Idiopathic Short Stature: A Clinical Review

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Abstract

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.

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