帕金森氏病疾病进展和治疗效果评估的PARCOMS复合量表的开发和验证

IF 3.9 3区医学 Q1 CLINICAL NEUROLOGY

Neurology and Therapy Pub Date : 2025-06-16 DOI:10.1007/s40120-025-00771-5

Gil L'Italien, Basia Rogula, Lauren Powell, Michele Potashman, Samuel P Dickson, Nick Kozauer, Patrick O'Keefe, Ellen Korol, Madeleine Crabtree, Fernanda Nagase, Vlad Coric, Jordan Dubow, Liana S Rosenthal, Suzanne Hendrix

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引用次数: 0

摘要

摘要：设计用于全面评估帕金森病（PD）而不考虑疾病阶段和治疗状态的措施可能无法捕捉到疾病进展的细微差别，特别是在早期PD中。本文的目的是为三个离散队列的患者开发帕金森综合量表（PARCOMS），该量表使用运动障碍学会统一帕金森病评定量表（MDS-UPDRS）的项目，增加对临床衰退的反应性。方法：根据帕金森进展标志物倡议（PPMI）数据确诊的PD患者根据多巴胺能治疗的使用、疾病分期和运动并发症的存在分为三个队列。对于每个队列，MDS-UPDRS第一部分（PARCOMS-Non-Motor）和第二部分和第三部分（PARCOMS-Motor）的项目根据响应性使用偏最小二乘（PLS）回归进行选择。量表的响应性是用其变化值的均值-标准差比（msdr）来估计的。结果：与最初的MDS-UPDRS相比，PARCOMS-Motor的msdr增加了13.1%（未治疗组，n = 430）， 78.2%（未治疗组，n = 426）和100.6%（未治疗组，n = 538）。PARCOMS-Non-Motor的MSDR增加分别为13.9%、6.8%和20.7%。在整个队列中，在床上翻身和言语项目是PARCOMS-Motor量表的主要贡献者。认知障碍和泌尿系统问题的项目是PARCOMS-Non-Motor队列的重要贡献者。在每个组合的队列中，代表不同临床概念的项目的权重存在变异性，证实了不同疾病阶段疾病进展的异质性。结论：PD分期特异性复合指标被开发出来，与最初的MDS-UPDRS相比，对变化的敏感性更高，支持了针对疾病分期量身定制的加权复合指标的价值。

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Development and Validation of PARCOMS Composite Scales for Assessing Disease Progression and Treatment Effects in Parkinson's Disease.

Introduction: Measures designed to comprehensively assess Parkinson's disease (PD) irrespective of disease stage and treatment status may be unable to capture nuances in disease progression, particularly in early-stage PD. The objective of this paper is to develop PARkinson's COMposite Scales (PARCOMS) with increased responsiveness to clinical decline using items of the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) for three discrete cohorts of patients.

Methods: Patients with confirmed PD from the Parkinson's Progression Markers Initiative (PPMI) data were assigned to three cohorts based on use of dopaminergic treatment, stage of disease, and presence of motor complication. For each cohort, items from MDS-UPDRS Part I (PARCOMS-Non-Motor) and Parts II and III (PARCOMS-Motor) were selected based on responsiveness using partial least squares (PLS) regression. The responsiveness of the scales was estimated using mean-to-standard deviation ratios (MSDRs) of their change values.

Results: Compared to the original MDS-UPDRS, MSDRs for PARCOMS-Motor increased 13.1% (untreated cohort, n = 430), 78.2% (treated-without-motor-complications cohort, n = 426), and 100.6% (treated-with-motor-complications cohort, n = 538). The MSDR increases observed for PARCOMS-Non-Motor were 13.9%, 6.8%, and 20.7%, respectively. Across cohorts, turning in bed and speech items were large contributors to the PARCOMS-Motor scales. Items for cognitive impairment and urinary problems were substantial contributors to PARCOMS-Non-Motor across cohorts. There was variability in the weighting of items representing different clinical concepts across cohorts for each composite, confirming heterogeneity in disease progression across disease stages.

Conclusions: PD stage-specific composite measures were developed and demonstrated greater sensitivity to change than the original MDS-UPDRS, supporting the value of weighted composites tailored for disease stage.

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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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