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Treatment of children and adolescents with idiopathic short stature

Nature Reviews Endocrinology volume 9, pages 325–334 (2013)Cite this article

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Abstract

Idiopathic short stature (ISS) is defined as shortness in childhood without a specific cause. ISS may be familial or nonfamilial and may be associated with or without delay of pubertal development. Treatment can be considered in an attempt to reduce the psychological burden caused by short stature in childhood and adult life. If counselling alone is not sufficient, medical modifications of the growth process can be attempted. In cases with pubertal delay, sex steroids, such as testosterone and oxandrolone, can favourably influence height velocity and growth tempo, although adult height is not affected. Medications that prolong the process of growth—for example, gonadotropin-releasing hormone agonists or aromatase inhibitors—might increase adult height, but findings to date are still experimental. Growth hormone therapy is approved for the treatment of very short children with reduced adult height expectation, as evidence has accumulated that this therapy can increase height in childhood and in adult life. Sensitivity to growth hormone is impaired in patients with ISS; therefore, doses higher than a replacement dose have to be applied. This treatment still needs to be optimized in terms of efficacy, cost-effectiveness and long-term safety. A debate is ongoing concerning the psychological benefit of height increase, with clinicians warning against the medicalization of a deviation in height.

Key Points

  • Idiopathic short stature is defined as a condition of short stature after exclusion of specific causes

  • Children with idiopathic short stature often remain short in adult life

  • Shortness might cause a psychological burden, particularly in childhood

  • Children with shortness and/or developmental delay can be treated with sex steroids, medications that prolongate growth or growth factors

  • Therapy with recombinant human growth hormone is approved (in the USA) because of its potential to increase adult height

  • Although growth hormone therapy is safe in the short term (for example, <10 years), long-term safety needs to be explored further

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Figure 1: Algorithm for the diagnosis and treatment of ISS.

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  1. University Children's Hospital Tübingen, Hoppe-Seyler-Strasse 1, Tübingen, D-72076, Germany

    Michael B. Ranke

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The author declares associations with the following companies: Ipsen (honoraria), Novo Nordisk (honoraria), Pfizer (consultancy, honoraria).

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Ranke, M. Treatment of children and adolescents with idiopathic short stature. Nat Rev Endocrinol 9, 325–334 (2013). https://doi.org/10.1038/nrendo.2013.71

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