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.
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.
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