重组人生长激素治疗儿童生长激素缺乏症安全性和有效性的大样本临床研究

Large sample clinical study on the safety and efficacy of recombinant human growth hormone in the treatment of pediatric growth hormone deficiency

目的采用大样本再评价注射用重组人生长激素（安苏萌）治疗儿童生长激素缺乏症（GHD）的安全性和有效性。方法1012例GHD患者（观察组）按照0．1IU·kg-1·d-1的剂量行睡前皮下注射安苏萌，疗程6个月，观察平均身高、生长速率、身高落后标准差、骨龄、生长激素（GH）抗体的变化以及不良反应的发生情况，并与106例未使用安苏萌治疗的GHD患儿（对照组）比较。结果观察组患儿生长速率由（2．50±0．85）cm／年增至（13．44±4．27）cm／年，净增高速率为（10．94±3．69）cm／年。治疗前后差异均有统计学意义（P〈0．01）。治疗后抗GH抗体的阳性率为13．34％，且对疗效无影响。少数患儿注射部位出现一过性红肿反应，未做处理可自行消失。结论安苏萌治疗GHD的疗效显著，有明显地促进身高增长的效果，并且不会使青春发育提前，使用安全。

Objective Using large sample clinical study to reevaluate the safety and efficacy of recombinant human growth hormone（ANSOMONE） in treating pediatric growth hormone deficiency（GHD）. Methods 1012 cases were administered with ANSOMONE at a dose of 0.1 IU ~ kg- l . d- 1 in bedtime for 6 months. The alteration of mean height, growth rate, height standard deviation scores, bone age, and anti-GH antibody and occurrence of adverse effects were observed. Results The growth rate was promoted from （ 2.50 ±0.85 ） cm/year to （ 13.44 ±4.27 ） cm/ year, the net growth rate was（ 10.94 ±3.69）cm/year. There was significant difference between the pre-treatment and post-treatment patients（ P 〈 0.01 ）. The post-treatment anti-GH antibody positive rate was 13.34% ,which had no obvious effect on the efficacy. Few patients exert transient inflammation reactions, which disappeared with any treatment. Conclusion ANSOMONE had significant efficacy, and could promote the height increase obviously. It had good safety without any obvious side effect and early-maturing effect.