对于妊娠年龄来说出生小的儿童的somapacitan:从第二阶段的4年结果
Anders Juul1,2, Michael Højby3, Masanobu Kawai4
1Department of Growth and Reproduction, Copenhagen University Hospital-Rigshospitalet, Copenhagen 2100, Denmark.
European journal of endocrinology
|January 30, 2026
概括
每周一次的索马卡西坦在四年内对妊娠年龄 (SGA) 较小而身高矮的儿童中显示出持续的疗效和安全性. 与每日生长激素 (GH) 治疗相比,索马卡西坦改善了患者的偏好和坚持.
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
- 药理学 药理学是指药理学的学科.
背景情况:
- 对于妊娠年龄 (SGA) 来说出生小的儿童经常经历矮身.
- 增长激素 (GH) 治疗是一种标准的治疗方法,但每日注射可以影响坚持和生活质量.
- 长效GH配方旨在提高治疗的方便性和结果.
研究的目的:
- 评估每周服用一次索马卡西坦的长期疗效,安全性和耐受性,用于SGA出生小身高的儿童.
- 评估从每日GH切换到somapacitan后的结果.
- 为了比较患者的偏好和somapacitan和每日GH之间的坚持.
主要方法:
- 一项全球性,随机的,开放的,受控的第二阶段研究 (REAL5),涉及62名出生于SGA的儿童.
- 参与者每周服用一次索马卡西坦或每天服用GH,长达208周.
- 分析包括身高标准偏差得分,安全评估和患者偏好调查问卷.
主要成果:
- 从第52周到第208周观察到持续的身高标准偏差得分增加,包括转换为somapacitan的患者.
- 索马卡西坦的安全性和耐受性与每日GH相当.
- 显著的大多数患者更喜欢索马卡西坦 (87%) 和预期更好的坚持 (80%).
结论:
- 每周一次的索马卡西坦在出生于SGA的儿童中提供长期的持续疗效,安全性和耐受性.
- 索马帕西坦为每日GH提供了一个可行的替代品,改善了患者的偏好和坚持.
- 这些发现支持在这个儿科群体中使用多达四年的索马卡西坦.
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