吉林大学学报（医学版）2024，Vol.50Issue(6)：1703-1711,9.DOI:10.13481/j.1671-587X.20240624
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
摘要
Abstract
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 patients between two groups(P>0.05);all SMD of covariates except for term birth status were<0.2.Before balancing covariates,after meatched by PSM matching,and after weighted by IPTW weighting compared with GHD group,the GV and ΔHtSDS of the pediatric patients in ISS group were slightly increased,but the difference was not significant(P>0.05).In terms of adverse reactions,2 cases(2.68%)of fasting hyperglycemia and 7 cases(10.00%)of hypothyroidism occurred in GHD group;3 cases(4.84%)of fasting hyperglycemia and 2 cases(3.23%)of hypothyroidism occurred in ISS group.Conclusion:rhGH can promote the height increase in the patients with GHD and ISS,and there is no significant difference in the height-increasing efficacy between GHD and ISS children.The incidence of adverse reactions is relatively low during treatment,indicating good overall safety.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Key words
Growth hormone deficiency/Idiopathic short stature/Recombinant human growth hormone/Propensity score matching/Inverse probability of treatment weighting分类
医药卫生引用本文复制引用
杨希,石福艳,张旭,马艳霞,韩梅,陶子琨,卜伟晓,穆华夏,徐雅琪,王素珍..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[J].吉林大学学报（医学版）,2024,50(6):1703-1711,9.基金项目
国家自然科学基金项目(81872719) （81872719）
