重组人生长激素治疗生长激素缺乏症疗效分析及预测模型探讨  

Effect of recombinant human growth hormone therapy on GHD patients and establishment of prediction model

作　　者：武华红[1] 李辉[1] 孙淑英[1] 

机构地区：[1]首都儿科研究所生长发育研究室,北京100020

出　　处：《中国实用儿科杂志》2011年第4期289-293,共5页Chinese Journal of Practical Pediatrics

摘　　要：目的探讨重组人生长激素(recombinant human growth hormone,rhGH,简称GH)治疗生长激素缺乏症(growth hormone deficiency,GHD)患儿效果及影响因素,建立GH治疗效果预测模型。方法回顾性分析1996年8月至2010年9月首都儿科研究所生长发育门诊确诊为GHD和多垂体功能低下(multiple pituitary hormonedeficiency,MPHD)且接受规范GH治疗的矮身材患儿115例临床资料,采用2009年卫生部最新颁布的中国儿童体格发育标准对儿童身高、体重进行标化,标准差计算采用国际公认的LMS方法。以治疗过程中的身高标准差分值变化(delta in height SDS,ΔHtSDS)和生长速度(growth velocity,GV)为效果评价指标,进行疗效和影响因素分析。用多元回归方法以75例治疗满1年且随访较规律者为模型人群,建立治疗效果预测模型。同时前瞻性随访15例规范治疗的GHD患儿为模型验证对象,对模型进行验证。结果患儿治疗第1年身高平均增长(10.56±2.83)cm,ΔHtSDS升高0.93±0.52;治疗前3个月的ΔHtSDS及GV均大于>3～6个月、>6～9个月及>9～12个月,治疗前6个月增值也大于>6～12个月;第2年身高增长(7.99±3.03)cm,ΔHtSDS升高0.73±0.37。个体疗效差异与骨龄落后程度、遗传身高水平及早期治疗反应成正相关,与骨龄大小、治疗前体内生长激素水平及胰岛素样生长因子成负相关;多元回归分析得出治疗第1年ΔHtSDS和GV的预测模型,R2在0.20～0.48之间(P均<0.05)实测值和预测值之间配对t检验差异均无统计学意义(P>0.05)。结论 GH用于GHD患儿治疗早期效果明显,尤其是治疗前3个月;本研究建立的预测模型有一定的应用价值,可用治疗前3个月的效果预测第1年的治疗效果。Objectives Tonalyze the effect and influencing factors of GH therapy on GHD patients,and to establish the growth prediction model for patients on GH therapy.Methods Growth velocity （GV） and delta in height SDS （ΔHTSDS） were used as outcome variables of growth responses to rhGH therapy.We used the new growth standard of Chinese children in 2009 to calculate the HtSDS and WtSDS of patients by the generally accepted method of LMS.Seventy patients diagnosed with GHD and MPHD who accepted rhGH therapy and regularly revisited were chosen as model group.Then retrospectively analyze the effect and influencing factors of their GH therapy.Based on the result of simple correlation analysis,we established prediction models on therapy effect in the first year by multiple liner regression.Meanwhile,15 patients with growth deficiency under therapy of rhGH were prospectively followed up for at least one year to validate our models.Results The height increment of patients receiving therapy was 10.56 cm （0.93 SDS） in the first year,during which the ΔHtSDS and GV during the first 3 months were all larger than those in 3～6 months,6～9 months and 9～12 months.The height increment was 7.99 cm （0.73 SDS） in the second year.According to the simple correlation analysis and multiple liner regression,we found that GV and ΔHTSDS in the first year were negatively correlated with patients’ age and differences between height SDS and middle parent height SDS at start of therapy,while positively correlated with their weight SDS and bone age retardation.Also,the growth responses in the first 3 months of therapy were found positively correlated with the response later,so we established a model on ΔHTSDS in the first year for this.All these 3 models were found to be significant at a level of P 0.05,R2 ranging from 0.199～0.473.There were no significant differences between actual and predictive responses both in model and validation groups.Conclusion During therapy,we find the effect of earlier therapy is better than later

关 键 词：生长激素缺乏症 重组人生长激素 治疗效果 影响因素 预测模型 

分 类 号：R72[医药卫生—儿科]