描述自我报告的每日日记表测量在遗传性出血性长长管病的特征
C M Tarulli1, X Ma1, A M Bayoumi2,3,4,5,6,7
1Toronto HHT Centre, Division of Respirology, Department of Medicine St. Michael's Hospital Toronto Ontario Canada.
Laryngoscope investigative otolaryngology
|March 2, 2026
概括
每日鼻血日记准确地追踪HHT患者的表症严重程度. 患者报告的累积出血结果 (PRO-CB) 测量是临床试验的宝贵工具,尽管个体差异很大.
科学领域:
- 医学研究 医学研究
- 临床试验 临床试验
- 患者报告的结果
背景情况:
- 遗传性出血性长膜炎 (HHT) 是一种遗传性疾病,其特征是经常流鼻血 (表).
- 精确测量出血严重程度对于评估临床试验中治疗疗效至关重要.
- 现有的评估表症严重程度的方法可能缺乏精度或以患者为中心.
研究的目的:
- 为了验证一项新的临床试验结果测量,患者报告的累积出血结果 (PRO-CB),来自每日鼻血日记.
- 作为次要结果,评估每周累计喷出血持续时间 (PRO-CGB).
- 为了评估患者回忆的准确性,与日常日记条目相比,对表症严重性评分 (ESS) 项目进行评估.
主要方法:
- 一项前性观察性研究,涉及23名HHT患者,持续3个月.
- 参与者每天都会写日记,记录鼻血的发作,持续时间和强度 (鼻血与鼻血无关).
- 使用线性混合效应模型,相关性测试和部分自相关性分析分析的数据.
主要成果:
- 观察到高日记遵守率 (98.8%).
- 平均每周的PRO-CB为71.2分钟;喷的情节比非喷的情节要长得多.
- PRO-CB和ESS总分数没有显著的相关性,但ESS项目1和2与日记数据有很强的相关性.
结论:
- 每日鼻血日记是一个可行的,准确的方法来跟踪HHT中的表症严重程度.
- 在临床试验中,PRO-CB展示了作为一个有意义的结果测量器的潜力,承认了患者间的变化.
- 对特定ESS项目的患者回忆显示与日常日记数据相比,不同程度的准确性.
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