对于妊娠年龄来说出生小的儿童的somapacitan:一个随机对照的第三阶段试验
Agnès Linglart1, Volker Böttcher2, Michael Højby3
1Université Paris-Saclay, Inserm, AP-HP, Service d'Endocrinologie et dabète de l'enfant, Hôpital Bicêtre Paris Saclay, Le Kremlin-Bicêtre F-94270, France.
European journal of endocrinology
|March 5, 2026
概括
每周服用一次索马卡西坦的疗效和安全性与每日注射生长激素 (GH) 相同,用于治疗妊娠年龄 (SGA) 较小的婴儿的矮身. 这种长效的GH选择可能会减少治疗负担并改善坚持.
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
- 代谢障碍 代谢障碍 代谢障碍
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,需要生长激素 (GH) 治疗.
- 目前的治疗包括每天注射重组GH,这对患者和家人造成了重大负担.
- 探索长效GH配方的目的是改善治疗坚持和结果.
研究的目的:
- 评估每周服用一次长期作用的GH - - 索马卡西坦的疗效和安全性,用于SGA.出生于青春期前的儿童.
- 为了比较索马帕西坦的高度速度和安全性,与每日GH疗法.
- 评估索马卡西坦在此儿科群体中降低治疗负担的潜力.
主要方法:
- 一个跨国,随机的,开放的,第三阶段试验 (REAL8) 涉及142个未经治疗的SGA出生儿童.
- 参与者每周接受一次索马卡西坦或每天GH (0.035或0.067毫克/公斤/天) 的皮下注射,持续52周.
- 主要终点是52周的身高速度,并进行了非劣势和优势分析.
主要成果:
- 索马卡西坦实现了平均高度速度为11.0厘米/年,相当于每天的GH (9.4和11.1厘米/年).
- 索马卡西坦对每日两剂GH的非劣质性得到证实.
- 索马帕西坦在较低的每日GH剂量 (0.035毫克/公斤/天) 上显示出优越性,并且在各组中显示出类似的安全性.
结论:
- 每周服用一次的索马卡西坦是有效和安全的,用于治疗52周以上SGA出生儿童的矮身.
- 索马卡西坦的疗效与每日GH相当,但治疗负担降低.
- 这种长效的配方为每日注射提供了一个有希望的替代方案,可能会提高患者的坚持和治疗成功.
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