Quattrone Andrea, Franzmeier Nicolai, Levin Johannes, Petzold Gabor C, Spottke Annika, Brosseron Frederic, Falkenburger Björn, Prudlo Johannes, Gasser Thomas, Höglinger Günter U
Department of Neurology, LMU University Hospital, Ludwig-Maximilians-Universität (LMU) München, Munich, Germany.
Department of Medical and Surgical Sciences, Institute of Neurology, Magna Graecia University, Catanzaro, Italy.
Mov Disord. 2025 Mar;40(3):526-536. doi: 10.1002/mds.30112. Epub 2025 Jan 10.
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.
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.
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.
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.
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.
运动障碍协会（MDS）最近制定的进行性核上性麻痹（PSP）诊断标准将临床诊断确定性水平分为三级：基于细微临床体征进行敏感早期诊断的“提示PSP”；平衡敏感性和特异性的“可能PSP”；对PSP病理高度特异的“很可能PSP”。
本研究旨在通过长期临床随访对该标准进行前瞻性验证，并描述诊断确定性随时间的增加情况。
在两项德国多中心纵向观察性研究（ProPSP和DescribePSP）中招募诊断为“可能PSP”或“提示PSP”且有临床随访的患者。在长达2.5年的随访中评估纵向提高诊断确定性的患者累积百分比。估计在多个时间间隔内试验中检测诊断确定性增加率降低30%所需的每组样本量。
在254例有可用纵向数据的患者中，61例患者基线时诊断确定性较低（48例提示PSP，13例可能PSP）且进行了多次临床访视（中位数：3次，范围：2 - 4次）。诊断确定性增加的患者累积百分比随随访时间而增加（6个月时为30.4%，1年时为51.7%，2.5年时为80.4%）。在1年内检测诊断确定性增加率降低30%所需的样本量为163例，略小于使用PSP评定量表所需的样本量。
大多数“提示PSP”的患者在纵向随访后诊断确定性增加，这是对MDS - PSP诊断标准的首次前瞻性多中心验证。我们的数据支持为这些早期患者量身定制试验的设计，表明PSP评定量表和诊断确定性增加率可作为潜在的终点指标。© 2025作者。《运动障碍》由Wiley Periodicals LLC代表国际帕金森病和运动障碍协会出版。
