一种新的Y形基化复合人体生长激素用于生长激素缺乏的儿童:一项第三阶段研究
Yan Liang1, Haiyan Wei2, Fan Yang3
1Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
The Journal of clinical endocrinology and metabolism
|November 28, 2024
概括
每周Pegpesen® (pegylated复合人体生长激素) 在治疗儿科生长激素缺乏症时,表现为每天的rhGH的非劣势. 这种新的治疗方法在儿童中提供了可比的安全性概况和降低的剂量要求.
科学领域:
- 儿科内分泌学 儿科内分泌学
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
背景情况:
- 佩佩森 (Pegpesen®) 是一种新型Y形化复合人体生长激素 (rhGH),每周治疗一次.
- 生长激素缺乏 (GHD) 影响儿童的生长和发育.
研究的目的:
- 为了评估每周Pegpesen®的疗效和安全性,与每天的rhGH相比,在患有GHD的儿童中.
- 建立Pegpesen®作为一个可行的替代治疗选择.
主要方法:
- 这是一项为期52周的多中心随机对照的第三期临床试验.
- 391名患有GHD的儿科参与者被随机分为2:1获得每周Pegpesen®或每天的rhGH.
- 主要终点:52周生长速度 (GV);次要终点:身高标准偏差得分的变化.
主要成果:
- 每周Pegpesen®在实现GV方面与每日rhGH不逊色 (9.910厘米/年与10.037厘米/年).
- 在不同组的高度标准偏差得分 (ΔHt SDS CA和ΔHt SDS BA) 中没有发现显著差异.
- 对Pegpesen®和每天的rhGH报告了可比的安全性概况和治疗坚持.
结论:
- 与每日的rhGH相比,Pegpesen®为儿科GHD提供了一个非劣质的治疗选择.
- 该研究支持Pegpesen®作为一种安全有效的替代品,其剂量要求可能更低.
- 佩佩森 (Pegpesen®) 提出了一种新的治疗途径,用于治疗儿童的GHD.
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