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

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.