神经退行性疾病临床试验中的视频评分：PRION-1的经验
Video Rating in Neurodegenerative Disease Clinical Trials: The Experience of PRION-1.
作者信息
Carswell Christopher, Rañopa Michael, Pal Suvankar, Macfarlane Rebecca, Siddique Durre, Thomas Dafydd, Webb Tom, Wroe Steve, Walker Sarah, Darbyshire Janet, Collinge John, Mead Simon, Rudge Peter
机构信息
MRC Prion Unit and Department of Neurodegenerative Disease, University College London Institute of Neurology, and National Prion Clinic, National Hospital for Neurology and Neurosurgery, University College London Hospitals NHS Trust, London, UK.
出版信息
Dement Geriatr Cogn Dis Extra. 2012 Jan;2(1):286-97. doi: 10.1159/000339730. Epub 2012 Aug 8.
BACKGROUND/AIMS: Large clinical trials including patients with uncommon diseases involve assessors in different geographical locations, resulting in considerable inter-rater variability in assessment scores. As video recordings of examinations, which can be individually rated, may eliminate such variability, we measured the agreement between a single video rater and multiple examining physicians in the context of PRION-1, a clinical trial of the antimalarial drug quinacrine in human prion diseases.
METHODS
We analysed a 43-component neurocognitive assessment battery, on 101 patients with Creutzfeldt-Jakob disease, focusing on the correlation and agreement between examining physicians and a single video rater.
RESULTS
In total, 335 videos of examinations of 101 patients who were video-recorded over the 4-year trial period were assessed. For neurocognitive examination, inter-observer concordance was generally excellent. Highly visual neurological examination domains (e.g. finger-nose-finger assessment of ataxia) had good inter-rater correlation, whereas those dependent on non-visual clues (e.g. power or reflexes) correlated poorly. Some non-visual neurological domains were surprisingly concordant, such as limb muscle tone.
CONCLUSION
Cognitive assessments and selected neurological domains can be practically and accurately recorded in a clinical trial using video rating. Video recording of examinations is a valuable addition to any trial provided appropriate selection of assessment instruments is used and rigorous training of assessors is undertaken.
背景/目的：纳入罕见病患者的大型临床试验涉及不同地理位置的评估者，这导致评估分数存在相当大的评估者间变异性。由于可单独评分的检查视频记录可能消除这种变异性，我们在PRION-1（一项关于抗疟药奎纳克林治疗人类朊病毒病的临床试验）的背景下，测量了单一视频评分者与多名检查医师之间的一致性。
方法
我们分析了一个包含43个项目的神经认知评估量表，对101例克雅氏病患者进行评估，重点关注检查医师与单一视频评分者之间的相关性和一致性。
结果
在为期4年的试验期内，共评估了101例患者的335份检查视频。对于神经认知检查，观察者间的一致性总体良好。视觉性较强的神经学检查领域（如共济失调的指鼻指试验评估）具有良好的评估者间相关性，而那些依赖非视觉线索的领域（如肌力或反射）相关性较差。一些非视觉性神经学领域的一致性令人惊讶，如肢体肌张力。
结论
在临床试验中使用视频评分可以切实且准确地记录认知评估和选定的神经学领域。只要适当选择评估工具并对评估者进行严格培训，检查视频记录对任何试验都是一项有价值的补充。
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