Miller Bradley S, Zimmerman Donald
University of Minnesota, USA.
Pediatr Ann. 2004 Mar;33(3):177-81. doi: 10.3928/0090-4481-20040301-09.
Idiopathic short stature represents a group of conditions that are not definable by current biochemical criteria but usually respond to GH therapy. Natural-history studies confirm adult height will be short in untreated ISS individuals. Children and adults with short stature have disadvantages compared with their peers. The evidence for benefit from treatment of children with idiopathic short stature is strong. Numerous studies, now including a placebo-controlled study, have demonstrated the positive effect of GH treatment on final height. The effect of GH treatment is quantitatively similar to results seen in other non-GH-deficient conditions. Although currently very expensive, rhGH treatment is relatively safe. GH treatment of children with idiopathic short stature should not be withheld because of our inability to explain the etiology or because of the inadequacy of our current diagnostic tests. Continued efforts to delineate specific causes of poor growth in ISS individuals may result in our being able to predict subsets of individuals who will respond well to GH and subgroups who may be considered for other treatments, such as IGF-1 or a combination of IGF-1 and IGFBP-3.
特发性身材矮小代表了一组目前无法通过生化标准定义，但通常对生长激素（GH）治疗有反应的病症。自然史研究证实，未经治疗的特发性身材矮小个体成年后身高会偏矮。身材矮小的儿童和成人与同龄人相比存在劣势。有充分证据表明，治疗特发性身材矮小儿童会带来益处。包括一项安慰剂对照研究在内的众多研究均已证明生长激素治疗对最终身高有积极影响。生长激素治疗的效果在数量上与其他非生长激素缺乏病症的结果相似。尽管目前重组人生长激素（rhGH）治疗费用非常昂贵，但相对安全。不应因我们无法解释病因或当前诊断测试不完善，而不给特发性身材矮小儿童使用生长激素治疗。继续努力明确特发性身材矮小个体生长发育不良的具体原因，可能会使我们能够预测哪些个体亚组对生长激素反应良好，以及哪些亚组可能适合其他治疗方法，如胰岛素样生长因子-1（IGF-1）或IGF-1与胰岛素样生长因子结合蛋白-3（IGFBP-3）的联合治疗。
