Adult height of prepubertal short children born small for gestational age treated with GH

Abstract

Objective: Human GH (hGH) treatment leads to catch-up growth in children with short stature born small for gestational age (SGA). However, long-term efficacy and safety results in this patient group remain scarce. The present study assessed the efficacy and safety of late childhood treatment with biosynthetic hGH (Humatrope) in a group of short children born SGA (height ,22 standard deviation scores (SDS)). Design:Patients in this open-label, Phase III, multicenter study received a daily hGH dose of 0.067 mg/kg for 2 years, and then received no treatment for the following 2 years. After the fourth year on study, patients whose height had decreased more than 0.5 SDS but who still showed growth potential based on bone age were allowed to resume treatment until they reached adult height. Methods: Height gain SDS was assessed for 11 girls and 24 boys (mean age^S.D. 9.6^0.9 years) at the end of the 2 years of hGH treatment, during the subsequent 2-year off-treatment period, and upon reaching adult height. Results: At the end of the initial 2-year treatment period, 83% of patients had reached a height within the normal range, with a mean increase in height SDS vs baseline of 1.3^0.3 (P , 0.001). Adult heights (n ¼ 20) were within the normal range for 50% of patients, and mean height gain from baseline was statistically significant (0.7^0.8 SDS, P , 0.001). Fasting glucose and glycosylated hemoglobin levels were not significantly modified during treatment. Conclusions: High-dose hGH treatment for a minimum of 2 years in short children born SGA was well tolerated and resulted in a significant increase in adolescent and adult height.European Journal of Endocrinology 152 835-843

Height at Start, First-Year Growth Response and Cause of Shortness at Birth Are Major Determinants of Adult Height Outcomes of Short Children Born Small for Gestational Age and Silver-Russell Syndrome Treated with Growth Hormone: Analysis of Data from KIGS

“…So far only a few studies on SGA have documented the growth response to GH up to AH [5,6,26,27,28,29,30,31,32]. In comparison, the characteristics of KIGS patients in the present analysis, who were followed to near AH, do not show characteristics which would suggest a reporting bias.…”

Observed and Predicted Total Pubertal Growth during Treatment with Growth Hormone in Adolescents with Idiopathic Growth Hormone Deficiency, Turner Syndrome, Short Stature, Born Small for Gestational Age and Idiopathic Short Stature: KIGS Analysis and Review

“…Growth analyses of TPG from Lazar et al [39] and Vicens-Calvet et al [40] suggest that TPG in SGA is normal for age at puberty onset and sex. During long-term GH therapy TPG was documented in the studies of Rosilio et al [41] and Boonstra et al [42]. In the latter study doubling of the GH dose from 1 to 2 mg/m 2 /day resulted in a mean gain of TPG of 4.4 cm in boys, while no effect was observed in girls.…”

European Multicentre Study in Children Born Small for Gestational Age with Persistent Short Stature: Comparison of Continuous and Discontinuous Growth Hormone Treatment Regimens

“…This was, however, of limited magnitude compared to the overall height gain during the study. Other authors have reported a reduction in height velocity [13] or mean loss in the height SDS [11, 17] in SGA children after stopping GH treatment. Interestingly, the mean gain in overall height in our study was not different for patients in the mid-dose GH group who started GH after 1 year and the group on the continuous low-dose regimen.…”

“…In a small cohort of short-statured SGA children, preliminary data showed that a similar gain in height was achieved with the use of high-dose GH (0.100 mg/kg/day) for a period of 2 years as compared with continuous low-dose GH for up to 6 years [15]. However, a marked catch-down in growth has been reported following discontinuation of GH treatment, especially during the first off-treatment year [11, 16, 17]. …”