MDS“提示PSP”诊断标准的前瞻性多中心评价。

IF 7.4 1区医学 Q1 CLINICAL NEUROLOGY

Movement Disorders Pub Date : 2025-01-10 DOI:10.1002/mds.30112

Andrea Quattrone MD, Nicolai Franzmeier PhD, Johannes Levin MD, Gabor C. Petzold MD, Annika Spottke MD, Frederic Brosseron PhD, Björn Falkenburger MD, Johannes Prudlo MD, Thomas Gasser MD, The DESCRIBE-PSP Group, The ProPSP Group, Günter U. Höglinger MD

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引用次数: 0

摘要

背景：最近的运动障碍学会(MDS)-进行性核上性麻痹（PSP）诊断标准概念化了三个临床诊断确定性水平：基于细微临床体征的敏感早期诊断“提示PSP”，平衡敏感性和特异性的“可能PSP”，以及对PSP病理高度特异性的“可能PSP”。目的：本研究的目的是对长期临床随访的标准进行前瞻性验证，并描述诊断确定性随时间增加的特征。方法：在德国两项多中心纵向观察研究（ProPSP和DescribePSP）中招募“可能的PSP”或“提示的PSP”诊断和临床随访的患者。在长达2.5年的随访中，评估了纵向增加诊断确定性的患者的累积百分比。在多个时间间隔内估计试验中诊断确定性增加中检测30%衰减率所需的每组样本量。结果：在254例可获得的纵向数据中，61例患者基线诊断确定性较低（48例提示PSP， 13例可能PSP）和多次临床就诊（中位数：3，范围：2-4）。随着随访时间的延长，增加诊断确定性的患者的累积百分比有所进展（6个月时为30.4%，1年时为51.7%，2.5年时为80.4%）。检测1年内诊断确定性增加率降低30%所需的样本量为163，略小于使用PSP评定量表所需的样本量。结论：大多数“提示PSP”的患者在纵向随访中增加了诊断的确定性，首次提供了MDS-PSP诊断标准的前瞻性多中心验证。我们的数据支持为这些早期患者量身定制的试验设计，表明PSP评分量表和诊断确定性增加率作为潜在的终点测量。©2025作者。Wiley期刊有限责任公司代表国际帕金森和运动障碍学会出版的《运动障碍》。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

Prospective Multicenter Evaluation of the MDS “Suggestive of PSP” Diagnostic Criteria

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Prospective Multicenter Evaluation of the MDS “Suggestive of PSP” Diagnostic Criteria

Background

The recent Movement Disorders Society (MDS)-progressive supranuclear palsy (PSP) diagnostic criteria conceptualized three clinical diagnostic certainty levels: “suggestive of PSP” for sensitive early diagnosis based on subtle clinical signs, “possible PSP” balancing sensitivity and specificity, and “probable PSP” highly specific for PSP pathology.

Objective

The aim of this study was to prospectively validate the criteria against long-term clinical follow-up and characterize the diagnostic certainty increase over time.

Methods

Patients with “possible PSP” or “suggestive of PSP” diagnosis and clinical follow-up were recruited in two German multicenter longitudinal observational studies (ProPSP and DescribePSP). The cumulative percentage of patients longitudinally increasing diagnostic certainty was assessed over up to 2.5 years of follow-up. The sample size per arm required to detect 30% attenuated rate in diagnostic certainty increase in trials was estimated over multiple time intervals.

Results

Of 254 patients with available longitudinal data, 61 patients had low diagnostic certainty at baseline (48 suggestive of PSP, 13 possible PSP) and multiple clinical visits (median: 3, range: 2–4). The cumulative percentage of patients increasing diagnostic certainty progressed with follow-up duration (30.4% at 6 months, 51.7% at 1 year, 80.4% at 2.5 years). The sample size required to detect 30% reduction in diagnostic certainty increase rate within 1 year was 163, slightly smaller than that required using the PSP rating scale.

Conclusions

Most “suggestive of PSP” patients increased diagnostic certainty upon longitudinal follow-up, providing the first prospective multicenter validation of MDS-PSP diagnostic criteria. Our data support the design of trials tailored for these early-stage patients, suggesting the PSP rating scale and the diagnostic certainty increase rate as potential endpoint measures. © 2025 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

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来源期刊

Movement Disorders 医学-临床神经学

CiteScore

13.30

自引率

8.10%

发文量

371

审稿时长

12 months

期刊介绍： Movement Disorders publishes a variety of content types including Reviews, Viewpoints, Full Length Articles, Historical Reports, Brief Reports, and Letters. The journal considers original manuscripts on topics related to the diagnosis, therapeutics, pharmacology, biochemistry, physiology, etiology, genetics, and epidemiology of movement disorders. Appropriate topics include Parkinsonism, Chorea, Tremors, Dystonia, Myoclonus, Tics, Tardive Dyskinesia, Spasticity, and Ataxia.