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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效预测模型的建立和验证 被引量:3

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
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摘要 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(AHtSDS)。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AHtSDS的2个预测方程(Model-GHD和Model-total)。前瞻性分析另14例(验证组),将资料代人前述方程进行验证。结果单因素相关分析显示,与HV和AHtSDS显著(负)相关的是同一组影响因素。所得4个预测方程,R^2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AHtSDS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。 It has been proved that to analyze the factors that determine responsiveness to rhGH and to develop growth prediction models can help doctors to individualize the treatment 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 (AHtSDS), in 62 prepubertal short stature children with various GH secretary statuses were analyzed retrospectively. There were 27 patients with complete growth hormone deficiency (cGHD), 23 with partial GHD (pGHD) and 12 with idiopathic short stature (ISS) in the model group. According to the peak GH value in GH provocative test, the group of pGHD was divided into pGHD-1 (5 -6. 9μg/L, 12 patients) and pGHD-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 AHtSDS were set up by the way of multiple regression analysis, based on the results of simple correlation analysis. Other 14 children were included 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 calculated by different models so that the predictive models could be validated. Results The simple correlation analysis showed that HV and AHtSDS 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 height 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 ModeI-GHD for AHtSDS 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.
出处 《中华儿科杂志》 CAS CSCD 北大核心 2008年第10期757-762,共6页 Chinese Journal of Pediatrics
关键词 生长激素 生长激素缺乏症 特发性矮身材 预测模型 Growth hormone Growth hormone deficiency Idiopathic short stature Predictive model
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参考文献13

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二级参考文献37

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