重组人生长激素治疗不同生长激素分泌状态青春前期矮身材患儿近期疗预测模型的建立和验证

目的 建立重组人生长激素(rhGH)治疗生长激素不同分泌状态青春前期矮身材患儿近期(1年)疗效的预测模型,并进行初步验证.方法回顾性分析62例生长激素不同分泌状态的青春前期矮身材患儿模型组,分为全模型组(模型组全部病例)和生长激素缺乏症模型组(模型组中生长激素缺乏症的病例)]经rhGH治疗1年后的追赶性生长指标:生长速度(HV)和身高Z分增值(ΔHtSDS).根据单因素相关分析的结果,通过多元回归的方法,分别建立对HV和ΔHtSDS的2个预测方程(Model-GHD和Model-total).前瞻性分析另14例(验证组),将资料代入前述方程进行验证.结果单因素相关分析显示,与HV和ΔHtSDS显著(负)相关的是同一组影响因素.所得4个预测方程,R2在0.244～0.519,P值均＜0.05.HV的2个预测方程和对生长激素缺乏症患儿1ΔHtSDS的预测方程(实测值和预测值呈显著正相关,r在0.753～0.996;配对t检验示两者差异无统计学意义).结论预测模型建立成功,有助于预测不同生长激素分泌状态青春期矮身材患儿的生长激素的近期疗效.

Establishment and validation of predictive model of short term responses to recombinant human growth hormone treatment in prepubertal short stature children with various growth hormone secretary statuses

It has been proved that to analyze the factors that determine responsiveness to rhGH and to develop growth prediction models Call help doctors to individtudize the rteatment and maximize the effect.Objectives To set up and validate the predictive models of growth responses to rhGH treatment in the first year in prepubertal short stature children with various GH secretary statuses.Methods Growth responses to rhGH treatment in the first year,height velocities (HV) and increases in height SDS(ΔHtSDS),in 62 prepubeml short stature children with various GH secretary statuses were analyzed retrospectively.There were 27 patients with complete growth hormone deficiency (cCHD),23 with partial GHD (pCHD) and 12 with idiopathic short stature (ISS) in the model group.According to the peak CH value in GH provocative test,the group of pCHD was divided into pGHD-1 (5-6.9 μg/L,12 patients) and pCHO-2 (7-9.9 μg/L,11 patients).All the cases in model group were used for setting up Model-total and the cases of growth hormone deficiency for Model-GHD.Predictive models,including Model-GHD and Model-total,to HV and ΔHtSDS were set up by the way of multiple regression analysis,based on the results of simple correlation analysis.Other 14 children were ineluded according to the same criteria with the model group,the validation group.The validation group was analyzed prospectively.The actual growth responses were compared with the predicted values calculsted by different models so that the predictive models could be validated.Results The simple correlation analysis showed that HV and ΔHtSDS in the first year were negatively correlated with the same group factors at baseline:chronological age,bone age,height SDS,differences between the height SDS and the target heisht SDS,peak value in GH provocative test and IGF-1SDS.All the 4 predictive models were found to be significant at a level of P＜0.05,R2 ranged from 0.244 to 0.519.The two models predicted HV and Model-GHD for ΔHtSDS were proved to be validated.The observed and predicted responses positively and significantly correlated with each other,r value ranged from 0.753 to 0.996.And there was no significant difference between them when tested by paired t test.Conclusions The availability of the predictive model will help to individualize the growth hormone treatment in prepubertal short stature children with various growthhormone secretary status.