Therapy with recombinant human IGF-1 for children with primary insulin-like growth factor-I deficiency

Growth Horm IGF Res. 2020 Apr:51:22-26. doi: 10.1016/j.ghir.2020.01.001. Epub 2020 Jan 13.


The efficacy and safety of IGF-1 therapy in patients with severe primary IGF-I deficiency has been evaluated for more than two decades. Most of the therapeutic experience comes from treating the more severe IGF-I deficient patients, who usually present with a phenotype characteristic of growth hormone receptor deficiency or Laron syndrome. Although most of these patients do not experience enough catchup growth to bring their height into normal range, many individuals achieve an adult height significantly greater than what would have been predicted in the absence of IGF-1 therapy. In the last couple of years a few reports on the benefit of IGF-1 therapy for patients with milder types of IGF-I deficiency have also been published, with variable height outcomes. More short children with prior diagnosis of idiopathic short stature are now being diagnosed with specific molecular defects of the growth hormone/IGF-I axis. Because of this, the clinical spectrum of primary IGF-I deficiency is widening to include many patients with such a milder phenotype, creating a need for well-designed long-term clinical studies evaluating the growth response to growth promoting agents such as rhIGF-1 in these individuals.

Keywords: Growth hormone; Growth hormone insensitivity; Growth hormone receptor; Growth hormone resistance; Insulin-like growth factor-I; Primary IGF-I deficiency; Recombinant human IGF-1.

MeSH terms

  • Child
  • Child, Preschool
  • Human Growth Hormone / metabolism
  • Humans
  • Insulin-Like Growth Factor Binding Protein 3 / metabolism
  • Insulin-Like Growth Factor I / deficiency*
  • Insulin-Like Growth Factor I / therapeutic use
  • Laron Syndrome / drug therapy*
  • Laron Syndrome / genetics
  • Laron Syndrome / metabolism
  • Pituitary Function Tests
  • Receptors, Somatotropin / genetics
  • Recombinant Proteins
  • Severity of Illness Index


  • IGF1 protein, human
  • IGFBP3 protein, human
  • Insulin-Like Growth Factor Binding Protein 3
  • Receptors, Somatotropin
  • Recombinant Proteins
  • Human Growth Hormone
  • Insulin-Like Growth Factor I