重组人生长激素治疗特发性矮小症患儿的疗效

Efficacy of recombinant human growth hormone therapy in children with idiopathic short stature

目的探讨重组人生长激素(rhGH)治疗特发性矮小症(ISS)患儿的疗效。方法选择2020年10月至2022年12月于潍坊医学院附属医院儿科门诊就诊的84例ISS患儿为研究对象。首先根据是否采取rhGH治疗,将其分为治疗组(n=45,注射rhGH者)和对照组(n=39,未注射rhGH者)。其次,再根据患儿年龄,将治疗组分为治疗亚组1(n=21,年龄<8岁者),治疗亚组2(n=24,年龄≥8岁者),进一步将对照组分为对照亚组1(n=19,年龄<8岁者)和对照亚组2(n=20,年龄≥8岁者)。观察治疗组和对照组患儿的性别、年龄、身高、生长速率(GV)、骨龄、胰岛素样生长因子(IGF)-1、25羟维生素D[25(OH)D]及空腹血糖(FBG)水平。本研究遵循的程序符合潍坊医学院附属医院伦理委员会批准(审批文号:wyf-2022-ky-217),所有患儿及家属对本研究充分知晓,并签署知情同意书。结果①治疗前,治疗组和对照组患儿年龄、身高、GV、骨龄及IGF-1、25(OH)D及FBG水平比较,差异均无统计学意义(P>0.05)。②治疗2年后,治疗组和对照组患儿身高、GV、IGF-1和25(OH)D水平比较,差异均有统计学意义(t=3.80、17.27、6.34、2.34,P=0.006、0.008、<0.001、<0.001),而骨龄、FBG比较,差异均无统计学意义(P>0.05)。③治疗2年后,治疗亚组1、2患儿的身高和年GV分别优于对照1、2组,并且差异均有统计学意义(t=5.00、64.35,P=0.007、0.009)。此外,治疗亚组1患儿的年GV大于治疗亚组2,并且差异有统计学意义(t=35.60,P=0.006);而对照亚组1、2患儿的年GV比较,差异无统计学意义(P>0.05)。④治疗1年后,4个亚组患儿GV比较,治疗亚组2于治疗后7~12个月的GV明显高于0~6个月,组内比较,差异有统计学意义(t=0.37,P=0.049)。⑤研究组与对照组患儿治疗过程中出现关节疼痛、肝肾功能异常、甲状腺功能异常、FBG升高等总发生率比较,差异均无统计学意义(P>0.05)。结论rhGH治疗ISS患儿,可显著提高其身高和GV,尤其在<8岁儿童中效果更佳,并且治疗过程中未发现严重不良反应。

Objective To explore the efficacy of recombinant human growth hormone(rhGH)in the treatment of idiopathic short stature(ISS).Methods A total of 84 children with ISS diagnosed from October 2020 to December 2022 at the pediatric outpatient department of Weifang Medical College Affiliated Hospital were selected into this study.They were divided into treatment group(n=45,those who received rhGH injections)and control group(n=39,those who did not receive rhGH injections).Further stratification was done based on the children′s age into treatment group 1(n=21,children under 8 years old),treatment group 2(n=24,children aged 8 years or older),control group 1(n=19,children under 8 years old),and control group 2(n=20,children aged 8 years or older).The gender,age,height,growth velocity(GV),bone age,insulin-like growth factor(IGF)-1,25-hydroxyvitamin D[25(OH)D],and blood glucose levels of the children were observed.The procedures of this study were in accordance with the approval of the Ethics Committee of Weifang Medical College Affiliated Hospital(Approval No.wyf-2022-ky-217),and informed consents were obtained form all children and their families.Results①There were no statistically significant differences in age,height,GV,bone age,IGF-1,25(OH)D,and blood glucose levels between the treatment and control groups before treatment(P>0.05).②After 2 years of treatment,there were statistically significant differences in height,GV,IGF-1,and 25(OH)D levels between treatment and control groups(t=3.80,17.27,6.34,2.34;P=0.006,0.008,<0.001,<0.001),while there were no significant differences in bone age and blood glucose levels(P>0.05).③After 2 years of treatment,the height and annual GV of children in treatment groups 1 and 2 were significantly better than those in control groups 1 and 2,respectively,and the differences were statistically significant(t=5.00,64.35;P=0.007,0.009).Moreover,the annual GV of children in treatment group 1 was greater than that in treatment group 2,and the difference was statistically significant(t=35.60,P=0.006).However,there was no significant difference in the annual GV of children in control groups 1 and 2(P>0.05).④When comparing the GV of children in each subgroup after 1 year of treatment,the GV of treatment group 2 during months 7 to 12 was significantly higher than that during the first 6 months,and the difference was statistically significant within the group(t=0.37,P=0.049).⑤There were no statistically significant differences in the incidence of joint pain,abnormal liver and kidney function,thyroid dysfunction,and increased blood sugar during the treatment process between the two groups(P>0.05).Conclusions Treatment with rhGH can significantly improve the height and GV of children with ISS,especially in those under 8 years old,and no serious adverse reactions were found during the treatment.