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

• Published in: Clinical Pediatric Endocrinology Vol. 29; no. 4; pp. 159 - 171
• Main Authors: Reiko Horikawa, Toshiaki Tanaka, Hiromi Nishinaga, Yosuke Nishiba, Susumu Yokoya
• Format: Journal Article
• Language: Japanese
• Published: The Japanese Society for Pediatric Endocrinology 01.10.2020
• ISSN: 0918-5739; 1347-7358

[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 naive and 46 non-naive (previously treated) children were enrolled. GH treatment was well-tolerated, with SADRs occurring in 1.3% (6/452) and 0% (0/46) of naive and non-naive children, respectively. No new safety concerns or notable changes in glucose metabolism were identified during long-term treatment. Altogether, 57 children (32 naive and 25 non-naive) reached near adult height (NAH). In naive and non-naive 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 (naive) and 7.26 (non-naive) 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.