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Idiopathic short stature: a clinical review

Laurie E Cohen1

  • 1Director of the Neuroendocrinology Program, Division of Endocrinology, Boston Children's Hospital, and Assistant Professor of Pediatrics, Harvard Medical School, Boston, Massachusetts.

JAMA
|May 6, 2014
PubMed

Insights

Recombinant human growth hormone therapy can increase adult height in children with idiopathic short stature by approximately 5.2 cm. While short-term safety is suggested, long-term data remain limited.

Area of Science:

  • Pediatric endocrinology
  • Growth disorders
  • Pharmacological interventions

Background:

  • Idiopathic short stature (ISS) affects approximately 2% of children.
  • Management decisions for growth hormone therapy in ISS are often debated.

Observation:

  • A systematic literature review evaluated diagnostic and therapeutic strategies for pediatric ISS.
  • Studies focused on adult height outcomes and systematic reviews were prioritized.

Findings:

  • Recombinant human growth hormone (rhGH) therapy demonstrated a mean adult height increase of 5.2 cm in some children with ISS.
  • Short-term rhGH treatment appears safe, but long-term safety data are limited due to insufficient study power.

Implications:

  • Growth hormone therapy is a potential consideration for select pediatric patients with idiopathic short stature.
  • Further research is needed to establish long-term safety and efficacy profiles of rhGH therapy in ISS.
Abstract

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