Several studies have evaluated the effects of growth hormone (GH) on auxological and biochemical parameters in children with non-GH-deficient, idiopathic short stature (ISS). This study evaluated the efficacy and safety of GrowtropinR-II (recombinant human GH) in Korean patients with ISS. This was a 1-year, open-label, multicenter, phase III randomized trial of Growtropin-II in Korean patients with ISS. In total, 70 prepubertal subjects (39 males, 31 females) between 4 and 12 years of age were included in the study. All patients were naive to GH treatment. Annual height velocity was significantly higher in the treatment group (10.68±1.95 cm/year) than the control group (5.72±1.72, p<0.001). Increases in height and weight standar deviation scores (SDSs) at 26 weeks were 0.63±0.16 and 0.64±0.46, repectively, for the treatment group, and 0.06±0.15 and 0.06±0.28, respectively, for the control group (p<0.001). Serum insulin-like growth factor (IGF-1) and insulin-like growth factor binding protein-3 (IGFBP-3) increased significantly in the treatment group at week 26 compared to baseline. However, the SDS for body mass index(BMI) at 26 weeks did not change significantly in either group. GrowtropinR-II was well tolerated and safe over 1 year of treatment. One-year GH treatment for prepubertal children with ISS demonstrated increased annualized velocity, height and weight SDSs, and IGF-1 and IGFBP-3 levels, with a favorable safety profile. Further evaluations are needed to determine the optimal dose, final adult height, and long-term effects of ISS treatment.
27 Sep 2018 - 29 Sep 2018