用患者报告的共济失调结果测量脊髓小脑性共济失调进展。

IF 7.4 1区医学 Q1 CLINICAL NEUROLOGY

Movement Disorders Pub Date : 2025-03-04 DOI:10.1002/mds.30158

Anna L. Burt AB, Gilbert L'Italien PhD, Susan L. Perlman MD, Liana S. Rosenthal MD, PhD, Sheng-Han Kuo MD, Tetsuo Ashizawa MD, Theresa Zesiewicz MD, Cameron Dietiker MD, Puneet Opal MD, PhD, Antoine Duquette MD, George R. Wilmot MD, PhD, Vikram G. Shakkottai MD, PhD, Christopher M. Gomez MD, Sharan R. Srinivasan MD, PhD, Henry Paulson MD, PhD, Michael D. Geschwind MD, PhD, Sandie Worley MD, Chiadi U. Onyike MD, Andrew Billnitzer MD, Amy Ferng MD, Kristen Matulis DNP, Marie Y. Davis MD, PhD, Sub H. Subramony MD, Anoopum Gupta MD, PhD, Christopher D. Stephen MBChB, Jeremy D. Schmahmann MD

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引用次数: 0

摘要

背景：患者报告的共济失调结果测量（PROM-Ataxia）已经得到了横断面验证，但没有纵向验证。目的：我们旨在验证prom -共济失调作为患者疾病经历的一种衡量标准，检查总体和特定领域的进展，并测试与其他临床结果评估（coa）的趋同效度。方法：我们从小脑共济失调临床研究联盟的176例脊髓小脑共济失调1、2、3、6、7、8或10型患者中获得了基线和1年的promm -共济失调数据。我们根据弗里德里希共济失调评定量表功能分期将患者的共济失调严重程度分期（“严重程度”）分为轻度、中度和重度亚组。整个队列和严重程度亚组的分析包括内部一致性、对疾病严重程度的敏感性、评分变化的预测建模、与coa的相关性：简短共济失调评定量表、共济失调评定量表、疲劳严重程度量表、小脑认知情感综合征量表、EuroQol 5维量表和对疾病进展的反应性。结果：prom -共济失调具有较高的内部一致性，并与其他coa相关。评分显示了对疾病严重程度和不断发展的患者经验的敏感性。进展呈s型，中度患者变化最大。与其他coa相比，prom -共济失调变化最大。轻度患者的精神特征恶化最快，中度患者的身体特征恶化最快，重度患者的日常生活活动恶化最快。结论：prom -共济失调比共济失调coa对变化更敏感，可捕捉1年以上患者疾病经历的演变，并揭示特定领域的进展。对更大的队列和更长时间的不同共济失调诊断的研究可能为进一步加强临床护理和研究提供见解。©2025国际帕金森和运动障碍学会。

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Spinocerebellar Ataxia Progression Measured with the Patient-Reported Outcome Measure of Ataxia

Background

The Patient-Reported Outcome Measure of Ataxia (PROM-Ataxia) has been validated cross-sectionally but not longitudinally.

Objective

We aimed to validate PROM-Ataxia as a measure of patient experience of disease over time, examine overall and domain-specific progression, and test convergent validity with other clinical outcome assessments (COAs).

Methods

We derived PROM-Ataxia data from 176 patients with spinocerebellar ataxia types 1, 2, 3, 6, 7, 8, or 10 in the Clinical Research Consortium for the Study of Cerebellar Ataxia at baseline and 1 year. We classified patients' ataxia severity stage (“severity”) according to the Friedreich's Ataxia Rating Scale Functional Staging into mild, moderate, and severe subgroups. Analyses of the entire cohort and by severity subgroup included internal consistency, sensitivity to disease severity, predictive modeling of score changes, correlations with COAs: Brief Ataxia Rating Scale, Scale for Assessment and Rating of Ataxia, Fatigue Severity Scale, Cerebellar Cognitive Affective Syndrome scale, EuroQol 5-Dimension, and responsiveness to disease progression.

Results

The PROM-Ataxia exhibited high internal consistency and correlated with other COAs. Scores demonstrated sensitivity to disease severity and evolving patient experience. Progression was sigmoidal, with the greatest change in moderate patients. Compared with other COAs, PROM-Ataxia captured the most change. Mental features worsened fastest in mild patients, physical in moderate patients, and activities of daily living in severe patients.

Conclusion

PROM-Ataxia is more sensitive to change than ataxia COAs, captures the evolution of patients' experience of disease over 1 year, and reveals domain-specific progression. Studies of larger cohorts and different ataxia diagnoses over longer periods may provide insights to further enhance clinical care and research. © 2025 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.