Thalidomide Therapy for Very Early Onset Inflammatory Bowel Disease
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- endoscopic response rate
研究概览
简要总结
This is a single center, observational study to investigate the clinical outcomes of thalidomide treatment for very early onset inflammatory bowel disease
详细描述
Very early onset inflammatory bowel disease (VEOIBD) refers to IBD diagnosed before 6 years of age. VEOIBD comprises a distinct subset of pediatric IBD characterized by stronger genetic predisposition, predominant colonic involvement, more extensive inflammation, more severe course, and peculiar response to treatments. Previous studies have been demonstrated that thalidomide, an oral molecule with immunomodulatory, antiangiogenic, and TNF-suppressing properties, is an effective and safe treatment for adults and children with IBD refractory to conventional therapies. This study is aimed to evaluate the efficacy and tolerance of thalidomide in VEOIBD.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 28 Days 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diagnosed with inflammatory bowel disease
- •disease onset less than 6 years old
- •Pediatric Crohn's disease Activity Index (PCDAI)>30 or Pediatric Ulcerative Colitis Activity Index (PUCAI)>35
- •patients and their legal guardians were willing to receive thalidomide treatment and participate in this study.
排除标准
- •liver dysfunction
- •allergy to thalidomide
- •with Neuropathy
- •with thrombosis
- •thalidomide treatment in the previous 30 days
- •biologics treatment in the previous 8 weeks
- •not suitable participated in this study
结局指标
主要结局
endoscopic response rate
时间窗: week 52
The Simple Endoscopic Score for Crohn Disease decreased 50% compared with baseline OR Mayo score 0-1 (higher scores mean a worse outcome)
次要结局
- growth development(week52)
- adverse event(within one year)
- biomarker remission rate(week8, week26, week52)
- clinical response rate(week8, week26, week52)
- treatment persistence(within one year)
- clinical remission rate with steroid-free(week26)
研究者
Ying HUANG
Head of gastroenterology department
Children's Hospital of Fudan University