Zou Haotian, Goetz Christopher G, Stebbins Glenn T, Mestre Tiago A, Luo Sheng
Department of Biostatistics & Bioinformatics, Duke University, Durham, North Carolina, USA.
Department of Neurological Sciences, Rush University Medical Center, Chicago, Illinois, USA.
Mov Disord Clin Pract. 2025 Feb;12(2):148-156. doi: 10.1002/mdc3.14229. Epub 2024 Oct 18.
The MDS-UPDRS Parts IB and II are self-reported items providing a direct patient voice to the experiences of PD.
To determine the most sensitive combination of MDS-UPDRS Parts IB and II items that accurately predicted the clinically relevant target of dopaminergic therapy initiation.
Utilizing a longitudinal cohort of de novo non-treated PD patients, we applied item response theory (IRT) and survival analysis to assess the relationship between baseline patient-reported symptoms and the later initiation of dopaminergic therapy. The 20 MDS-UPDRS Parts IB and II items were analyzed for their relationship to PD severity (discrimination) and the amount of information they provided in this determination (information). These parameters were used to develop models of predictive accuracy for initiation of dopaminergic therapy.
A six-item version showed a significantly higher C-index as compared to the full 20 item model (P = 0.001). This shortened version of the MDS-UPDRS contained only Part II items and provided a predictive accuracy for initiation of dopaminergic therapy better than the total combined scale score or any other combination.
A six-item "Baseline Outcome Voice" version of patient-reported MDS-UPDRS items significantly increases the sensitivity of predicting the key future clinical outcome of starting dopaminergic treatment in early PD. This study also demonstrates how IRT modeling can provide information useful to refining existing measures to identify the most sensitive combination of items honoring the voice of the patient in determining key clinically pertinent decisions. Further research is needed to validate these findings in underrepresented populations.
MDS-UPDRS 第一部分 B 和第二部分是自我报告项目，能直接体现患者对帕金森病（PD）体验的看法。
确定 MDS-UPDRS 第一部分 B 和第二部分项目中最敏感的组合，以准确预测启动多巴胺能治疗的临床相关指标。
利用初发未治疗的 PD 患者纵向队列，应用项目反应理论（IRT）和生存分析来评估基线患者报告症状与后续多巴胺能治疗启动之间的关系。分析了 MDS-UPDRS 第一部分 B 和第二部分的 20 个项目与 PD 严重程度的关系（区分度）以及它们在这一判断中提供的信息量（信息度）。这些参数用于建立多巴胺能治疗启动预测准确性的模型。
与完整的 20 项模型相比，六项版本的 C 指数显著更高（P = 0.001）。这个缩短版的 MDS-UPDRS 仅包含第二部分的项目，对多巴胺能治疗启动的预测准确性优于总综合量表评分或任何其他组合。
患者报告的 MDS-UPDRS 项目的六项“基线结果声音”版本显著提高了预测早期 PD 患者开始多巴胺能治疗这一关键未来临床结果的敏感性。本研究还展示了 IRT 建模如何能提供有用信息，以完善现有测量方法，从而在确定关键临床相关决策时找到最敏感的项目组合，同时尊重患者的看法。需要进一步研究以在代表性不足的人群中验证这些发现。
