Recombinant growth hormone for idiopathic short stature in children and adolescents

There is some evidence that recombinant human growth hormone improves short term growth and (near) final adult height in children with idiopathic short stature.

Idiopathic short stature is the term used when children are very short compared with others of their age for unknown or hereditary reasons. They do not have a disease. Recombinant human growth hormone has been used to try to overcome growth failure in these children. It must be injected under the skin six to seven times a week until adult height is reached. Existing evidence suggests that growth hormone can increase short term growth and improve final or near final adult height.
Ten studies included altogether 741 children and lasted between six months and 6.2 years. Results showed that individuals treated with growth hormone remain relatively short when compared with peers of normal stature. Girls treated with growth hormone were 7.5 cm taller than untreated controls (growth hormone treated group 155.3 cm and control group 147.8 cm); another trial found that children treated with growth hormone were 3.7 cm taller than children in a placebo-treated group. No serious adverse effects were reported in the included studies. Although serious adverse effects (there has been concern that growth hormone would induce new tumours or increase the likelihood of tumour relapse) may be rare, their possibility must also be taken into consideration.

Authors' conclusions: 

GH therapy can increase short-term growth and improve (near) final height. Increases in height are such that treated individuals remain relatively short when compared with peers of normal stature. Large, multicentre RCTs are required which should focus on final height and address quality of life and cost issues.

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Background: 

Idiopathic short stature (ISS) refers to children who are very short compared with their peers for unknown or hereditary reasons. Recombinant human growth hormone (GH) has been used to increase growth and final height in children with ISS.

Objectives: 

To assess the effects of recombinant human GH on short-term growth and final height in children with ISS.

Search strategy: 

Studies were obtained from computerised searches of MEDLINE, EMBASE, The Cochrane Library, Science Citation Index, BIOSIS and Current Controlled Trials. Article reference lists were assessed for trials and experts and pharmaceutical companies were contacted.

Selection criteria: 

Randomised controlled trials were included if they were carried out in children with ISS with normal GH secretion. GH had to be administered for a minimum of six months and be compared with placebo or no treatment. A growth or height outcome measure had to be assessed.

Data collection and analysis: 

Two reviewers assessed studies for inclusion criteria and for methodological quality. Data were extracted by one reviewer and checked by a second. The primary outcome was final height and secondary outcomes included short term growth, health related quality of life and adverse effects. To estimate summary treatment effects, data were pooled, when appropriate using a random effects model.

Main results: 

Ten RCTs were included. One trial reported near final height in girls and found that girls treated with GH were 7.5 cm taller than untreated controls (GH group, 155.3 cm ± 6.4; control, 147.8 cm ± 2.6; P = 0.003); another trial which reported adult height standard deviation score found that children treated with GH were 3.7 cm taller than children in a placebo-treated group (95% confidence intervals 0.03 to 1.10; P < 0.04). The other trials reported short term outcomes. Results suggest that short-term height gains can range from none to approximately 0.7 SD over one year. One study reported health related quality of life and showed no significant improvement in GH treated children compared with those in the control group, whilst another found no significant evidence that GH treatment impacts psychological adaptation or self-perception in children with ISS. No serious adverse effects of treatment were reported.

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