国产重组人生长激素对特发性矮身材儿童生长的促进效果(英文)

Use of home-made recombinant human growth hormone in the growth of children with idiopathic short stature

背景:国外已有许多研究证实重组人生长激素治疗特发性矮身材安全有效,但国内尚未见此类长期系列研究。目的:观察重组人生长激素对特发性矮身材患儿的促生长效果,并与生长激素缺乏症患儿进行比较。设计:病例对比观察。单位:青岛大学医学院附属医院儿内科。对象:选择2004-12/2006-03于青岛大学医学院儿内科就诊的矮身材患儿98例,患儿监护人均知情同意。按病因分为2组,特发性矮身材组30例,生长激素缺乏症组68例。方法:特发性矮身材组及生长激素缺乏症组患儿均给予国产重组人生长激素(长春金赛药业有限责任公司生产)治疗,剂量分别为0.15IU/(kg·d),0.1IU/(kg·d),每晚睡前皮下注射,疗程6个月。治疗前及治疗后3,6个月分别测定患儿的身高、体质量、骨龄,观察注射部位局部反应,计算生长速度。骨龄按照GP图谱法测算,预测身高按BP法计算。主要观察指标:两组患儿治疗前及治疗3,6个月后身高、体质量、骨龄及生长速度的变化情况。结果:98例患儿全部进入结果分析,无脱落。①组内比较结果:治疗后3,6个月后两组患儿的身高、生长速度均显著高于治疗前[特发性矮身材组身高:(126.5±9.4),(129.1±8.6),(121.1±11.0)cm(P<0.01),生长速度:(7.3±2.9),(7.5±2.7),(3.5±2.1)cm/年(P<0.01);生长激素缺乏症组身高:(111.0±13.0),(114.0±13.0),(108.0±12.0)cm(P<0.01),生长速度:(13.2±3.5),(13.5±3.6),(4.0±2.9)cm/年(P<0.01)]。治疗6个月后,特发性矮身材组30例患儿中27例促进了身高增长,生长激素缺乏症组68例患儿全部促进了身高增长。②组间比较:治疗3,6个月后两组患儿同期的生长速度比较,生长激素缺乏症组高于特发性矮身材组(P<0.01)。③不良反应和副作用:2例患儿治疗1～3个月出现甲状腺功能减低现象,给予甲状腺素片口服治疗后未影响生长。1例注射局部红肿,1周后自行消失,未停药。结论:国产重组人生长激素治疗特发性矮身材儿童安全,总体有效,能明显提高患儿身高及生长速度,但疗效存在不均一性,且差于生长激素缺乏症儿童。

BACKGROUND： Many foreign studies confirm that recombinant human growth hormone （rhGH） is a safe and effective agent for treatment of idiopathic short stature （ISS）, but there are no long-period and systematical researches reported in China. OBJECTIVE： To observe the promoting effect of rhGH on the growth of children with ISS. DESIGN ： Case-control observation. SETTING ： Department of Pediatrics, the Affiliated Hospital of Medical College of Qingdao University. PARTICIPANTS： Ninety-eight children with ISS who were treated in the Department of Pediatrics, the Affiliated Hospital of Medical College of Qingdao University during December 2004 to March 2006 were involved in this study. Informed consents were obtained from the guardians of these children with ISS. According to the etiological factors, the children were assigned into 2 groups： ISS group （n =30） and growth hormone deficiency （GHD） group （n =68）. METHODS： The children in ISS group and GHD group received subcutaneous administration of home-made rhGH （Jinsai Pharmaceutical Co.,Ltd., Changchun at 0.15 IU/（kg·d） and 0.1 IU/（kg·d） respectively before sleeping within 6 months. The body height, body mass and bone age were measured before, 3 and 6 months after treatment. The local region of injection was observed and the growth rate was calculated. Bone age was calculated by graphic atlas method and body height was predicated by BP method. MAIN OUTCOME MEASURES ： Body height, body mass, bone age and growth rate of children in two groups before, 3 and 6 months after treatment. RESULTS ： All the 98 children were＇involved in the result analysis, without deletion. （1）lntragroup comparison： The body height, growth rate of children in two groups 3 and 6 months after treatment were significantly superior to those before treatment [ISS group： body height：（126.5±9.4）, （129.1 ±8.6）, （121.1 ±11.0） cm （P 〈 0.01）, growth rate： （7.3±2.9）, （7.5± 2.7）,（3.5±2.1） cm/year （P 〈 0.01）; GHD group： body height： （111.0±13.0）,（114.0±13.0）,（108.0±12.0） cm（P 〈 0.01）, growth rate： （13.2±3.5）,（13.5±3.6）,（4.0±2.9） cm/year（P 〈 0.01）]. Six months after treatment, body height was increased in 27 of 30 children in ISS groups, and in all the 68 children in GHD group. （2） Intergroup comparison： The growth rate of children in GHD group was concurrently higher than that in ISS group 3 and 6 months after treatment. （3）Adverse reaction and side effect： During the treatment, hypothyroidism was observed in 2 children within 1 to 3 months of treatment, but there was no influence on growth rate after oral administration of thyroxin tablets. Local red swelling of skin was observed in another patient, which stopped automatically after one week duration, but the injection continued. CONCLUSION ： Home-made rhGH is a safe and effective reagent for treatment of ISS. It can obviously enhance the body height and growth rate of children. Whereas the inhomogeneity of treatment effect does exist, what＇s more, therapeutic effect in ISS children is inferior to that in GHD children.