基因重组人生长激素治疗青春期前特发性生长激素缺乏症临床试验

Efficacy and safety of LG recombinant human growth hormone in the treatment of idiopathic growth hormone deficiency

目的:评价基因重组人生长激素(rhGH)治疗青春期前特发性生长激素缺乏症(IGHD)患儿6个月的疗效和安全性。方法:对49例IGHD患儿进行rhGH治疗,每晚睡前皮下注射0．5IU·kg^(-1),每周分5次注射,共26周。评价治疗前后身高、生长速度、身高标准差计数(SDS)等指标。结果:在rhGH治疗期间,46例患儿生长速度由每年(2．5±1．0)cm提高到(11．5±2．5)cm(P＜0．005)。身高SDS由治疗前(-4．2±1．8)增为(-3．7±1．8)。同时患儿13和26周血清胰岛素样生长因子(IGF-1)和IGF结合蛋白(IGFBP,)水平均较治疗前明显升高(P＜0．05)。其体重与骨龄无明显变化。治疗后共有15．7%患儿出现甲状腺功能降低,抗hGH抗体阳性率为21．7%,但所有这些现象均未影响患者体格的线性增长。在治疗期间所有患者肝肾功能、血尿常规和代谢性指标(如血糖和血脂水平)等均保持在正常范围。结论:rhGH是治疗IGHD安全有效的药物。

Objective：To assess the efficacy and safety of recombinant growth hormone LG Eutropin^TM for children with idiopathic growth hormone deficiency（IGHD）. Methods：49 children with IGHD were subcutaneously injected with Eutropin^TM in a dose of 0. 5 IU/kg/week, divided by 5 times, before sleep for 26 weeks. The primary endpoints are to measure the changes of height, growth velocity and mean height SDS pre and post therapy. Results：The mean growth velocity and mean height SDS increased from （2.45±0.97） cm/year, （-4.2±1.8） to （11.5±2.5） cm/year, （-3.7±1.8）, respectively; the p values were P 〈 0. 005. The creased significantly from the serum IGF-1 and IGFBP3 levels at the 13 and 26 weeks of the therapy inbaseline levels. No bone maturation in advance and weight gain were found. 15.7% and 21.7% patients who received Eutropin Tu experienced hypothyroidism and increment of low titer serum anti-rhGH antibody, respectively, all of which had no an effect on their linear body growth. No abnormalities in the liver and kidney functions, blood and urinary tests and metabolism index were found. Conclusion：This LG rhGH is an effective and safe growth stimulator in children with IGHD.