两年的实践经验,在儿童和青少年中使用生长激素缺乏症的索马特罗贡
Gianluca Tamaro1,2, Chiara Rodaro2, Alice Fachin2
1Institute for Maternal and Child Health IRCCS "Burlo Garofolo", Trieste, Italy.
Frontiers in endocrinology
|February 13, 2026
概括
每周的索马特龙有效地支持患有生长激素缺乏症 (GHD) 的儿科患者的生长,改善了坚持. 现实世界的数据显示持续的身高增长,尽管初步结果与临床试验相比有所不同.
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.
- 临床研究 临床研究
背景情况:
- 儿童的生长激素缺乏症 (GHD) 需要长期的复合人体生长激素 (rhGH) 治疗.
- 每日注射的rhGH会带来坚持性挑战,导致长效配方 (LAGH) 的需求.
- 每周一次的索马特罗贡是一种LAGH选择,但对其使用的真实数据有限.
研究的目的:
- 评估GHD的儿科患者每周服用一次索马特罗贡的真实安全性和疗效.
- 为了比较从未接受过治疗的患者和从每日RhGH疗法转换的患者的结果.
主要方法:
- 在意大利第三级内分泌中心对儿科GHD患者进行索马特罗贡启动的回顾性研究.
- 纳入标准:在2023年3月至2025年1月期间开始治疗,至少接受6个月的治疗.
- 收集了关于身高SDS,IGF-1SDS,坚持和不良事件的数据.
主要成果:
- 分析了40名患者 (50%原始,50%切换).
- 在6个月后,在两组中都观察到显著的身高SDS增加;以前的患者在18-24个月后显示出更大的增长.
- IGF-1 SDS仅在先天患者中显著增加;治疗耐受性良好,报告有改善的坚持.
结论:
- 在现实世界中,每周一次的索马特罗贡对儿科GHD是安全有效的.
- 虽然身高增长低于临床试验,但每周给药对坚持和生活质量有实际好处.
- 这项研究提供了关于儿科GHD. somatrogon的第一个真实世界队列数据.
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