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Impact of growth hormone therapy on adult height of children with idiopathic short stature: systematic review

BMJ 2011; 342 doi: (Published 11 March 2011) Cite this as: BMJ 2011;342:c7157
  1. Annalisa Deodati, research fellow,
  2. Stefano Cianfarani, associate professor
  1. 1Molecular Endocrinology Unit-DPUO, Bambino Gesù Children’s Hospital—“Rina Balducci” Center of Pediatric Endocrinology, Tor Vergata University, Rome, Italy
  1. Correspondence to: S Cianfarani, Department of Public Health and Cell Biology, Tor Vergata University, 00133, Rome, Italy stefano.cianfarani{at}
  • Accepted 10 November 2010


Objective To systematically determine the impact of growth hormone therapy on adult height of children with idiopathic short stature.

Design Systematic review.

Data sources Cochrane Central Register of Controlled Trials, Medline, and the bibliographic references from retrieved articles of randomised and non-randomised controlled trials from 1985 to April 2010.

Data extraction Height in adulthood (standard deviation score) and overall gain in height (SD score) from baseline measurement in childhood.

Study selection Randomised and non-randomised controlled trials with height measurements for adults. Inclusion criteria were initial short stature (defined as height >2 SD score below the mean), peak growth hormone responses >10 μg/L, prepubertal stage, no previous growth hormone therapy, and no comorbid conditions that would impair growth. Adult height was considered achieved when growth rate was <1.5 cm/year or bone age was 15 years in females and 16 years in males.

Results Three randomised controlled trials (115 children) met the inclusion criteria. The adult height of the growth hormone treated children exceeded that of the controls by 0.65 SD score (about 4 cm). The mean height gain in treated children was 1.2 SD score compared with 0.34 SD score in untreated children. A slight difference of about 1.2 cm in adult height was observed between the two growth hormone dose regimens. In the seven non-randomised controlled trials the adult height of the growth hormone treated group exceeded that of the controls by 0.45 SD score (about 3 cm).

Conclusions Growth hormone therapy in children with idiopathic short stature seems to be effective in partially reducing the deficit in height as adults, although the magnitude of effectiveness is on average less than that achieved in other conditions for which growth hormone is licensed. The individual response to therapy is highly variable, and additional studies are needed to identify the responders.


  • We thank Kerstin Albertsson-Wikland and Bjorn Jonsson for their helpful critical comments on the manuscript.

  • Contributors: AD and SC contributed equally to the study concept and design, literature review, data extraction, data analysis, and meta-analysis of results. Both are guarantors.

  • Competing interests: All authors have completed the Unified Competing Interest form at (available on request from the corresponding author) and declare: no support from any organisation for the submitted work; SC received lecture fees from Ipsen, Eli Lilly, Novo Nordisk, and Pfizer, consulting fees from Ipsen, Eli Lilly, and Pfizer, and research funds from Merck-Serono, Pfizer, Eli Lilly, and Ferring; no other relationships or activities that could appear to have influenced the submitted work.

  • Ethical approval: Not required.

  • Data sharing: No additional data available.

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