检验临床痴呆评分方框总和作为路易体痴呆临床试验的结果。

IF 4.8 3区医学 Q1 CLINICAL NEUROLOGY

Neurology and Therapy Pub Date : 2025-09-19 DOI:10.1007/s40120-025-00822-x

James E Galvin, Andres Salcedo

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引用次数: 0

摘要

导论：路易体痴呆（DLB）是一种常见的痴呆病因，尚无fda批准的治疗方法，迄今为止的临床试验证明其疗效的能力有限。缺乏有效的dlb特异性临床试验结果可能会阻碍这些努力。在这里，我们测试临床痴呆评分（CDR）和其他常用的阿尔茨海默病（AD）和帕金森病（PD）的临床评估工具是否有可能在未来的DLB临床试验中用作结果测量。方法：对600例患者（359例AD， 241例DLB）进行回顾性横断面分析，这些患者在10年内完成了全面的临床、认知、功能和行为评估。从轻度认知障碍（CDR 0.5）到中重度痴呆（CDR 2），对CDR的表现、其盒数之和（CDR- sb）以及其他AD和PD评估指标进行评估。结果：CDR和CDR- sb在不同疾病严重程度的横截面阶段表征AD和DLB之间的重要差异，其中CDR 0.5阶段差异最大。在CDR 0.5阶段，DLB在常用的AD功能和行为测量中显示出更大的缺陷，而更多的DLB特异性测量在整个疾病谱系中显示出与AD的显着差异。患者版本的快速痴呆评分系统显示，DLB患者比AD患者有更大的阶段损害，支持将其作为患者报告的结果。蒙特利尔认知评估显示AD患者比DLB患者更严重的全阶段损害，这表明缺乏敏感性作为DLB临床试验的结果衡量标准。结论：在DLB临床试验中，改进研究设计和选择合适的结局指标可以促进疗效的证明。虽然CDR-SB可以进行DLB临床试验，但该领域最先进的是开发针对DLB的全球评级工具。

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Testing the Clinical Dementia Rating Sum of Boxes as an Outcome for Dementia with Lewy Bodies Clinical Trials.

Introduction: Dementia with Lewy bodies (DLB), a common cause of dementia, has no FDA-approved therapies, and clinical trials to date have had limited ability to demonstrate efficacy. The lack of validated DLB-specific clinical trial outcomes may hinder these efforts. Here, we test whether the Clinical Dementia Rating (CDR) and other commonly used clinical evaluation tools for Alzheimer's disease (AD) and Parkinson's disease (PD) could potentially be used as outcome measures in future DLB clinical trials.

Methods: A retrospective, cross-sectional chart review of 600 patients (359 AD, 241 DLB) who completed a comprehensive clinical, cognitive, functional, and behavioral evaluation over a 10-year period was carried out. Performance of the CDR, its sum of boxes (CDR-SB), and other AD and PD evaluation measures were assessed for stage-wide performance from mild cognitive impairment (CDR 0.5) to moderate-severe dementia (CDR 2).

Results: The CDR and CDR-SB characterize important differences between AD and DLB across different cross-sectional stages of disease severity, with the greatest differences seen at the CDR 0.5 stage. DLB showed greater deficits in commonly used AD functional and behavioral measures at the CDR 0.5 stage, while more DLB-specific measures showed significant differences from AD across the entire disease spectrum. The patient version of the Quick Dementia Rating System showed greater stage-wide impairment in DLB than AD, supporting its use as a patient-reported outcome. The Montreal Cognitive Assessment showed greater stage-wide impairment in AD than in DLB patients, suggesting lack of sensitivity as an outcome measure for DLB clinical trials.

Conclusion: Improved study design and selection of appropriate outcome measures in DLB clinical trials can facilitate demonstration of efficacy. While the CDR-SB could work on a DLB clinical trial, the field would be most advanced by the development of a DLB-specific global rating instrument.

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来源期刊

Neurology and Therapy CLINICAL NEUROLOGY-

CiteScore

5.40

自引率

8.10%

发文量

103

审稿时长

6 weeks

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