Yokoya Susumu, Hasegawa Tomonobu, Ozono Keiichi, Tanaka Hiroyuki, Kanzaki Susumu, Tanaka Toshiaki, Chihara Kazuo, Jia Nan, Child Christopher J, Ihara Katsuichiro, Funai Jumpei, Iwamoto Noriyuki, Seino Yoshiki
Department of Medical Subspecialties, National Center for Child Health and Development, Tokyo, Japan.
Department of Pediatrics, School of Medicine, Keio University, Tokyo, Japan.
Clin Pediatr Endocrinol. 2017;26(4):229-241. doi: 10.1297/cpe.26.229. Epub 2017 Sep 28.
The primary goal of the Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS) was to assess the safety and effectiveness of Humatrope, a GH preparation, in the treatment of pediatric patients with short stature. We report our findings in the GH-treated Japanese pediatric population focusing on the incidence of type 2 diabetes (T2D) and occurrence of neoplasms. A total of 2,345 Japanese patients were assessed for safety. During a mean observation period of 3.2 yr, T2D occurred in 3 patients (0.13%) and slowly progressive insulin-dependent diabetes mellitus (SPIDDM) related to underlying mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS) in 1 patient (0.04%). Neoplasms were reported in 13 patients (0.56%), including 1 patient with brain tumor (germinoma) and 5 with craniopharyngiomas (4 recurrences); the remainder were benign, typically dermatological, neoplasms. The incidence of diabetes mellitus determined in the study did not differ from previous reports in GH-treated pediatric patients, and there was no apparent increase in the risk of new neoplastic lesions or malignant tumors.
矮小症国际研究（GeNeSIS）中的遗传学与神经内分泌学的主要目标是评估生长激素制剂Humatrope治疗矮小症儿科患者的安全性和有效性。我们报告了在接受生长激素治疗的日本儿科人群中的研究结果，重点关注2型糖尿病（T2D）的发病率和肿瘤的发生情况。共有2345名日本患者接受了安全性评估。在平均3.2年的观察期内，3名患者（0.13%）发生了T2D，1名患者（0.04%）发生了与潜在的线粒体脑肌病、乳酸酸中毒和卒中样发作（MELAS）相关的缓慢进展性胰岛素依赖型糖尿病（SPIDDM）。13名患者（0.56%）报告患有肿瘤，其中1名患者患有脑肿瘤（生殖细胞瘤），5名患有颅咽管瘤（4例复发）；其余为良性肿瘤，通常为皮肤肿瘤。该研究中确定的糖尿病发病率与先前关于生长激素治疗儿科患者的报告没有差异，并且新的肿瘤性病变或恶性肿瘤的风险没有明显增加。
