在2岁以下接受治疗的GH缺乏症儿童中,增长结果更好
Tilman Robert Rohrer1, Primož Kotnik2,3, Bradley S Miller4
1Division of Pediatric Endocrinology, Department of Pediatrics and Neonatology, University Children's Hospital, Saarland University Medical Center, Homburg, Germany.
Endocrine connections
|September 8, 2025
概括
在2岁之前开始对GH缺乏症 (GHD) 进行生长激素 (GH) 治疗可以改善身高结果,但会增加严重的不良事件. 早期诊断和监测对于年轻的GHD患者至关重要.
科学领域:
- 儿科内分泌学 儿科内分泌学
- 增长激素治疗疗法 增长激素治疗
- 临床结果研究研究
背景情况:
- 有限的数据存在于生长激素 (GH) 治疗响应在非常年幼的儿童与GH缺乏症 (GHD).
- 这项研究基于GH治疗开始时的年龄 (<2岁与≥2岁) 对比了GH儿童的临床结果.
研究的目的:
- 根据治疗开始时的年龄,评估儿童GHD患者GH治疗的疗效和安全性.
- 为了比较高度标准偏差得分 (SDS) 变化和早期 (<2年) 和晚期 (≥2年) 开始治疗之间的不良事件.
主要方法:
- 来自两个观察性研究 (NordiNet® IOS和ANSWER计划) 的数据汇总了接受Norditropin®的儿科患者的数据.
- 患有GHD的患者,在1年后仍然处于青春期前,根据治疗开始时的年龄分组.
- 主要结局:1岁和10岁时身高SDS变化;次要结局:药物不良反应 (ADR) 和严重不良事件 (SAE).
主要成果:
- 开始GH治疗2年以下的儿童 (n=507) 在1年 (1.4比0.75) 和10年 (3.2比2.2) 时,与≥2年 (n=7,486) 的儿童相比,显示出更大的高度SDS改善.
- 严重不良事件 (SAE) 在<2年组 (3.3%) 与≥2年组 (0.67%) 相比更频繁.
结论:
- 在2岁之前在患有GHD的儿童中开始GH治疗会导致优异的身高结果.
- 早期开始GH治疗与更高的SAE发病率有关,这强调了需要仔细监测的必要性.
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