Alexander S K, Rittman T, Xuereb J H, Bak T H, Hodges J R, Rowe J B
Addenbrooke's Hospital, Cambridge, UK.
Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK.
J Neurol Neurosurg Psychiatry. 2014 Aug;85(8):925-9. doi: 10.1136/jnnp-2013-307035. Epub 2014 Feb 12.
Corticobasal degeneration (CBD) is a complex neurodegenerative disorder. Accurate diagnosis is increasingly important, with the advent of clinical trials of drugs aimed at modifying the underlying tau pathology. CBD often presents with a 'corticobasal syndrome' including impairments of movement and cognition. However, patients with similar corticobasal syndromes can have neurodegenerative pathologies that are not CBD. In addition, patients with CBD may present with aphasia or behavioural change. The clinical diversity of CBD and mimicry by non-CBD pathologies hinders accurate diagnosis.
We applied the new consensus criteria of Armstrong and colleagues et al 1 to a cohort of patients with detailed longitudinal clinical evaluation and neuropathology.
In patients with pathologically confirmed CBD, accuracy of diagnosis was similar under the new and previous criteria: 9/19 (47%) met criteria for probable CBD at presentation, 13/19 (68%) at last clinical assessment. Patients with a corticobasal syndrome but without CBD pathology all (14/14) met the new diagnostic criteria of probable or possible CBD, demonstrating that the new criteria lacks the necessary specificity for an accurate ante mortem clinical diagnosis of CBD. None of the clinical features used in the new criteria were more common in the patients with CBD pathology (n=19) than without (n=14).
The Armstrong criteria usefully broadens the recognised clinical phenotype of CBD but does not sufficiently improve the specificity of diagnosis to increase the power of clinical trials or targeted applications of tau-based disease-modifying therapies. Further work is required to show whether biomarkers could be more effective than clinical signs in the diagnosis of CBD.
皮质基底节变性（CBD）是一种复杂的神经退行性疾病。随着针对潜在tau蛋白病理改变的药物临床试验的出现，准确诊断变得越来越重要。CBD常表现为“皮质基底节综合征”，包括运动和认知障碍。然而，具有相似皮质基底节综合征的患者可能患有非CBD的神经退行性病变。此外，CBD患者可能出现失语或行为改变。CBD的临床多样性以及非CBD病理改变的模仿性阻碍了准确诊断。
我们将阿姆斯特朗及其同事等人的新共识标准应用于一组经过详细纵向临床评估和神经病理学检查的患者。
在病理确诊为CBD的患者中，新标准和先前标准下的诊断准确性相似：9/19（47%）在初诊时符合可能CBD的标准，13/19（68%）在最后一次临床评估时符合标准。患有皮质基底节综合征但无CBD病理改变的所有患者（14/14）均符合可能或可能CBD的新诊断标准，这表明新标准缺乏对CBD进行准确生前临床诊断所需的特异性。新标准中使用的任何临床特征在有CBD病理改变的患者（n = 19）中并不比无CBD病理改变的患者（n = 14）更常见。
阿姆斯特朗标准有效地拓宽了公认的CBD临床表型，但并未充分提高诊断的特异性，以增强临床试验或基于tau蛋白的疾病修饰疗法的靶向应用的效力。需要进一步的研究来表明生物标志物在CBD诊断中是否比临床体征更有效。
