Growth response to rhIGF‐I 80 μg/kg twice daily in children with growth hormone insensitivity syndrome: relationship to severity of clinical phenotype

Abstract

This study reveals that IGF-I treatment at a dose of 80 microg/kg twice daily is effective in patients with growth hormone insensitivity syndrome. During the first 12 months of therapy, there was a significant inverse relationship between growth response to IGF-I therapy and the severity of the phenotype of growth hormone insensitivity syndrome, as measured by height SDS, at the start of therapy. Patients with a more severe clinical phenotype of growth hormone insensitivity syndrome, who also had most severe IGFBP-3 deficiency, responded better than those who were more mildly affected. An analogous situation has been shown to be the case in GH-deficient patients treated with hGH.