Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
Curr Opin Endocrinol Diabetes Obes. 2011 Feb;18(1):3-8. doi: 10.1097/MED.0b013e328341f873.
We review recent developments in the therapy of growth disorders, focusing mainly on recent developments in the field of growth hormone (GH) therapy.
Review of current practice reveals wide variation in the approach to short stature and the institution of GH therapy. No new indications for GH treatment have been approved by the Food and Drug Administration since 2007, but off-label use has been explored in several chronic illnesses. There is also increasing focus on GH effects beyond increase in stature (e.g. effects on body composition and bone density). As the experience with GH increases, the long-term risks and outcomes are better understood and risks appear to be low. Data regarding beneficial effects of GH in Prader-Willi syndrome are accumulating but questions remain about patient selection, start age and safety. Recombinant insulin-like growth factor 1 has become more widely available and has been aggressively promoted for Food and Drug Administration-approved indications as well as off-label uses in the last few years. The use of aromatase inhibitors has been tempered by concern about side-effects and lack of efficacy.
Growth hormone remains the mainstay of therapy for growth disorders, though other options, including recombinant insulin-like growth factor 1, are being investigated in various settings.
我们主要关注生长激素(GH)治疗领域的最新进展,对生长障碍的治疗进展进行综述。
目前的治疗实践回顾表明,对身材矮小的处理和 GH 治疗的实施存在广泛的差异。自 2007 年以来,美国食品和药物管理局(FDA)尚未批准任何新的 GH 治疗适应症,但在几种慢性疾病中已经探索了 GH 的超适应症使用。人们也越来越关注 GH 除了增加身高之外的作用(例如对身体成分和骨密度的影响)。随着 GH 应用经验的增加,其长期风险和结果得到了更好的理解,风险似乎很低。关于 GH 在普拉德-威利综合征中的有益作用的数据正在积累,但关于患者选择、起始年龄和安全性的问题仍然存在。重组胰岛素样生长因子 1 的应用越来越广泛,并且在过去几年中,因其在 FDA 批准的适应症以及超适应症方面的应用而被大力推广。由于对副作用和疗效的担忧,芳香化酶抑制剂的应用有所减少。
生长激素仍然是治疗生长障碍的主要方法,尽管其他选择,包括重组胰岛素样生长因子 1,也在各种情况下得到了研究。