托法西提尼布治疗小儿性结肠炎:回顾性多中心体验
Oren Ledder1,2, Michael Dolinger3, Marla C Dubinsky3
1Juliet Keidan Institute of Paediatric Gastroenterology, Shaare Zedek Medical Center, Jerusalem, Israel.
Inflammatory bowel diseases
|June 3, 2024
概括
托法西提尼布在儿科性结肠炎 (UC) 患者中显示出有效性,其中16%的患者在第8周内实现了缓解. 副作用是轻微的,这表明这种重要治疗在儿童中耐火性UC的安全概况是有利的.
科学领域:
- 儿科胃肠病学 儿科胃肠病学
- 免疫学 免疫学 免疫学
- 药理学 药理学是指药理学的学科.
背景情况:
- 对于中度至重度性结肠炎 (UC) 中的托法西提尼布的儿科数据有限.
- 托法西提尼布已被批准用于成人UC治疗.
- 这项研究解决了对儿科有效性和安全性数据的需求.
研究的目的:
- 评估托法西提尼布在患有性结肠炎 (UC) 的儿科患者中的有效性和安全性.
- 在第8周和第24周确定无皮质类固醇临床缓解率.
- 在这个儿科队列中评估切除术率和不良事件.
主要方法:
- 来自16个国际中心的101名儿科UC患者的回顾性审查.
- 主要结局:8周无皮质类固醇的临床缓解 (儿童性结肠炎活动指数<10).
- 二次结局:临床反应,第24周缓解,切除肠道和不良事件.
主要成果:
- 16%的患者在第8周实现了无皮质类固醇缓解;23%的人在第24周实现了缓解.
- 30%的患者在第8周表现出临床反应.
- 25%的患者接受了小肠切除术;不良反应是轻微的,包括一种疹病例.
结论:
- 托法西替尼在高度耐火的儿科UC队列中表现出有效性,至少16%的患者在第8周内实现了缓解.
- 副作用是轻微的,与成人数据一致.
- 这项研究代表了最大的现实世界儿科队列评估托法西提尼布UC.
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