Adult height and health-related quality of life after growth hormone therapy in small for gestational age subjects

OBJECTIVE: To estimate health-related quality of life (HRQoL) in non-growth hormone deficient (GHD) small for gestational age (SGA) children before and after growth hormone (GH) treatment to adult height (AH). METHODS: This was a multicentre, two-arm trial. Following an initial 2-year double-blind study period, patients entered a 2-year extension period followed by treatment to AH. At baseline patients were randomised to GH (0.033 or 0.067 mg/kg/day) and continued treatment at that dose until AH. Height was assessed at baseline and 3-monthly intervals to AH (height velocity <2 cm/year). Height standard deviation score (SDS) before and after GH therapy was mapped onto estimated HRQoL scores up to AH. RESULTS: Of the 79 children randomised into the study 53 were non-GHD (defined as peak GH >20 mU/L [peak 24-h GH value and peak arginine tolerance test]). At baseline these children had a mean (mean [+/-SD]) height SDS of -3.2 (0.7), height velocity SDS -0.6 (1.2) and age, 8.1 (1.9) years. Estimated HRQoL scores were significantly (p < 0.001) increased from baseline at AH (ΔHRQoL, 95% CI) (0.033 mg/kg/day, 0.112 [0.092, 0.132]; 0.067 mg/kg/day, 0.115 [0.094, 0.136]). HRQoL was not different between treatment groups. A significant gain in AH, relative to an SGA reference population, was reported in GH-treated patients. Mean (95% CI) ΔAH SDS (0.033 mg/kg/day, +1.4 [1.1, 1.6]. 0.067 mg/kg/day, +1.7[1.4, 2.0]). LIMITATIONS: The analysis assumes HRQoL can be mapped onto height SDS. CONCLUSIONS: GH treatment in short children born SGA without signs of persistent catch-up growth was associated with significant improvement in HRQoL and normalisation of AH.