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晚期帕金森病中“良好按时性”的临床、人文及经济价值:一项跨国真实世界研究

Clinical, Humanistic, and Economic Value of 'Good ON-Time' in Advanced Parkinson's Disease: A Multinational Real-World Study.

作者信息

Jimenez-Shahed Joohi, Malaty Irene A, Azulay Jean-Philippe, Parab Ashwini, Yan Connie H, Kandukuri Prasanna L, Kukreja Pavnit, Zamudio Jorge, Gillespie Alexander, Antonini Angelo

机构信息

Department of Neurology, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1137, New York, NY, 10029, USA.

Department of Neurology, University of Florida, Fixel Institute for Neurological Diseases, Gainesville, FL, USA.

出版信息

Neurol Ther. 2025 Jun 2. doi: 10.1007/s40120-025-00765-3.

DOI:10.1007/s40120-025-00765-3
PMID:40455370
Abstract

INTRODUCTION

In advanced Parkinson's disease (aPD), 'ON-time' indicates periods of better symptom control, with 'good ON-time (GOT)' indicating control without troublesome dyskinesia. Despite its importance, the impact of increased 'GOT' on aPD outcomes is understudied. This study aims to evaluate the clinical, humanistic, and economic value of incremental hourly increases in 'GOT' for people with aPD.

METHODS

The study analyzed data from people with aPD across seven countries, using the Adelphi Parkinson's Disease Specific Program survey (2017-2020). 'GOT' (calculated from self-reported ON/OFF-time and the proportion of troublesome dyskinesia time) was normalized to a 16-h day. Outcomes included symptom control, medication use, falls, activities of daily living (ADLs), quality of life (QoL), and healthcare resource utilization (HRU). Regression models evaluated relationships between incremental 'GOT' hours and outcomes.

RESULTS

Of 802 patients (mean [standard deviation; SD] age, 76.1 [8.9] years; male, 60.3%) included in the analysis, mean (SD) 'GOT' was 13.1 (2.7) hours/day. Hourly increases in 'GOT' were associated with lower likelihood of reporting uncontrolled motor (odds ratio [OR] 0.79; 95% confidence interval (CI) [0.62, 1.01]) and non-motor symptoms (OR 0.88; 95% CI [0.80, 0.96]), taking ≥ 2 PD medication classes (OR 0.91; 95% CI [0.86, 0.97]) and lower fall risk (incidence rate ratio 0.91; 95% CI [0.87, 0.95]). Hourly increases in 'GOT' were significantly associated with reduced humanistic burden (greater ADL independence, OR 1.19; 95% CI [1.04 1.37]) and improved QoL (for Parkinson's Disease Questionnaire [PDQ]-39: coefficient - 1.49; 95% CI [- 2.46, - 0.52]) and with reduced economic burden, with annual total HRU cost-savings of $8602.24 (95% CI - $12,192.70 to $5011.77).

CONCLUSIONS

In this multi-country, real-world study of people with aPD, hourly increases in 'GOT' were associated with improved clinical outcomes, greater humanistic value, and reduced economic burden. Interventions that maximize improvement of 'GOT' should be considered for people with aPD adequately controlled on current therapy.

摘要

引言

在晚期帕金森病(aPD)中,“开期”指症状控制较好的时段,“良好开期(GOT)”表示无棘手异动症的控制状态。尽管其很重要,但“GOT”增加对aPD结局的影响研究不足。本研究旨在评估aPD患者每小时“GOT”增加的临床、人文和经济价值。

方法

该研究分析了来自七个国家aPD患者的数据,使用阿德尔菲帕金森病专项计划调查(2017 - 2020年)。“GOT”(根据自我报告的开/关期和棘手异动症时间比例计算)被标准化为16小时的一天。结局包括症状控制、药物使用、跌倒、日常生活活动(ADL)、生活质量(QoL)和医疗资源利用(HRU)。回归模型评估了“GOT”小时数增加与结局之间的关系。

结果

纳入分析的802例患者(平均[标准差;SD]年龄,76.1[8.9]岁;男性,60.3%),平均(SD)“GOT”为13.1(2.7)小时/天。每小时“GOT”增加与报告运动症状未得到控制(比值比[OR]0.79;95%置信区间(CI)[0.62, 1.01])和非运动症状(OR 0.88;95% CI[0.80, 0.96])的可能性较低、服用≥2类帕金森病药物(OR 0.91;95% CI[0.86, 0.97])以及跌倒风险较低(发病率比0.91;95% CI[0.87, 0.95])相关。每小时“GOT”增加与人文负担减轻(ADL独立性增强,OR 1.19;95% CI[1.04, 1.37])、QoL改善(帕金森病问卷[PDQ]-39:系数 - 1.49;95% CI[-2.46, -0.52])以及经济负担减轻显著相关,每年HRU总成本节省8602.24美元(95% CI - 12,192.70美元至5011.77美元)。

结论

在这项针对aPD患者的多国真实世界研究中,每小时“GOT”增加与临床结局改善、更大的人文价值和经济负担减轻相关。对于当前治疗控制良好的aPD患者,应考虑采取干预措施以最大程度提高“GOT”。

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