The long-term safety and effectiveness of growth hormone treatment in Japanese children with short stature born small for gestational age

摘要

Abstract. This study aimed to characterize the safety and effectiveness of GH treatments, in usual clinical practice, in children with short stature born small for gestational age (SGA). This was a multicenter, open-label, non-interventional study ( NCT01110928 ) conducted at 150 sites in Japan (2009–2018). The primary objective was to assess the type and frequency of serious adverse drug reactions (SADRs) associated with long-term GH use. Overall, 452 na?ve and 46 non-na?ve (previously treated) children were enrolled. GH treatment was well?tolerated, with SADRs occurring in 1.3% (6/452) and 0% (0/46) of na?ve and non-na?ve children, respectively. No new safety concerns or notable changes in glucose metabolism were identified during long-term treatment. Altogether, 57 children (32 na?ve and 25 non-na?ve) reached near adult height (NAH). In na?ve and non-na?ve children, mean ± standard deviation (SD) height standard deviation score (SDS) at NAH were –2.03 ± 0.77 and –1.53 ± 0.81, respectively, representing a change of +0.85 ± 0.72 and +1.24 ± 0.66 from baseline height SDS, respectively. Mean treatment duration to NAH was 4.29 (na?ve) and 7.26 (non-na?ve) yr. Thus, long-term GH treatment for short stature in children born SGA was confirmed to have a good safety profile and was effective for improving adult height.