Long-term safety and efficacy of daily recombinant human growth hormone treatment in Japanese short children born small for gestational age: final report from an open and multi-center study

Our study aimed at evaluating the safety and efficacy of GH treatment up to near adult height (NAH) for short children born small for gestational age (SGA). This was a multi-center, open-label, long-term extension study after a one-year, randomized, open-label, dose-response study. The primary objective was to assess safety, determined by adverse events and laboratory test parameters. Height parameters were evaluated as a secondary objective. The final data after all patients completed the study were reported. Overall, 61 patients were enrolled in the study. GH treatment was well tolerated. No notable changes in HbA1c levels, oral glucose tolerance tests and glucose metabolism were observed. No new safety concerns related to long-term treatment up to NAH were identified. Twenty patients (11 boys and 9 girls) reached NAH with a mean height of 159.1 cm and 146.9 cm, respectively. The mean change in height SDS from baseline to NAH was +1.9 in boys and +1.8 in girls. Long-term GH treatment for SGA short stature was confirmed to be safe and effective for the normalization of adult height.