生长激素治疗综合征与非综合征矮身材小于胎龄儿的效果比较

Efficacy of recombinant human growth hormone treatment in children born small for gestational age with syndromic and non-syndromic short stature

目的比较重组人生长激素治疗综合征型和非综合征型矮身材的小于胎龄儿的效果及代谢参数变化。方法回顾性分析2012年7月至2021年6月就诊于北京儿童医院内分泌遗传代谢科且应用重组人生长激素治疗的59例矮身材小于胎龄儿患儿的临床资料,根据2019年矮身材共识分为综合征型与非综合征型,在治疗前、治疗后6、12、18及24个月时,组间采用独立t检验或Kruskal-Wallis秩和检验,比较身高标准差积分(Ht-SDS)、身高标准差积分差值(∆Ht-SDS)及稳态模型-胰岛素抵抗指数(HOMA-IR);组内采用配对t检验或U检验,比较治疗前后Ht-SDS及HOMA-IR。结果59例患儿中男37例、女22例,年龄(5.5±2.3)岁,综合征型27例、非综合征型32例。综合征型及非综合征型患儿在治疗12及24个月后ΔHt-SDS差异均无统计学意义(0.9±0.4比1.2±0.4,1.4±0.7比1.9±0.5,t=1.68、1.52,P=0.104、0.151);治疗12个月时非综合征型患儿的HOMA-IR较综合征型更高[2.29(1.43,2.99)比0.90(0.55,1.40),Z=-2.95,P=0.003];综合征型仅在治疗6个月时较治疗前、12个月时较6个月时Ht-SDS的差异有统计学意义(Z=7.65、2.83,P<0.001、P=0.020),非综合征型治疗6个月时较治疗前、12个月较6个月、18个月较12个月,24个月较18个月时Ht-SDS的差异均有统计学意义(Z=11.95、7.54、4.26、3.83,均P<0.001)。结论综合征型与非综合征型矮身材的小于胎龄儿应用重组人生长激素疗效相当,但前者身高增长主要在治疗第1年;对于非综合征型患儿应用重组人生长激素治疗时更需要关注胰岛素抵抗程度,可能需要缩短随访间期。

Objective To analyse the efficacy of recombinant human growth hormone(rhGH)treatment in children born small for gestational age(SGA)with syndormic and non-syndormic short stature.Methods The clinical data of 59 children born SGA who were diagnosed as short stature and admitted to the Center of Endocrinology,Genetics and Metabolism,Beijing Children′s Hospital from July 2012 to June 2021 were collected and analyzed.According to the 2019 consensus on short stature,they were divided into syndromic group and non-syndromic group.Before treatment and 6,12,18 and 24 months after treatment,height standard deviation score(△Ht-SDS),difference of height standard deviation(∆Ht-SDS)and homeostasis model assessment-insulin resistance index(HOMA-IR)were compared between groups,while Ht-SDS and HOMA-IR were compared before and after treatment.Independent t test or Kruskal-Wallis test were used for comparison between the 2 groups,and paired t test or Mann-Whitney U test were used for the intra-group comparison.Results Among the 59 cases,37 were males and 22 females,aged(5.5±2.3)years.There was no significant difference in Ht-SDS after 12 months of treatment between 2 groups(0.9±0.4 vs.1.2±0.4,t=1.68,P=0.104)or in height SDS after 24 months of treatment(1.4±0.7 vs.1.9±0.5,t=1.52,P=0.151).After 12 months of treatment,the insulin resistance index of the non-syndromic group was significantly higher than that of the syndromic group(2.29(1.43,2.99)vs.0.90(0.55,1.40),Z=-2.95,P=0.003).There were significant differences in Ht-SDS between 6 months and before treatment,12 months and 6 months in syndromic type(Z=7.65,2.83 P<0.001,P=0.020),but all were significant differences in non-syndromic type between 6 months and before treatment,12 months and 6 months,18 months and 12 months,24 months and 18 months(Z=11.95,7.54,4.26,3.83,all P<0.001).Conclusion The efficacy of rhGH treatment in children born SGA is comparable between syndromic and non-syndromic short stature cases,but non-syndromic children treated with rhGH need more frequent follow-up due to the risk of insulin resistance.