guiding clinical medication.【Methods】A total of 37 cases of children with prepubertal growth hormone deficiency children diagnosed in the Children Growth and Development Center of the First Affiliated Hospital of Sun Yatsen 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)and serum IGF-1 level were monitored and adverse events information were collected during the treatment.【Results】The HV,HtSDS and serum IGF-1 level of both groups were significantly higher after treatment(P<0.001).The HV,HtSDS increment(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,re⁃spectively).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 degrees[3 cases(16.7%)in the high-dose group,and 3 cases(15.8%)in the low-dose group],related to the fixed injection site occurred,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 t

关 键 词：长效生长激素 生长激素缺乏症 剂量 效果 对比研究 

分 类 号：R725[医药卫生—儿科]