对MDS"暗示PSP"诊断标准的前性多中心评估
Andrea Quattrone1,2,3, Nicolai Franzmeier4,5,6, Johannes Levin1,5,7
1Department of Neurology, LMU University Hospital, Ludwig-Maximilians-Universität (LMU) München, Munich, Germany.
概括
大多数被诊断为早期渐进性超核性麻 (PSP) 的患者随着时间的推移,诊断确定性增加. 这项研究验证了运动障碍学会 (MDS) -PSP标准,并提出了早期PSP的新试验终点.
科学领域:
- 神经学 神经学
- 运动障碍 运动障碍
- 临床诊断 临床诊断 临床诊断
背景情况:
- 运动障碍学会 (MDS) 建立了渐进性超核性 (PSP) 诊断标准,具有三个确定性级别:暗示性,可能性和可能性.
- 这些水平旨在进行敏感的早期诊断和特定的PSP病理识别.
研究的目的:
- 通过长期临床随访来前性验证MDS-PSP诊断标准.
- 在早期PSP患者中,随着时间的推移,诊断确定性的进展的特征.
主要方法:
- 两项德国多中心纵向观察研究 (ProPSP和DescribePSP) 招募了患有"暗示PSP"或"可能PSP"诊断的患者.
- 评估了在长达2.5年的随访期间增加诊断确定性的患者的累积百分比.
- 预计所需的样本大小,以检测诊断确定性增加的30%减弱率,用于临床试验.
主要成果:
- 在254名具有纵向数据的患者中,61人具有较低的基线确定性 (48人"暗示性",13人"可能").
- 患者增加诊断确定性的累计百分比为6个月后30.4%,1年后51.7%,2.5年后80.4%.
- 估计163个样本大小可以在一年内检测出诊断确定性增加率的30%降低,这比使用PSP评级表更小.
结论:
- 该研究提供了MDS-PSP诊断标准的第一个前性多中心验证.
- 大多数最初被归类为"暗示PSP"的患者进展到更高的诊断确定性.
- 这些发现支持为早期PSP患者设计临床试验,并提出PSP评级尺度和诊断确定性增加率作为潜在的终点.
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