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Efficacy and Safety of Human Growth Hormone in Idiopathic Short Stature. OBJECTIVE: To explore the efficacy and safety of medium dose of recombinant human growth hormone (rhGH) in the treatment of prepubescent girls with idiopathic short stature (ISS). METHODS: Fifty-one prepubescent girls with ISS were recruited into the study and divided into the treatment group (n = 27) and the control group (n = 24) depending on whether or not they accepted rhGH therapy respectively. A therapeutic dose of 0.35-0.42 mg of rhGH·(kg·wk)(-1) was administered for 2 y. The control group patients did not receive rhGH treatment. The height, growth rate, height standard deviation score (HtSDS), bone age (BA), the expected adult height (PAH), and potential side effects of rhGH treatment were analysed and compared between the groups. RESULTS: The growth rate and htSDS during years 1 and 2 were significantly higher in the treatment group than in the control group. The expected adult height was significantly higher after 2 y of treatment as compared to the corresponding value in the control group. The BA after years 1 and 2 did not significantly differ between the two groups. CONCLUSIONS: Thus, short-term therapy with a medium dose of rhGH was found to have a precise effect on prepubertal children with ISS. At this dose, it can significantly increase the growth rate of children and improve the expected adult height without accelerating bone maturation. No serious adverse reactions were found in association with rhGH use during the two-year study period.