Idiopathic short stature: Results of a one-year controlled study of human growth hormone treatment

A multicenter randomized trial of human growth hormone treatment was carried out in 121 children with short stature who did not meet the classic criteria for growth hormone deficiency. Patients treated for 1 year with recombinant somatotropin, 0.1 mg/kg three times a week, had a significant increase in mean growth rate from 4.6 ± 1.1 to 7.5 ± 1.2 cm/yr, whereas untreated children's growth rate did not change significantly (4.2 ± 1.3 vs 5.0 ± 1.4 cm/yr). There was a 1-year advance in bone age for each group; thus there was a significant increase in the predicted height of the treated but not the control group. Among the treated children, the growth response did not differ among those classified on the basis of parental height and bone age as having familial short stature or constitutional delay of growth and development. Prestudy anthropomorphic features were not related to subsequent growth in either the treated or control groups. The baseline plasma insulin-like growth factor I concentration was inversely related to the growth response to growth hormone treatment (r = -0.50, p = 0.0003). By contrast, the serum growth hormone concentration measured in samples obtained at 20-minute intervals for 12 or 24 hours or after clonidine administration did not predict the future growth rate. There were no side effects of growth hormone treatment. The results suggest that children who have significantly short stature and slow growth may benefit from a trial of growth hormone therapy.