Supplementary Material for: Growth Hormone Treatment for Short Stature in the USA, Germany and France: 15 Years of Surveillance in the Genetics and Neuroendocrinology of Short-Stature International Study (GeNeSIS)

<b><i>Background/Aims:</i></b> To describe characteristics, auxological outcomes and safety in paediatric patients with growth disorders treated with growth hormone (GH), for cohorts from the USA, Germany and France enrolled in GeNeSIS, a post-authorisation surveillance programme. <b><i>Methods:</i></b> Diagnosis and biochemical measurement data were based on reporting from, and GH treatment was initiated at the discretion of, treating physicians. Auxological outcomes during the first 4 years of GH treatment and at near-adult height (NAH) were analysed. Serious and treatment-emergent adverse events were described. <b><i>Results:</i></b> Children in the USA (<i>n</i> = 9,810), Germany (<i>n</i> = 2,682) and France (<i>n</i> = 1,667) received GH (dose varied between countries), most commonly for GH deficiency. Across diagnostic groups and countries, mean height velocity standard deviation score (SDS) was &gt; 0 and height SDS increased from baseline during the first 4 years of treatment, with greatest improvements during year 1. Most children achieved NAH within the normal range (height SDS &gt;−2). No new or unexpected safety concerns were noted. <b><i>Conclusion:</i></b> GH treatment improved growth indices to a similar extent for patients in all three countries despite variations in GH doses. Data from these three countries, which together contributed &gt; 60% of patients to GeNeSIS, indicated no new safety signals and the benefit-risk profile of GH remains unchanged.

背景与目的：针对纳入上市后监测项目GeNeSIS的美国、德国与法国队列，本研究旨在描述接受生长激素（growth hormone，GH）治疗的生长障碍儿科患者的特征、生长学转归与安全性情况。
方法：诊断与生化检测数据均源自经治医师上报，GH治疗方案的启动则由经治医师自行决定。本研究分析了GH治疗前4年期间以及接近成人身高（near-adult height，NAH）阶段的生长学转归情况，并对严重不良事件与治疗期间出现的不良事件进行了描述。
结果：美国（n=9810）、德国（n=2682）与法国（n=1667）的儿童均接受了GH治疗，各国给药剂量存在差异，其中最常见的适应证为生长激素缺乏症。在所有诊断分组与国家中，身高增长速度标准差评分（height velocity standard deviation score，SDS）均值均大于0，且治疗前4年期间身高标准差评分较基线水平有所提升，其中以治疗第1年的改善幅度最为显著。多数患儿的接近成人身高处于正常范围（身高SDS＞−2），未观察到新增或非预期的安全性相关问题。
结论：尽管各国GH给药剂量存在差异，但GH治疗对三个国家患者生长指标的改善程度相近。上述三个国家的患者占GeNeSIS项目总入组患者的60%以上，其数据显示未出现新的安全性信号，GH治疗的获益-风险比未发生改变。