重组人生长激素治疗骨及软骨相关矮小症的疗效及其影响因素  

Analysis of factors influencing and efficacy of recombinant human growth hormone in the treatment of children with bone and cartilage associated short stature

作　　者：陈雅楠 谭鑫 邓德明 康权[4] 钟立霖 CHEN Ya-nan;TAN Xin;DENG De-ming;KANG Quan;ZHONG Li-lin(Department of Child Health,Neijiang Maternal and Child Health Hospital,Sichuan Neijiang 641100,China;Department of Obstetrics,Neijiang Maternal and Child Health Hospital,Sichuan Neijiang 641100,China;Department of Pediatrics,Neijiang Maternal and Child Health Hospital,Sichuan Neijiang 641100,China;Department of General Trauma Surgery,Children's Hospital Affiliated to Chongqing Medical University,Chongqing 400014,China;Department of Pediatrics,Xiamen Maternal and Child Health Hospital,Fujian Xiamen 361003,China)

机构地区：[1]内江市妇幼保健院儿童保健科,四川内江641100 [2]内江市妇幼保健院产科,四川内江641100 [3]内江市妇幼保健院儿科,四川内江641100 [4]重庆医科大学附属儿童医院普外创伤外科,重庆400014 [5]厦门市妇幼保健院儿科,福建厦门361003

出　　处：《临床药物治疗杂志》2022年第9期67-71,共5页Clinical Medication Journal

基　　金：国家卫生健康委医药卫生科技发展研究中心项目(WA2020HK39)。

摘　　要：目的 探讨重组人生长激素(rhGH)治疗骨及软骨相关矮小症(BCD)的疗效及其影响因素,为临床诊治BCD提供科学依据。方法 回顾性选择2016年1月至2018年10月内江市妇幼保健院收治的226例BCD患儿为研究对象。按照rhGH治疗效果,分为有效组(n=180)和无效组(n=46),比较两组治疗前后的骨龄(BA)、生长速率(GR)、身高(Ht);多因素logistic回归分析rhGH治疗BCD的影响因素;构建影响rhGH治疗疗效不佳的列线图模型,并对列线图模型的区分度和校准度进行评价。结果 治疗前,两组BA、GR、Ht水平差异均无统计学意义(均P>0.05);有效组治疗后BA、GR、Ht均优于无效组,差异有统计学意义(P<0.05)。有效组与无效组在身高标准差积分(HtSDS)、生长激素峰值(GH peak)、胰岛素样生长因子1(IGF-1)、骨龄指数(BAI)、尿酸(UA)、骨源性碱性磷酸酶(BAP)方面比较,差异均有统计学意义(均P<0.05)。多因素logistic回归分析显示,HtSDS、GH peak、IGF-1、BAI、UA、BAP是rhGH治疗BCD的影响因素,各因素比较差异有统计学意义(P<0.05)。列线图模型显示,rhGH治疗BCD疗效不佳的影响率为37.57%,且评价区分度的受试者工作特征(ROC)曲线下面积为0.821(95%CI:0.754~0.926,P<0.001),区分度较好。预测模型GiViTI校准曲线带的95%CI区域均未穿过45°对角平分线(P=0.188),模型的预测概率与实际观测概率接近,具有较强的一致性。结论 BCD患儿应用rhGH治疗,总体疗效显著,HtSDS、GH peak、IGF-1、BAI、UA、BAP是rhGH治疗BCD的影响因素。Objective This study was designed to investigate the efficacy and influencing factors of recombinant human growth hormone(rhGH)in the treatment of bone and cartilage associated short stature(BCD),and provide scientific basis for clinical diagnosis and treatment of BCD. Methods 226 BCD patients admitted to Neijiang Materal and Child Health Hospital from January 2016 to October 2018 were retrospectively selected as the research objects. According to the effect of rhGH treatment,they were divided into effective group(n=180) and ineffective group(n=46). Bone age(BA),growth rate(GR),height(Ht)were compared between the two groups before and after treatment. Multiple logistic regression analysis of rhGH treatment of BCD factors was conducted and nomogram model that affects poor efficacy of rhGH treatment was constructed.The degree of discrimination and calibration of the nomogram model were evaluated. Results Before treatment,the levels of BA,GR,and Ht in the two groups were not statistically significant(all P >0. 05);after treatment,the BA,GR,and Ht levels of the effective group were better than those of the ineffective group,and the difference was statistically significant(P <0. 05).There were statistically significant differences between the effective group and the ineffective group in height standard deviation score(HtSDS),growth hormone peak(GH peak),IGF-1,BAI,uric acid(UA),and BAP(all P <0. 05). Multivariate logistic regression analysis showed that HtSDS,GH peak,IGF-1,BAI,UA,and BAP were the influencing factors of rhGH treatment for BCD and the differences among these factors were statistically significant(P <0. 05). The nomogram model showed that the influence rate of rhGH in the treatment of BCD was 37. 57%,and the area under the receiver operating characteristic(ROC)curve for evaluating the discrimination was 0. 821(95%CI:0. 754-0. 926,P<0. 001),and the discrimination was good. The 95%CI area of the GiViTI calibration curve belt of the prediction model did not cross the 45-degree diagonal bisector(P=0. 188). Th

关 键 词：骨及软骨相关矮小症 重组人生长激素 疗效 

分 类 号：R977[医药卫生—药品]