Achondroplasia: Effect of Growth Hormone in 40 Patients

This is the authors' abstract. We don't add key points for this paper.

We administered growth hormone (GH) to 40 patients with achondroplasia, 9 of whom were suspected to have insufficient GH secretion. The patients received 0.5 or 1.0IU/kg/week recombinant human GH. At the end of the first year, the annual height gain was significantly increased compared to before treatment. There was no difference between patients with normal or insufficient GH secretion in the annual height gain, either before or during GH treatment. The annual height gain during treatment of patients treated with GH at 0.5IU/kg/week did not differ from those treated with 1.0IU/kg/week. The annual height gain before and during GH treatment of patients in the pubertal stage was less than those in the prepubertal stage. Although the annual height gain in the second year of GH treatment was less than in the first year, it was still greater than before GH treatment. The lower limb length to height ratio was significantly increased by GH treatment. No adverse effects were observed during the treatment period of about two years.

rhGH was associated with greater height velocity in achondroplasia; hypothesis-generating and requires RCT confirmation before practice change.