Kappelgaard AM et al, endocrinology, University Hospital Munich, Germany, conducted a prospective, open, non-comparative, multicenter, long-term phase IV clinical study to validate the efficacy and safety of recombinant human growth hormone (rhGH) in the treatment of small for gestational age at birth (SGA) children with short stature, while observing the effect on the incidence of diabetes. This article is a one-year interim data analysis of the study.
A total of 278 prepubertal SGA patients were included in the study. Inclusion criteria included: height standard deviation score (HSDS) <-2.5, parent-corrected SDS <-1, birth weight and/or length <-2SD, and demonstrated failure to reach normal height standards at ≥4 years of age. The rhGH dose for all patients was 0.035 mg/kg/day. the primary endpoint of the study was the effect of long-term rhGH treatment on carbohydrate metabolism, including fasting glucose, stimulated glucose (2-hour oral glucose tolerance test, OGTT), and glycated hemoglobin (HbA1c). Secondary endpoints included: height parameters (height, HSDS, growth rate [HV], HVSDS), insulin growth factor female 1 (IGF-I) and insulin-like growth factor binding protein-3) during treatment, and the incidence and severity of adverse events. The results showed that no patient developed diabetes during the first year of treatment. No significant fluctuations in the mean fasting glucose and HbA1ct 2-h OGTT were observed during the course of treatment. All height parameters improved significantly. Mean HSDS increased from baseline indeed 3.39 to -2.57 and mean HV increased from baseline 4.25 cm/year to 8.99 cm/year. Mean HVSDS increased from 2.13 at baseline to +4.16. Mean IGF-I SDS and IGFBP-3 SDS also increased, at +1.80 and +0.41, respectively. 13 patients (4.7%) did not respond adequately to treatment (HVSDS <1) and were excluded from the study. Adverse reactions occurred in 192 patients (69.3%), most of them (98.7%) were mild to moderate, and the majority (96.5%) were not related to drug administration. The results of this interim study suggest that recombinant human growth hormone is safe and effective in the treatment of short stature in patients born younger than gestational age and has no adverse effects on glucose metabolism.