对于生长激素缺乏儿童的生长激素治疗报告结果的趋势
Xanthippi Tseretopoulou1,2, Jiajia Chen3, Angela Lucas-Herald1,2
1Developmental Endocrinology Research Group, University of Glasgow, Royal Hospital for Children, Glasgow, UK.
The Journal of clinical endocrinology and metabolism
|September 5, 2025
概括
本综述分析了儿童的生长激素治疗结果. 最常见的是身高和安全性指标,这表明需要为生长激素缺乏症 (GHD) 治疗制定标准化的核心结果.
科学领域:
- 儿童内分泌学
- 药物监督管理
- 临床试验方法
背景情况:
- 儿童生长激素缺乏症 (GHD) 是一种严重的内分泌疾病.
- 重组人体生长激素 (rhGH) 治疗是儿童群体中GHD的主要治疗方法.
- 了解报告结果的范围对于优化rhGH治疗至关重要.
研究的目的:
- 系统地回顾GHD儿童治疗的安全性和有效性结果的频率和趋势.
- 确定 rhGH 治疗研究中最常被评估的参数.
- 为儿童GHD制定标准化的核心结果.
主要方法:
- 在2003年至2022年间在英语和中文数据库中发表的研究的系统审查.
- 纳入标准:参与者在16岁之前开始使用rhGH.
- 数据提取集中在报告的结果,研究设计和参与者人口统计学上.
主要成果:
- 包括219个研究 (171个队列,39个受控试验,9个病例对照).
- 最常见的结果:身高SDS (53%),身高速度 (48%),IGF-1 (30%) 和注射部位的不良反应 (20%).
- 超过50%的结局侧重于安全性,有效性结局也经常报告;生活质量结局是罕见的 (3%).
结论:
- 在过去二十年中,高度SDS和高度速度一直是最常见的疗效结果.
- 报告的结果中很大一部分与安全性有关,包括不良事件和生化标志物.
- 这些发现支持开发和实施一套核心结果,用于对患有GHD的儿童进行一致的评估.
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