基于倾向评分法对重组人生长激素治疗生长激素缺乏症和特发性矮小症患儿的疗效及安全性评价  

Efficacy and safety evaluation of recombinant human growth hormone in treatment of pediatric patients with GHD and ISS based on propensity scores

作　　者：杨希 张旭 马艳霞 韩梅 陶子琨 卜伟晓 穆华夏 徐雅琪 王素珍 石福艳 YANG Xi;ZHANG Xu;MA Yanxia;HAN Mei;TAO Zikun;BU Weixiao;MU Huaxia;XU Yaqi;WANG Suzhen;SHI Fuyan(Department of Health Statistics,School of Public Health,Shandong Second Medical University,Weifang 261053,China;Department of Cardiovascular Medicine,People’s Hospital,Qufu City,Shandong Province,Qufu 273100,China;Department of Endocrinology,People’s Hospital,Weifang City,Shandong Province,Weifang 261041,China)

机构地区：[1]山东第二医科大学公共卫生学院卫生统计学系,山东潍坊261053 [2]山东省曲阜市人民医院心血管内科,山东曲阜273100 [3]山东省潍坊市人民医院内分泌科,山东潍坊261041

出　　处：《吉林大学学报（医学版）》2024年第6期1703-1711,共9页Journal of Jilin University：Medicine Edition

基　　金：国家自然科学基金项目(81872719)。

摘　　要：目的:探讨重组人生长激素(rhGH)治疗生长激素缺乏症(GHD)和特发性矮小症(ISS)患儿的临床疗效,阐明其在不同病因矮身材患儿中的临床应用价值。方法:收集2018年1月—2023年1月就诊并接受rhGH治疗的132例矮身材患儿的临床资料,按照病因不同分为GHD组(n=70)和ISS组(n=62),选取并计算患儿骨龄、靶身高(TH)、体质量指数(BMI)、身高标准差分值(HtSDS)、治疗前和治疗6个月后身高标准差分值变化(ΔHtSDS)及生长速率(GV)等生长指标,采用倾向性评分匹配法(PSM)和逆概率加权法(IPTW)均衡2组患儿混杂因素,评价2组患儿临床疗效和安全性。结果:2组患儿是否为足月生产、骨龄、骨龄成熟度和TH比较差异有统计学意义(P<0.05)。与治疗前比较,GHD组和ISS组患儿治疗6个月后身高和HtSDS均明显增加(P<0.05)。PSM法匹配前,2组患儿是否为足月生产、骨龄、骨龄成熟度和TH组间比较差异均有统计学意义(P<0.05);PSM法匹配后,2组患儿性别、地区、是否为足月生产、分娩方式、喂养方式、年龄、骨龄、身高、BMI、TH和治疗前HtSDS组间比较差异均无统计学意义(P>0.05);除地区外,各协变量的标准均值差(SMD)均<0.2。IPTW法加权后,2组患儿性别、地区、是否为足月生产、分娩方式、喂养方式、年龄、骨龄、身高、BMI、TH和治疗前HtSDS各协变量组间比较差异均无统计学意义(P>0.05);除是否为足月生产外,各协变量SMD均<0.2。均衡协变量前、PSM法匹配后和IPTW法加权后,与GHD组比较,ISS组患儿GV和ΔHTSDS均略有升高,但差异均无统计学意义(P>0.05)。2组患儿不良反应,GHD组患儿发生空腹高血糖2例(2.68%),甲状腺功能减退7例(10.00%);ISS组患儿发生空腹高血糖3例(4.84%),甲状腺功能减退2例(3.23%)。结论:rhGH可促进GHD与ISS患儿身高增长,且对GHD与ISS患儿增高疗效无明显差异。在用药过程中,患儿不良反应发生率较低,整体安全性良好。Objective:To discuss the clinical efficacy of recombinant human growth hormone(rhGH)in the treatment of the pediatric patients with growth hormone deficiency(GHD)and idiopathic short stature(ISS),and to clarify its clinical application value in the pediatric patients with short stature of different etiologies.Methods:The clinical data of 132 children with short stature who treated with rhGH from January 2018 to January 2023 were collected.They were divided into GHD group(n=70)and ISS group(n=62)based on different etiologies.The bone age,target height(TH),body mass index(BMI),height standard deviation score(HtSDS),changes in height standard deviation scores(ΔHtSDS)before treatment and 6 months after treatment,and growth velocity(GV)of the pediatric patients were calculated.Propensity score matching(PSM)and inverse probability of treatment weighting(IPTW)were used to balance the confounding factors between the pediatric patients in two groups and the efficacy and safety of the pediatric patients in two groups were evaluated.Results:There were significant differences in whether children were full-term,bone age,bone age maturity,and TH of the pediatric patients between two groups(P<0.05).Compared with before treatment,the height and HtSDS of the pediatric patients in both GHD and ISS groups were significantly increased after treated for 6 months(P<0.05).Before matched by PSM,there were significant differences in full-term,bone age,bone age maturity,and TH of the pediatric patients between two groups(P<0.05).After matched by PSM,there were no significant differences in gender,region,term birth status,mode of delivery,feeding method,age,bone age,height,BMI,TH,and pretreatment HtSDS of the pediatric patients between two groups(P>0.05);the standardized mean difference(SMD)differences of covariates except for region were<0.2.After weighted by IPTW,there were no significant differences in gender,region,term birth status,mode of delivery,feeding method,age,bone age,height,BMI,TH,and pretreatment HtSDS of the pediatric pati

关 键 词：生长激素缺乏症 特发性矮小症 重组人生长激素 倾向性评分匹配法 逆概率加权法 

分 类 号：R725.8[医药卫生—儿科]