Lamichhane Dronacharya, Gruber-Baldini Ann L, Reich Stephen G, Shulman Lisa M
Illinois Neurological Institute, University of Illinois College of Medicine-Peoria, 100 NE Randolph Avenue, Peoria, IL, 61606, USA.
Division of Gerontology, Department of Epidemiology and Public Health, University of Maryland School of Medicine, Baltimore, MD, USA.
Qual Life Res. 2016 Dec;25(12):3139-3145. doi: 10.1007/s11136-016-1351-1. Epub 2016 Jun 30.
Clinical trials in Parkinson's disease commonly employ outcome measures of disability and quality of life. Responsiveness of these outcomes measures to symptomatic decline versus improvement has not been studied. We wanted to study the responsiveness of Schwab & England Activities of Daily Living Scale (SE) and Short Form-12 (SF-12) to symptomatic decline versus improvement in Parkinson's disease over a 4-year period among a naturalistic cohort of patients.
Parkinson's disease patients (N = 228, disease duration 6.1 years) were followed for 4 years with assessments of disease severity, Unified Parkinson's Disease Rating Scale (UPDRS), health-related quality of life (SF-12 physical/mental health), and disability (SE). The sample was subdivided into those who declined (N = 118) or improved (N = 102) on total-UPDRS. Responsiveness was assessed with Cohen's effect size and standardized response mean.
At baseline, patients who improved over 4 years had greater disease severity and worse quality of life than decliners (p < .05). Decliners had a 13.5-point worsening on total-UPDRS, 26.3-39.8; p < .001) associated with concomitant decline on the SF-12 (physical health 42.9-39.2, mental health 50.0-46.6; both p < .001) and the SE (85-74 %; p < .001). Improvers had a 13.0-point improvement on total-UPDRS (39.8-26.8; p < .001) associated with minimal change on the SF-12 (physical health 40.8-39.5, mental health 47.1-46.3) and SE (79-79 %). Based on effect size, the rank order of responsiveness of measures for decliners from high to low was SE (-0.78), Short Form-12 mental health (-0.45), and SF-12 physical health (-0.34). Rank order of responsiveness for improvers was Short Form-12 physical health (-0.11), SF-12 mental health (-0.10), and SE (-0.03).
Among decliners, measures of disability and quality of life were moderate to highly responsive to change in disease severity. Among improvers, both disability and quality of life were poorly responsive despite UPDRS improvement of comparable magnitude.
帕金森病临床试验通常采用残疾和生活质量的结局指标。尚未对这些结局指标对症状恶化与改善的反应性进行研究。我们希望研究施瓦布与英格兰日常生活活动量表（SE）和简明健康调查简表12（SF - 12）在4年期间对帕金森病自然队列患者症状恶化与改善的反应性。
对帕金森病患者（N = 228，病程为6.1年）进行了4年的随访，评估疾病严重程度、统一帕金森病评定量表（UPDRS）、健康相关生活质量（SF - 12身体/心理健康）和残疾情况（SE）。样本根据UPDRS总分分为病情恶化组（N = 118）和病情改善组（N = 102）。采用科恩效应量和标准化反应均值评估反应性。
在基线时，4年中病情改善的患者比病情恶化的患者疾病严重程度更高，生活质量更差（p < 0.05）。病情恶化组UPDRS总分恶化13.5分（26.3 - 39.8；p < 0.001），同时SF - 12（身体健康从42.9降至39.2，心理健康从50.0降至46.6；均p < 0.001）和SE（从85%降至74%；p < 0.001）也下降。病情改善组UPDRS总分改善13.0分（39.8 - 26.8；p < 0.001），而SF - 12（身体健康从40.8变为39.5，心理健康从47.1变为46.3）和SE（从79%变为79%）变化极小。基于效应量，病情恶化组各指标反应性从高到低的排序为SE（-0.78）、简明健康调查简表12心理健康（-0.45）和SF - 12身体健康（-0.34）。病情改善组反应性排序为SF - 12身体健康（-0.11）、SF - 12心理健康（-0.10）和SE（-0.03）。
在病情恶化组中，残疾和生活质量指标对疾病严重程度变化的反应为中度至高度敏感。在病情改善组中，尽管UPDRS有相当程度的改善，但残疾和生活质量指标的反应性都很差。
