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  • Review Article
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Use of growth hormone in children

Nature Clinical Practice Endocrinology & Metabolism volume 2, pages 260–268 (2006)Cite this article

Abstract

The introduction of recombinant DNA-derived human growth hormone (rhGH) in the mid-1980s allowed studies to be undertaken in a number of growth disorders other than the classic indication—growth-hormone deficiency (GHD). In patients with GHD, optimizing the dose and frequency of rhGH administration, and early instigation of therapy, has led to near-normalization of final height. The use of rhGH in the treatment of Turner syndrome, Prader–Willi syndrome, intrauterine growth restriction, and chronic renal failure demonstrated the efficacy of therapy, although the increase in final height (5–7 cm) is less than that achieved in GHD. Cost–benefit implications need to be considered in the next phases of evaluating the role of rhGH therapy in these indications. To date, rhGH has only received approval for the management of idiopathic short stature in the US; as with the other wider growth indications, the lack of formal randomized, controlled trials hampers the full evaluation of efficacy, and a cautious approach should, therefore, be adopted for this particular indication. rhGH has a good safety record, although there are current concerns about the possible long-term increased risk of colonic and lymphatic malignancy, which will require monitoring through national cancer registries.

Key Points

  • Recombinant DNA-derived human growth hormone (rhGH) therapy is effective in growth-hormone deficiency; if rhGH is used at a daily dose of 0.025–0.035 mg/kg body weight, administered by subcutaneous injection from an early age, normalization of final height to within the target height of the family can be achieved

  • At the end of therapy, all patients with isolated growth-hormone deficiency should be retested and rhGH therapy continued into adulthood in the most severely affected; for individuals with reduced GH secretion, careful follow-up by an adult endocrinologist is warranted

  • Data from the only randomized, controlled trial of rhGH therapy in patients with Turner syndrome showed a net gain of 7.3 cm with therapy: at a daily dose of rhGH of 0.05 mg/kg body weight, six patients needed to be treated in order that one would reach a height of over 150 cm

  • The other indications for rhGH therapy—chronic renal failure and intrauterine growth restriction—require similar doses to Turner syndrome, but efficacy remains to be demonstrated in formal studies

  • There is insufficient information from randomized, controlled studies to advise on the use of rhGH therapy in patients with idiopathic short stature

  • rhGH has a good safety record to date, but ongoing pharmacologic vigilance is required, particularly in the uncertain area of long-term risk of malignancy

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Author information

Authors and Affiliations

  1. University College London, London, UK

    Peter C Hindmarsh & Mehul T Dattani

  2. Great Ormond Street Hospital for Children and University College London Hospitals, London

    Peter C Hindmarsh & Mehul T Dattani

  3. Biochemistry, Endocrinology and Metabolism Unit, Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 3EH, UK

    Peter C Hindmarsh

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Correspondence to Peter C Hindmarsh.

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Hindmarsh, P., Dattani, M. Use of growth hormone in children. Nat Rev Endocrinol 2, 260–268 (2006). https://doi.org/10.1038/ncpendmet0169

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