Review of Growth Hormone Randomized Controlled Trials in Children with Idiopathic Short Stature

Abstract

Recombinant human growth hormone (GH) has been used to increase growth and adult height in children with idiopathic short stature (ISS) despite the lack of definitive, long-term studies of efficacy. Our aim was to systematically review all randomized controlled trials (RCTs) of GH use in children with ISS.We performeda formal search of the Cochrane Central Register of Controlled Trials, Medline and the bibliographic references from all retrieved articles describing such trials up to August 2009. Three long-term RCTs conducted to the achievement of adult height were identified. The mean age at start of treatment in the three trials was 10.1 ± 3.4 years. The mean duration of GH therapy was 5.4 ± 0.9 years. The mean difference between treated and untreated children was 0.77 ± 0.4 standard deviation score (SDS) (approximately 5.5 cm). The mean adult height achievedafter GH therapy at conventional doses was –1.5 ± 0.3 SDS. GH improves adult height in children with ISS. However, treated individuals remain relatively short compared with peers of normal height. The individual response to therapy is highly variable and further studies are required to identify responders. Lastly, high-quality evidence from long-term RCTs of GH therapy that continue until achievement of adult height is necessary to determine the ideal dosage and long-term safety.