Damiano A M, Snyder C, Strausser B, Willian M K
Covance Health Economics and Outcomes Services Inc., Washington, DC, USA.
Qual Life Res. 1999 May;8(3):235-43. doi: 10.1023/a:1008823222574.
Parkinson's disease affects individuals health-related quality of life (HQL). Including standardized HQL assessments in therapeutic clinical trials will broaden our understanding of treatment efficacy. Selecting appropriate HQL measures for clinical studies requires consideration of their comprehensiveness, psychometric properties and feasibility. To facilitate selection, this manuscript reviews the HQL areas affected by Parkinson's disease and available Parkinson's disease-specific HQL measures: the Parkinson's Disease Questionnaire--39 (PDQ-39) and the Parkinson's Disease Quality-of-Life Questionnaire (PDQL). Based on a literature review and consultation with HQL experts, five clinicians and three patients, 12 areas of HQL were identified as particularly relevant to Parkinson's disease: physical function, mental health/emotional well being, self-image, social function, health-related distress, cognitive function, communication, sleep and rest, eating, role function, energy/fatigue, and sexual function. The PDQ-39 measures all areas except for self-image and sexual function. The PDQL measures all areas except for eating and role function. Both measures are brief and are designed and validated to be self-completed by patients. Both measures demonstrate adequate internal consistency (PDQ-39: 0.72-0.95; PDQL: 0.80-0.87) and evidence of cross-sectional validity with patient-reported measures of similar concepts. The PDQ-39 also demonstrates reproducibility (0.68-0.94), significant associations with clinical measures and preliminary evidence of responsiveness. Applications of the PDQ-39 and PDQL to clinical trials will contribute greatly towards their continued validation and interpretation.
帕金森病会影响个体的健康相关生活质量（HQL）。在治疗性临床试验中纳入标准化的HQL评估将拓宽我们对治疗效果的理解。为临床研究选择合适的HQL测量方法需要考虑其全面性、心理测量特性和可行性。为便于选择，本文回顾了受帕金森病影响的HQL领域以及现有的帕金森病特异性HQL测量方法：帕金森病问卷 - 39（PDQ - 39）和帕金森病生活质量问卷（PDQL）。基于文献综述以及与HQL专家、五位临床医生和三位患者的咨询，确定了12个与帕金森病特别相关的HQL领域：身体功能、心理健康/情绪状态、自我形象、社会功能、健康相关困扰、认知功能、沟通、睡眠与休息、饮食、角色功能、能量/疲劳和性功能。PDQ - 39测量除自我形象和性功能之外的所有领域。PDQL测量除饮食和角色功能之外的所有领域。这两种测量方法都很简短，并且设计并经验证可由患者自行完成。两种测量方法都显示出足够的内部一致性（PDQ - 39：0.72 - 0.95；PDQL：0.80 - 0.87），并且与患者报告的类似概念测量方法具有横断面效度的证据。PDQ - 39还显示出可重复性（0.68 - 0.94）、与临床测量方法的显著相关性以及反应性的初步证据。将PDQ - 39和PDQL应用于临床试验将极大地有助于它们的持续验证和解读。
