Clinical Inquiries

Can recombinant growth hormone effectively treat idiopathic short stature?

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EVIDENCE-BASED ANSWER

Yes, treatment can increase a child’s final height. Injections of recombinant human growth hormone (rGH) at least 3 times a week for 4 to 6 years add 3.7 to 7.5 cm to final height in children between 8 and 16 years of age with idiopathic short stature (strength of recommendation [SOR]: B, 2 small, low-quality, randomized controlled trials [RCTs]). This population comprises children who are otherwise physically and developmentally normal with a height standard deviation score (SDS) of ≤–2.0—comparable to the bottom 2.5% percentile of height—and an adequate response to growth hormone stimulation testing.

Evidence summary

rGH has been available since 1985. The Food and Drug Administration has approved it for such conditions as growth hormone deficiency, chronic renal insufficiency, Turner syndrome, small size for gestational age, and Prader-Willi syndrome.1 The use of rGH to treat idiopathic short stature introduces many clinical, economic, and ethical questions. We have attempted to discern the clinical effectiveness of treatment by focusing on RCTs of rGH therapy while leaving the other substantive issues unexplored.

Final height is arguably the most important outcome measure for the effects of rGH and may be represented as actual height or as a standard deviation score (SDS)—actual height minus mean height for age divided by standard deviation of height for age.2 This measure standardizes height comparisons for different age groups and is comparable to the percentile values on growth charts.

Growth hormone increases height in girls and boys

A 2003 Cochrane systematic review identified 9 RCTs that evaluated treatment with rGH in children with idiopathic short stature. Only 1 used near final height as its main outcome. Inclusion criteria for this RCT comprised prepubertal girls in the bottom third percentile for height without a known cause.

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Evidence-based answers from the Family Physicians Inquiries Network

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