Bryant J, Baxter L, Cave C B, Milne R
University of Southampton, Wessex Institute for Health Research and Development, Mailpoint 728, Biomedical Sciences Building, Bassett Crescent East, Southampton, Hants, UK, SO16 7PX.
Cochrane Database Syst Rev. 2007 Jul 18(3):CD004440. doi: 10.1002/14651858.CD004440.pub2.
Idiopathic short stature (ISS) refers to children who are very short compared with their peers for unknown or hereditary reasons. Recombinant human growth hormone (GH) has been used to increase growth and final height in children with ISS.
To assess the effects of recombinant human GH on short-term growth and final height in children with ISS.
Studies were obtained from computerised searches of MEDLINE, EMBASE, The Cochrane Library, Science Citation Index, BIOSIS and Current Controlled Trials. Article reference lists were assessed for trials and experts and pharmaceutical companies were contacted.
Randomised controlled trials were included if they were carried out in children with ISS with normal GH secretion. GH had to be administered for a minimum of six months and be compared with placebo or no treatment. A growth or height outcome measure had to be assessed.
Two reviewers assessed studies for inclusion criteria and for methodological quality. Data were extracted by one reviewer and checked by a second. The primary outcome was final height and secondary outcomes included short term growth, health related quality of life and adverse effects. To estimate summary treatment effects, data were pooled, when appropriate using a random effects model.
Ten RCTs were included. One trial reported near final height in girls and found that girls treated with GH were 7.5 cm taller than untreated controls (GH group, 155.3 cm +/- 6.4; control, 147.8 cm +/- 2.6; P = 0.003); another trial which reported adult height standard deviation score found that children treated with GH were 3.7 cm taller than children in a placebo-treated group (95% confidence intervals 0.03 to 1.10; P < 0.04). The other trials reported short term outcomes. Results suggest that short-term height gains can range from none to approximately 0.7 SD over one year. One study reported health related quality of life and showed no significant improvement in GH treated children compared with those in the control group, whilst another found no significant evidence that GH treatment impacts psychological adaptation or self-perception in children with ISS. No serious adverse effects of treatment were reported.
AUTHORS' CONCLUSIONS: GH therapy can increase short-term growth and improve (near) final height. Increases in height are such that treated individuals remain relatively short when compared with peers of normal stature. Large, multicentre RCTs are required which should focus on final height and address quality of life and cost issues.
特发性身材矮小（ISS）是指由于不明原因或遗传因素导致比同龄人矮小的儿童。重组人生长激素（GH）已被用于促进ISS患儿的生长及提高最终身高。
评估重组人生长激素对ISS患儿短期生长及最终身高的影响。
通过计算机检索MEDLINE、EMBASE、Cochrane图书馆、科学引文索引、BIOSIS和当前对照试验获取研究。评估文章参考文献列表以查找试验，并联系专家及制药公司。
纳入在GH分泌正常的ISS患儿中进行的随机对照试验。GH必须至少给药6个月，并与安慰剂或未治疗进行比较。必须评估生长或身高结局指标。
两名评审员评估研究是否符合纳入标准及方法学质量。由一名评审员提取数据并由另一名进行核对。主要结局为最终身高，次要结局包括短期生长、健康相关生活质量及不良反应。为估计汇总治疗效果，在适当情况下使用随机效应模型合并数据。
纳入10项随机对照试验。一项试验报告了女孩的接近最终身高情况，发现接受GH治疗的女孩比未治疗的对照组高7.5厘米（GH组，155.3厘米±6.4；对照组，147.8厘米±2.6；P = 0.003）；另一项报告成人身高标准差得分的试验发现，接受GH治疗的儿童比接受安慰剂治疗的儿童高3.7厘米（95%置信区间0.03至1.10；P < 0.04）。其他试验报告了短期结局。结果表明，短期身高增长在1年内可能从无增长到约0.7标准差不等。一项研究报告了健康相关生活质量，显示与对照组相比，接受GH治疗的儿童无显著改善，而另一项研究未发现显著证据表明GH治疗会影响ISS患儿的心理适应或自我认知。未报告治疗的严重不良反应。
GH治疗可促进短期生长并改善（接近）最终身高。与正常身高的同龄人相比，接受治疗的个体身高增加后仍相对较矮。需要开展大型多中心随机对照试验，应关注最终身高并解决生活质量和成本问题。
