A 2-year multicentre, open-label, randomized, controlled study of growth hormone (Genotropin®) treatment in very young children born small for gestational age: Early Growth and Neurodevelopment (EGN) Study

ObjectiveIn Europe, growth hormone (GH) treatment for children born small for gestational age (SGA) can only be initiated after 4 years of age. However, younger age at treatment initiation is a predictor of favourable response. To assess the effect of GH treatment on early growth and cognitive functioning in very young (<30 months), short-stature children born SGA. DesignA 2-year, randomized controlled, multicentre study (NCT00627523; EGN study), in which patients received either GH treatment or no treatment for 24 months. PatientsChildren aged 19-29 months diagnosed as SGA at birth, and for whom sufficient early growth data were available, were eligible. Patients were randomized (1:1) to GH treatment (Genotropin((R)), Pfizer Inc.) at a dose of 0035 mg/kg/day by subcutaneous injection, or no treatment. MeasurementsThe primary objective was to assess the change from baseline in height standard deviation score (SDS) after 24 months of GH treatment. ResultsChange from baseline in height SDS was significantly greater in the GH treatment vs control group at both month 12 (103 vs 014) and month 24 (163 vs 043; both P < 0001). Growth velocity SDS was significantly higher in the GH treatment vs control group at 12 months (P < 0001), but not at 24 months. There was no significant difference in mental or psychomotor development indices between the two groups. ConclusionsGH treatment for 24 months in very young short-stature children born SGA resulted in a significant increase in height SDS compared with no treatment.