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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
【摘要】 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。
【Abstract】 Objectlve:To assess the efficacy and safety of recombinant growth hormone LG Eu- tropinTMfor children with idiopathic growth hormone deficiency(IGHD).Methods:49 children with IGHD were subcutaneously injected with EutropinTMin 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 serum IGF-1 and IGFBP3 levels at the 13 and 26 weeks of the therapy in- creased significantly from the baseline levels.No bone maturation in advance and weight gain were found. 15.7% and 21.7% patients who received EutropinTMexperienced hypothyroidism and increment of low titer serum anti-rhGH antibody,respectively,all of which had no an effect on their linear body growth.No ab- normalities in the liver and kidney functions,blood and urinary tests and metabolism index were found. Condusion:This LG rhGH is an effective and safe growth stimulator in children with IGHD.
【Key words】 idiopathic growth hormone deficiency(IGHD); recombinant human growth hormone(rhGH); growth; height;
- 【文献出处】 中国新药杂志 ,Chinese Journal of New Drugs , 编辑部邮箱 ,2006年05期
- 【分类号】R725.8
- 【被引频次】3
- 【下载频次】232