依据类胰岛素生长因子水平调整重组人生长激素治疗特发性矮小的有效性分析

Efficacy analysis of idiopathic short stature treatment with recombinant human growth hormone based on the level of insulin-like growth factor-1

目的探索在特发性矮小患儿的治疗中,根据类胰岛素生长因子(IGF)水平调整重组人生长激素(rh GH)剂量的有效性。方法采用随机、前瞻性、平行观察研究,对62例青春前期特发性矮身材患儿分为rh GH固定剂量组、低水平IGF-1组和高水平IGF-1组,分别进行rh GH治疗,定期随访共2年。由专人专用量具测量身高并记录身高变化(ΔHT),生长速率(GV,cm/年);每隔6个月拍摄1次左手正位X线片,判断骨成熟情况(骨成熟度=ΔBA/ΔCA)。观察各治疗组身高标准差积分(Ht SDS)、GV、骨成熟度(ΔBA/ΔCA)变化情况等各项临床及生化指标,采用方差分析或配对t检验对所得结果进行分析。结果三组患儿治疗后身高及GV、Ht SDS均较治疗前改善(P<0.01);高水平IGF-1组在Ht SDS改变及身高增长速率各方面均明显优于低水平IGF-1组及生长激素固定剂量组(F=9.505,P<0.01)。低IGF-1组及生长激素固定剂量组之间无明显差异(F=0.058,P>0.05),治疗后各组骨成熟度无明显差异(F=0.576,P>0.05)。结论高水平IGF-1对矮小患儿的线性生长有更显著的促进作用。IGF水平在特发性矮小患儿进行rh GH治疗中可作为个体对生长激素治疗敏感性的良好参考指标,对rh GH剂量的调整具有指导意义。

Objective To explore the efficacy and safety of recombinant human growth hormone(rh GH) in the treatment of children with idiopathic short stature. Methods A randomized, prospective, parallel study was made in 62 children with prepubertal idiopathic short stature. Patients were randomly assigned to recombinant human growth hormone fixed dose group, low level insulin like growth factor 1(IGF-1) group and high level IGF-1 group, and treated with rh GH respectively with a follow-up of 2 years. Height was measured and the changes of height(ΔHT), growth rate(GV, cm/year) were recorded by special medical person with special instruments. The radiograph of left hand was took every 6 months to judge bone maturation(bone maturation degree=ΔBA/ΔCA). Variation of clinical and biochemical indexes of different treatment groups, including the height standard deviation score(Ht SDS), growth rate(GV), bone maturation degree(ΔBA/ΔCA), were observed and analyzed with ANOVA or paired t test. Results Results showed that the height, growth rate, height standard deviation score were all improved in three groups after treatment(P<0.01); The height, growth rate, height standard deviation score of high level IGF-1 group were significantly improved better than that of low level IGF-1 group and growth hormone fixed dose group(F=9.505, P<0.01). There was no significant difference between low level IGF-1 group and the growth hormone fixed dose group(F=0.058, P>0.05). There was no significant difference in bone maturation(F=0.576, P>0.05) between three groups after treatment. Conclusions High level of IGF-1 has a more significant effect on the linear growth of children with idiopathic short stature. Insulin-like growth factor levels of idiopathic short stature children in recombinant human growth hormone therapy can be considered a good indicator of individual sensitivity to GH treatment, and may have significant implications in rh GH dose adjustment.