Outcome measures for Parkinson's disease dementia: a systematic review.
Outcome measures for Parkinson's disease dementia: a systematic review.
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DOI：
10.1002/mdc3.12225
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发表时间：
2016-01
影响因子：
4
通讯作者：
Kluger BM
中科院分区：
文献类型：
--
作者：
Holden SK;Jones WE;Baker KA;Boersma IM;Kluger BM
帕金森氏病痴呆(PDD)是帕金森氏病(PD)发病和死亡的主要原因，严重影响患者的功能和生活质量，增加疗养院入院的风险。不幸的是，目前PDD的治疗选择有限，治疗效果微乎其微。随着新疗法的开发，将需要在精心设计的随机对照试验中评估其疗效。然而，对于PDD临床试验中使用的最佳结果指标，还没有达成共识。对PDD的临床试验和用于PDD的结果测量的经验研究进行了系统的综述。结果测量分为五类：1)认知；2)行为和情绪；3)日常生活能力和生活质量；4)总体；5)照顾者负担。共确定了20个PDD药理临床试验。这些试验纳入了一系列广泛的结果测量，但在试验中使用的结果并不一致。我们总结了心理测量学特性和其他使用的结果测量的相关数据，包括它们的诊断效用、评分者之间的可靠性、重测可靠性、响应性、临床意义的变化以及替代形式的可用性。我们已经确定了每个领域中最有证据的PDD结果衡量标准。需要进一步的研究来评估这些措施在PDD中的有效性、可靠性和临床意义的变化，以便为未来临床试验的设计提供信息，并提高临床医生、研究人员和政策制定者解释研究结果的能力。此外，可能有必要制定针对PDD的结果衡量标准。
Parkinson’s disease dementia (PDD) is a major cause of morbidity and mortality in Parkinson’s disease (PD), which severely affects patient functioning and quality of life and increases the risk for nursing home admission. Unfortunately, current treatment options for PDD are limited and have only marginal therapeutic effects. As novel treatments are developed, there will be a need to assess their efficacy in well-designed randomized controlled trials. However, there is no consensus on the optimal outcome measures for use in PDD clinical trials. A systematic review of PDD clinical trials and empiric studies of outcome measures used in PDD was performed. Outcome measures were divided into five categories: 1) cognitive; 2) behavioral and mood; 3) activities of daily living and quality of life; 4) global; and 5) caregiver burden. A total of 20 PDD pharmacologic clinical trials were identified. These trials incorporated a broad array of outcome measures, which were used inconsistently across trials. We summarize the psychometric properties and other relevant data on outcome measures used, including their diagnostic utility, inter-rater reliability, test-retest reliability, responsiveness, clinically meaningful change, and availability of alternate forms. We have identified the best-evidenced PDD outcome measures in each domain. Further research is needed to assess the validity, reliability, and clinically meaningful change of these measures in PDD to inform the design of future clinical trials and enhance the ability of clinicians, researchers and policy-makers to interpret study results. In addition, the development of outcome measures specific to PDD may be warranted.