开发和验证微观结肠炎的新型患者报告结果-微观结肠炎评分 (MCS)
Katarina Pihl Lesnovska1,2, Samuel Schäfer1,2,3, Yamile Zabana4,5
1Department of Gastroenterology and Hepatology, Linköping University Hospital, Linköping, Sweden.
新的微观结肠炎评分 (MCS) 和经过验证的微观结肠炎症状问卷 (MCSQ) 准确评估疾病的严重程度. 这些工具符合监管标准,并为微观结肠炎患者提供细微的评估.
科学领域:
- 胃肠病学 胃肠病学
- 临床试验 临床试验
- 患者报告的结果
背景情况:
- 微观性结肠炎 (MC) 缺乏标准化的疾病严重程度指数,这阻碍了对新疗法批准的监管标准的协调.
- 现有的评估差距阻碍了对FDA和EMA评估至关重要的患者报告结果措施 (PROM) 的使用.
- 这项研究解决了对验证工具的需求,以测量MC症状严重程度和对生活质量的影响.
研究的目的:
- 为了验证微观结肠炎症状问卷 (MCSQ) 评估MC症状.
- 开发和验证一种新的微观结肠炎评分 (MCS) 作为疾病严重程度指数.
- 确保开发的工具符合FDA和EMA在临床试验中的PROM标准.
主要方法:
- 一项前性多中心研究,涉及131名活检确认的微观结肠炎患者.
- 患者完成了MCSQ和与健康有关的生活质量 (HRQoL) 评估 (IBDQ-32,SHS).
- 集群分析和因子分析被用于分别开发和验证MCS和MCSQ.
主要成果:
- 该MCSQ表现出良好的内部一致性 (克朗巴赫α=0.88) 和测试复试可靠性 (ICC=0.88).
- 新型微观结肠炎评分 (MCS) 范围在0-15之间,与HRQoL指标有很强的相关性 (例如,IBDQ-32总分数的rp=-0.78).
- 受体运行特征曲线显示MCS准确识别疾病状态,包括缓解 (AUC=0.85) 和不同严重程度 (AUCs 0.93-0.97).
结论:
- MCSQ和MCS是评估微观结肠炎的有效,可靠和响应的工具.
- 这些仪器符合FDA和EMA对PROM的标准,反映了多种MC症状.
- MCS提供了对疾病活性的细微评估,为未来的治疗疗效试验提供了希望.
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