重组人生长激素治疗不同病因矮小症患儿效果观察  

Efficacy of recombinant human growth hormone therapy for children with different causes of short stature

作　　者：张雪霜[1] 熊丰[1] 朱岷[1] 雷培芸[2] 邓蕾丽[1] 罗雁红[1] 曾燕[1] 王付丽[1] 

机构地区：[1]儿童发育疾病研究教育部重点实验室,儿童发育重大疾病国家国际科技合作基地,儿科学重庆市重点实验室,重庆医科大学附属儿童医院内分泌科,重庆400014 [2]儿童发育疾病研究教育部重点实验室,儿童发育重大疾病国家国际科技合作基地,儿科学重庆市重点实验室,重庆医科大学附属儿童医院核医学科,重庆400014

出　　处：《第三军医大学学报》2016年第16期1889-1894,共6页Journal of Third Military Medical University

摘　　要：目的探讨重组人生长激素（recombinant human growth hormone,rh GH）治疗不同病因矮小症患儿临床效果及安全性。方法回顾性分析2005年8月至2015年10月在重庆医科大学附属儿童医院内分泌专科门诊诊断为矮小症患儿200例,其中生长激素缺乏症（growth hormone deficiency,GHD）患儿73例[男性43例,女性30例,年龄（9.5±3.3）岁],特发性矮小症（idiopathic short stature,ISS）患儿56例[男性22例,女性34例,年龄（8.9±2.7）岁],特纳综合征（Turner syndrome,TS）患儿50例[年龄（9.9±2.9）岁],宫内发育迟缓（small for gestational age,SGA）患儿21例[男性8例,女性13例,年龄（6.3±2.2）岁],rh GH治疗2～4年,评价不同病因矮小症患儿身高变化、年生长速度（growth velocity,GV）、身高标准差分值（height standard deviation scores,Ht SDS）、体质指数（body mass index,BMI）、体质指数标准差分值（BMI standard deviation scores,BMI SDS）、骨龄（bone age,BA）、终身高（final adult height,FAH）的差异,并观察不良反应。结果 1经rh GH治疗后,前述不同病因矮小症患儿年生长速度较治疗前均有明显增长,且以第1年增长最明显,分别达到（11.23±2.63）、（9.91±1.67）、（8.45±1.83）、（9.78±1.72）cm/年;治疗第2～4年逐渐减缓。2经rh GH治疗后,不同病因矮小症患儿Ht SDS逐年改善,其中GHD患儿Ht SDS变化（ΔHt SDS）改善（2.12±1.12）优于ISS患儿（1.51±0.82）、TS患儿（1.23±0.73）及SGA患儿（2.07±1.04）。3随访部分已达终身高患儿,其终身高趋近于靶身高。4经rh GH治疗后,不同病因矮小症患儿BMI及BMI SDS均呈现缓慢上升趋势。5随访2～4年,不同病因矮小症患儿骨龄及青春期无明显提前,不良反应少见。结论 rh GH对GHD、ISS、TS、SGA患儿均有明显的促增长作用,但不同病因的矮小症患儿间rh GH疗效存在较大差异。Objective To investigate the differences in clinical efficacy and safety of recombinant human growth hormone（ rh GH） in treating Chinese children with different causes of short stature. Methods From August 2005 to October 2015,73 children（ 43 males and 30 females,aged 9. 5 ± 3. 3） with growth hormone deficiency（ GHD）,56 children（ 22 males and 34 females,aged 8. 9 ± 2. 7） with idiopathic short stature（ ISS）,50 children（ aged 9. 9 ± 2. 9） with Turner syndrome（ TS） and 21 children（ 8 males and 13 females,aged 9. 9 ± 2. 9） with small for gestational age（ SGA） were enrolled from the endocrine clinics of the children＇s hospital. The height（ Ht）,growth velocity（ GV）,height standard deviation score（ Ht SDS）,body mass index（ BMI）,body mass index standard deviation score（ BMI SDS）,bone age（ BA）,final adult height（ FAH） and side effects were observed during 2 ～ 4 years of rh GH treatment. Results The GVs of the enrolled children were significantly increased in the first year treatment,reaching 11. 23 ± 2. 63,9. 91 ±1. 67,8. 45 ± 1. 83 and 9. 78 ± 1. 72 cm / year,respectively,and then decreased progressively during the succeeding treatment. The Ht SDSs of the enrolled children were improved year by year,and the change of Ht SDS（ ΔHt SDS） in the children with GHD（ 2. 12 ± 1. 12） was greater than that in the children with ISS（ 1. 51 ± 0. 82）,TS（ 1. 23 ± 0. 73）,and SGA（ 2. 07 ± 1. 04）. The FAH in some of the enrolled children was documented and found to be close to their target height. After 2 ～ 4 years of rh GH treatment,the BMI and BMI SDS of the enrolled children were slowly increased. During 2 ～ 4 years of rh GH treatment,the BA and puberty of the enrolled children didn ＇t advance,and side effects were rare. Conclusion The rh GH treatment can effectively promote growth in the children with GHD,ISS,TS and SGA. However,the efficacy of rh GH differs greatly in the children with different causes of short stature. The child

关 键 词：重组人生长激素 矮小症 

分 类 号：R725.81[医药卫生—儿科] R725.84[医药卫生—临床医学]