生长激素治疗非生长激素缺乏性生长障碍。

Growth hormone treatment of non-growth hormone-deficient growth disorders.

作者信息

Quigley Charmian A

机构信息

Lilly Research Laboratories, Drop Code 5015, Lilly Corporate Center, Indianapolis, IN 46285, USA.

出版信息

Endocrinol Metab Clin North Am. 2007 Mar;36(1):131-86. doi: 10.1016/j.ecl.2006.11.006.

DOI:10.1016/j.ecl.2006.11.006

PMID:17336739

Abstract

Although a large body of data on efficacy and safety of growth hormone (GH) treatment for various non-growth hormone-deficient (GHD) growth disorders has accumulated from a combination of clinical trial and postmarketing sources in the last 20 years or more, there remain limitations. Clinical trial data have the advantage of direct comparison of well-matched, randomized patient groups receiving treatment (or not) under comparable conditions and, as such, provide the highest quality evidence of efficacy. Clinical trials, however, are typically too small for any statistically valid assessment for safety, which is more comprehensively addressed using postmarketing data. Consequently, while the efficacy of GH treatment in children with non-GHD growth disorders has been solidly established and, based on the combination of the rigor of the clinical trial data and numerical power of the postmarketing data, no major concerns exist regarding safety, additional long-term data are required.

摘要

尽管在过去20多年里，通过临床试验和上市后数据积累了大量关于生长激素（GH）治疗各种非生长激素缺乏（GHD）性生长障碍的疗效和安全性的数据，但仍存在局限性。临床试验数据的优势在于，能在可比条件下对接受治疗（或未接受治疗）的匹配良好的随机患者组进行直接比较，因此能提供最高质量的疗效证据。然而，临床试验通常规模太小，无法对安全性进行任何具有统计学效力的评估，而上市后数据能更全面地解决这一问题。因此，虽然生长激素治疗非生长激素缺乏性生长障碍儿童的疗效已得到确凿证实，并且基于临床试验数据的严谨性和上市后数据的数量优势，在安全性方面不存在重大担忧，但仍需要更多长期数据。