Abstract
PurposeSeveral 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 Growtropin®-II (recombinant human GH) in Korean patients with ISS.MethodsThis 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.ResultsAnnual 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 standard deviation scores (SDSs) at 26 weeks were 0.63 ± 0.16 and 0.64 ± 0.46, respectively, 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. Growtropin®-II was well tolerated and safe over 1 year of treatment.ConclusionsOne-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.
Highlights
Children with idiopathic short stature (ISS) are a heterogeneous group; there are many unidentified causes of short stature, and wide variation in causative biological factors, the extent of bone-age delay, and timing of puberty [1]
86 Korean subjects were recruited from 11 hospitals in February 2012, of whom 70 were enrolled in this study, which ended in April 2014
The subjects had no history of growth hormone (GH) therapy before this study, and there was no significant group difference in demographic information or baseline characteristics (Table 1)
Summary
Children with idiopathic short stature (ISS) are a heterogeneous group; there are many unidentified causes of short stature, and wide variation in causative biological factors, the extent of bone-age delay, and timing of puberty [1]. ISS is defined as a height more than two standard deviations (SD) below the age-, sex-, and population-matched mean, despite a normal or increased response to growth hormone (GH) on the GH stimulation test, and in the absence of any systemic, endocrine or metabolic disease, chromosome abnormality, or other disease that may compromise growth [2, 3]. In 2003, the FDA extended the indications for GH therapy to include children with ISS who are > 2.25 SD below the mean height, and who are unlikely to catch up in height [7]. Studies evaluating GH therapy in children with ISS for 4–7 years revealed an increase in adult height (AH) of 3–6 cm [1, 3, 8, 9], and a safety profile similar to other GH indications [1]. The mean height increased in response to GH therapy, there was significant variability in individual growth responses, including no measurable increase in height standard deviation scores (Ht SDSs) in some patients [9, 10]
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