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Growth hormone use in children with idiopathic short stature

Karen L Weise1, Milap C Nahata

  • 1PharmD Student, The Ohio State University, Columbus, OH 43210-1291, USA.

The Annals of Pharmacotherapy
|July 22, 2004
PubMed

Insights

Recombinant human growth hormone (somatropin) offers modest height benefits for children with idiopathic short stature (ISS). However, optimal treatment timing, duration, and patient selection remain unclear, alongside ethical considerations.

Area of Science:

  • Pediatric Endocrinology
  • Growth Hormone Therapy
  • Short Stature Research

Background:

  • Idiopathic Short Stature (ISS) affects numerous children, necessitating effective treatment options.
  • Recombinant human growth hormone (somatropin) is a therapeutic agent used in pediatric growth disorders.

Purpose of the Study:

  • To comprehensively review the use of recombinant human growth hormone (somatropin) in children diagnosed with idiopathic short stature (ISS).
  • Evaluate indications, pharmacology, pharmacokinetics, efficacy, and safety profile of somatropin for ISS treatment.

Main Methods:

  • Conducted a MEDLINE search (1966-2003) using keywords: human growth hormone, somatropin, Humatrope, normal children, somatrem, and idiopathic short stature.
  • Reviewed Food and Drug Administration Advisory Committee Meeting minutes and manufacturer data.
  • Included published literature, scientific meeting abstracts, and recent pediatric/endocrinology journal articles focusing on healthy children.

Main Results:

  • Somatropin is indicated for children with ISS, demonstrating modest improvements in final height.
  • No adverse effects were reported at the doses used for ISS treatment.
  • Significant uncertainties persist regarding optimal age and duration for initiating somatropin therapy.
  • Ethical concerns include patient selection and potential for off-label use.

Conclusions:

  • Recombinant human growth hormone (somatropin) shows modest efficacy in enhancing final height for children with idiopathic short stature (ISS).
  • Key areas requiring further investigation include identifying the patient population that benefits most, determining optimal dosage regimens, and understanding long-term adverse effects of extended therapy.
Abstract

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