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Clinical Crossroads
May 7, 2014

Idiopathic Short StatureA Clinical Review

Author Affiliations
  • 1Director of the Neuroendocrinology Program, Division of Endocrinology, Boston Children’s Hospital, and Assistant Professor of Pediatrics, Harvard Medical School, Boston, Massachusetts
JAMA. 2014;311(17):1787-1796. doi:10.1001/jama.2014.3970

Importance  Approximately 2% of children are defined as having short stature. Deciding when to pursue recombinant human growth hormone therapy to increase adult height is controversial.

Objective  To review the management of children with idiopathic short stature, including diagnostic evaluation and therapeutic options.

Evidence Review  Systematic literature search of PubMed, Embase, and the Cochrane Library databases. For height outcome, articles were limited to studies reporting adult height and to systematic reviews.

Findings  Recombinant human growth hormone therapy of children with idiopathic short stature increases height in some children. The estimated mean gain in adult height is 5.2 cm (2 in). The cost-benefit ratio is controversial. Treatment with growth hormone appears safe in the short term, while data on long-term effects are limited because studies of long-term efficacy were not powered to determine safety.

Conclusions and Relevance  Growth hormone treatment may be considered in some children with idiopathic short stature.