Tanaka Toshiaki, Yokoya Susumu, Hoshino Yuko, Hiro Shintaro, Ohki Nobuhiko
Tanaka Growth Clinic, Tokyo, Japan.
Thyroid and Endocrine Center, Fukushima Medical University, Fukushima, Japan.
Clin Pediatr Endocrinol. 2018;27(3):145-157. doi: 10.1297/cpe.27.145. Epub 2018 Jul 31.
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.
我们的研究旨在评估生长激素（GH）治疗对小于胎龄儿（SGA）出生的矮小儿童直至接近成人身高（NAH）的安全性和有效性。这是一项在为期一年的随机、开放标签、剂量反应研究之后进行的多中心、开放标签、长期扩展研究。主要目标是评估由不良事件和实验室检测参数所确定的安全性。身高参数作为次要目标进行评估。报告了所有患者完成研究后的最终数据。总体而言，61名患者纳入了该研究。生长激素治疗耐受性良好。未观察到糖化血红蛋白（HbA1c）水平、口服葡萄糖耐量试验和葡萄糖代谢有显著变化。未发现与直至NAH的长期治疗相关的新的安全问题。20名患者（11名男孩和9名女孩）达到了NAH，男孩的平均身高为159.1厘米，女孩为146.9厘米。从基线到NAH身高标准差评分（SDS）的平均变化在男孩中为+1.9，在女孩中为+1.8。生长激素长期治疗SGA矮小身材被证实对于成人身高正常化是安全有效的。
