Schrag Anette, Spottke Annika, Quinn Niall Patrick, Dodel Richard
Department of Clinical Neurosciences, Royal Free Hospital, University College, Rowland Hill Street, London, UK.
Mov Disord. 2009 Apr 30;24(6):813-8. doi: 10.1002/mds.22438.
The objective of the study is to examine the comparative responsiveness of outcome measures to assess progression over time in Parkinson's disease (PD). One hundred twenty-eight patients participating in a clinic-based naturalistic study of PD were assessed with the Hoehn and Yahr, UPDRS, MMSE, PDQ-39, PDQL, EQ-5D, and BDI scales at baseline and at 1 year. In addition, 82 patients in a community-based study of patients with PD who had completed self-rated Schwab and England, PDQ-39, EQ-5D, and BDI scales at baseline, were sent the same questionnaires at 1 and 4 years. Responsiveness was assessed using t-tests, standardised effect size, and standardised response mean. In both samples, the Hr-QoL measures were less responsive to change over time than the impairment and disability scales (Hoehn and Yahr, UPDRS, Schwab and England scales). In addition, in the clinic-based sample, Hoehn and Yahr and UPDRS ADL scale ("on") were more responsive to progression over time than UPDRS motor part and ADL part ("off"). Hr-QoL measures are less responsive to change over time than measures of impairment and disability. Although this suggests that these measures are less accurate in detecting subtle changes, it may also indicate that the multifactorial subjective assessment of Hr-QoL adapts to changes over time. Global assessment of overall impairment and disability (which incorporates motor and nonmotor features of PD), however, appeared relatively responsive to change over time in patients in a naturalistic setting.
本研究的目的是检验评估帕金森病（PD）随时间进展的结局指标的相对反应性。对128名参与基于门诊的PD自然主义研究的患者在基线和1年时使用Hoehn和Yahr量表、统一帕金森病评定量表（UPDRS）、简易精神状态检查表（MMSE）、帕金森病问卷-39项（PDQ-39）、帕金森病生活质量量表（PDQL）、欧洲五维度健康量表（EQ-5D）和贝克抑郁量表（BDI）进行评估。此外，对82名参与基于社区的PD患者研究的患者在基线时完成了自我评定的施瓦布和英格兰量表、PDQ-39、EQ-5D和BDI量表，并在1年和4年时向他们发送相同的问卷。使用t检验、标准化效应量和标准化反应均值评估反应性。在两个样本中，健康相关生活质量（Hr-QoL）指标随时间变化的反应性低于损伤和残疾量表（Hoehn和Yahr量表、UPDRS量表、施瓦布和英格兰量表）。此外，在基于门诊的样本中，Hoehn和Yahr量表以及UPDRS日常生活活动量表（“开”期）随时间进展的反应性高于UPDRS运动部分和日常生活活动部分（“关”期）。Hr-QoL指标随时间变化的反应性低于损伤和残疾指标。虽然这表明这些指标在检测细微变化方面不太准确，但这也可能表明对Hr-QoL的多因素主观评估会随时间变化而适应。然而，在自然主义环境中，对整体损伤和残疾的总体评估（其中纳入了PD运动和非运动特征）似乎对随时间变化的反应相对较好。
