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Review of growth hormone randomized controlled trials in children with idiopathic short stature

Annalisa Deodati1, Emanuela Peschiaroli, Stefano Cianfarani

  • 1Molecular Endocrinology Unit, DPUO Bambino Gesù Children's Hospital, Rina Balducci Center of Pediatric Endocrinology, Tor Vergata University, Rome, Italy.

Hormone Research in Paediatrics
|September 14, 2011
PubMed

Insights

Recombinant growth hormone (GH) therapy increases adult height in children with idiopathic short stature (ISS). However, treated children remain shorter than peers, necessitating further research on optimal dosage and long-term safety.

Area of Science:

  • Pediatrics
  • Endocrinology
  • Clinical Trials

Background:

  • Recombinant human growth hormone (GH) is used to enhance growth in children with idiopathic short stature (ISS).
  • Long-term efficacy and safety data for GH treatment in ISS are limited.
  • Systematic reviews are needed to consolidate evidence from randomized controlled trials (RCTs).

Purpose of the Study:

  • To systematically review all available RCTs on GH use in children with ISS.
  • To evaluate the long-term efficacy of GH therapy in achieving adult height.
  • To identify gaps in current research regarding GH treatment for ISS.

Main Methods:

  • A comprehensive literature search was conducted using Cochrane Central Register of Controlled Trials, Medline, and reference lists.
  • The search included RCTs published up to August 2009.
  • Three long-term RCTs assessing adult height outcomes were identified and analyzed.

Main Results:

  • GH therapy, initiated around age 10.1 years for a mean of 5.4 years, increased adult height by a mean of 0.77 SDS (approx. 5.5 cm).
  • Mean adult height achieved was -1.5 SDS, indicating treated children remain relatively short.
  • Significant variability in individual response to GH therapy was observed.

Conclusions:

  • GH therapy improves adult height in children with ISS but does not normalize height.
  • Further research is required to identify predictors of treatment response.
  • High-quality, long-term RCTs are necessary to establish optimal GH dosage and long-term safety in ISS.

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