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Idiopathic short stature: conundrums of definition and treatment

Arlan L Rosenbloom1

  • 1Department of Pediatrics, Children's Medical Services Center, University of Florida College of Medicine, 1701 SW 16th Ave, Gainesville, FL 32608, USA.

International Journal of Pediatric Endocrinology
|December 4, 2009
PubMed

Insights

Idiopathic short stature (ISS) in children is distinct from familial short stature and constitutional delay in growth and maturation (CDGM). Growth hormone (GH) treatment for ISS offers limited height gain with no proven quality of life benefits, making its high cost difficult to justify.

Area of Science:

  • Pediatric Endocrinology
  • Growth Disorders
  • Metabolic Bone Disease

Background:

  • Idiopathic short stature (ISS) is defined by height SDS < -2, distinct from familial short stature and constitutional delay in growth and maturation (CDGM).
  • Low IGF-I levels in ISS may stem from undernutrition or inappropriate reference standards in CDGM.
  • Distinguishing ISS from CDGM is crucial for accurate diagnosis and treatment planning.

Purpose of the Study:

  • To critically evaluate the efficacy and justification of growth hormone (GH) treatment for children with idiopathic short stature (ISS).
  • To review the evidence regarding the impact of GH treatment on quality of life and psychological adaptation in ISS.
  • To highlight the lack of data on IGF-I efficacy for ISS treatment.

Main Methods:

  • Meta-analysis of existing studies on growth hormone (GH) treatment outcomes in ISS.
  • Review of consensus statements from pediatric endocrinology societies.
  • Comparative analysis of diagnostic criteria for ISS, familial short stature, and CDGM.

Main Results:

  • Growth hormone (GH) treatment may increase adult height by 4-5 cm in children with ISS.
  • Meta-analysis found no documented evidence that GH treatment improves health-related quality of life or psychological adaptation.
  • The cost-effectiveness of GH treatment for ISS is questionable, with an estimated cost of US$52,000 per inch gained.

Conclusions:

  • The efficacy of growth hormone (GH) and IGF-I treatments for idiopathic short stature (ISS) requires further investigation.
  • Expectations that taller stature achieved through GH treatment will improve quality of life should be discouraged.
  • Accurate differentiation between ISS and CDGM is essential for appropriate patient management.

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