Investigational agents for the treatment of growth hormone-insensitivity syndrome

Stephen F. Kemp, Kathryn M. Thrailkill

Research output: Contribution to journalReview articlepeer-review

7 Scopus citations


Growth hormone-insensitivity syndrome (GHIS) is usually caused by mutations in the growth hormone receptor. Recombinant IGF-I has been used to treat GHIS either alone (mecasermin) or in combination with IGF-binding protein (IGFBP)-3 (mecasermin rinfabate). IGF-I increases the growth velocity of children with IGF deficiency, which is either as a result of GHIS or an IGF-I gene deletion. Hypoglycaemia has been reported with administration of unbound IGF-I and, in addition, the serum half-life of unbound IGF-I is shorter when administered to patients with GHIS (who have low serum concentrations of its binding protein IGFBP-3) than when administered to normal volunteers or to patients with an IGF-I gene deletion (but normal IGFBP-3 levels). The IGF-I-IGFBP-3 combination was developed to prolong the half-life and counteract the acute adverse events (particularly hypoglycaemia) that are associated with administration of IGF-I. It seems that the IGF-I-IGFBP-3 combination has a longer half-life in patients with GHIS than unbound IGF-I, with fewer reports of adverse events (including hypoglycaemia) when administered to patients with diabetes. There are no studies comparing the efficacy of mecasermin with mecasermin rinfabate; both drugs have been shown to be effective but cannot be differentiated in terms of efficacy.

Original languageEnglish
Pages (from-to)409-415
Number of pages7
JournalExpert Opinion on Investigational Drugs
Issue number4
StatePublished - Apr 2006


  • Growth hormone
  • Growth hormone-insensitivity syndrome
  • Insulin-like growth factors
  • Laron syndrome
  • Short stature hypoglycaemia

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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