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对抗性炎症性肠病青少年的乌帕达西尼布的单中心经验
Elizabeth A Spencer1, Suzannah Bergstein1, Michael Dolinger1
1Division of Pediatric Gastroenterology and Nutrition, Department of Pediatrics Mount Sinai, Icahn School of Medicine, 17 E. 102nd Street, Fifth Floor, New York, NY, 10029, USA.
乌帕达西丁 (UPA) 在儿科炎症性肠病 (IBD) 中表现有前途. 这项研究发现UPA在IBD青少年中有效实现无类固醇缓解,具有良好的安全性.
科学领域:
- 胃肠病学 胃肠病学
- 儿科胃肠病学 儿科胃肠病学
- 药理学 药理学是指药理学的学科.
背景情况:
- 乌帕达西丁 (UPA) 是一种被批准用于成人性结肠炎 (UC) 和克罗恩病 (CD) 的选择性JAK抑制剂.
- 儿科IBD患者经常使用UPA非标签,因为批准延迟.
- 需要关于UPA在儿科IBD的安全性和有效性的真实数据.
研究的目的:
- 评估乌帕达西替尼 (UPA) 在患有炎症性肠病 (IBD) 的青少年的安全性和有效性.
- 评估诱导后无类固醇临床缓解率 (SF-CR) 和其他疗效结局.
- 在这个儿科群体中记录不良事件.
主要方法:
- 在接受UPA治疗的IBD青少年 (12-17岁) 的回顾性病例系列.
- 主要结局:诱导后的SF-CR (PUCAI/PCDAI ≤10).主要结局:诱导后的SF-CR (PUCAI/PCDAI ≤10).
- 二次结局:临床反应,CRP正常化,6个月SF-CR,超声响应/缓解和不良事件.
主要成果:
- 75%的患者在第12周实现了SF-CR,其中80%实现了CRP正常化.
- 大约75%的人在6个月内实现了SF-CR.
- 不良事件很少发生 (10%),包括细胞巨乳病毒结肠炎和高脂血症.
结论:
- 乌帕达西丁 (UPA) 在诱导和维持高度耐火性IBD的青少年无类固醇临床缓解方面似乎有效.
- 在这个儿科队列中,UPA的安全性概况是可以接受的.
- 还在等待儿科注册试验的进一步数据.
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