Growth Hormone (GH) and its related Factors Study Committee and GH Treatment Study Committee, The Foundation for Growth Science in Japan, Tokyo, Japan.
Department of Pediatrics, Teikyo University School of Medicine, Tokyo, Japan.
Endocr J. 2022 Aug 29;69(8):927-939. doi: 10.1507/endocrj.EJ21-0520. Epub 2022 Mar 3.
Growth hormone (GH) deficiency (GHD) in children is a heterogeneous condition that includes several entities of various severities. GH treatment has been affected by various factors. Because comprehensive analyses for Japanese children with GHD over time are scarce, we investigated the baseline characteristics of patients with GHD at the start of GH treatment between 1996 and 2015 using data from the Foundation for Growth Science in Japan. During the registration period, 19,717 subjects were determined to be eligible for GH treatment as GHD. Overall analyses revealed that there were twice the number of male patients as female patients, and the etiology was idiopathic in 91.1%, central nervous system (CNS) tumor at the hypothalamus-pituitary area in 1.7%, CNS tumor distant from the hypothalamus-pituitary area in 0.68%, other tumors in 0.91%, congenital CNS malformations in 0.83%, and other diseases in 1.1% with their specific characteristics. The latest average age, height standard deviation score (SDS), insulin-like growth factor-1 SDS, and proportion of severe GHD at GH treatment initiation were 8.8 years, -2.76, -1.42, and 19.5%, respectively. The proportions of breech delivery and asphyxia gradually decreased, whereas that of caesarean section gradually increased during the registration period with the latest values of 2.2%, 4.9%, and 14.0%, respectively (all analyses: p < 0.0001). In contrast, the proportion of idiopathic GHD with breech delivery seemed to reach the lowest level among those with a birth year before 2000. This study identified the characteristics and changes of patients with GHD over 20 years.
儿童生长激素缺乏症（GHD）是一种异质性疾病，包括多种严重程度不同的疾病。GH 治疗受到多种因素的影响。由于缺乏对日本 GHD 儿童随时间变化的综合分析，我们使用日本生长科学基金会的数据，对 1996 年至 2015 年期间开始 GH 治疗的 GHD 患者的基线特征进行了研究。在登记期间，有 19717 名患者被确定为符合 GH 治疗标准的 GHD。总体分析显示，男性患者是女性患者的两倍，病因中特发性占 91.1%，下丘脑-垂体区中枢神经系统（CNS）肿瘤占 1.7%，远离下丘脑-垂体区的 CNS 肿瘤占 0.68%，其他肿瘤占 0.91%，先天性 CNS 畸形占 0.83%，其他疾病占 1.1%，各有其特点。最新的平均年龄、身高标准差评分（SDS）、胰岛素样生长因子-1 SDS 和严重 GHD 患者在开始 GH 治疗时的比例分别为 8.8 岁、-2.76、-1.42 和 19.5%。在登记期间，臀位分娩和窒息的比例逐渐下降，而剖宫产的比例逐渐增加，最新值分别为 2.2%、4.9%和 14.0%（所有分析：p < 0.0001）。相反，臀位分娩的特发性 GHD 比例似乎在 2000 年以前出生的患者中达到最低水平。本研究确定了 20 多年来 GHD 患者的特征和变化。
