Harrison Jennifer Kirsty, Noel-Storr Anna H, Demeyere Nele, Reynish Emma L, Quinn Terry J
Alzheimer Scotland Dementia Research Centre, University of Edinburgh, c/o Room S1642, Geriatric Medicine, Royal Infirmary of Edinburgh, 51 Little France Crescent, Edinburgh, EH16 4SB, UK.
Centre for Cognitive Ageing and Cognitive Epidemiology, University of Edinburgh, Edinburgh, UK.
Alzheimers Res Ther. 2016 Nov 21;8(1):48. doi: 10.1186/s13195-016-0216-8.
In a research study, to give a comprehensive evaluation of the impact of interventions, the outcome measures should reflect the lived experience of the condition. In dementia studies, this necessitates the use of outcome measures which capture the range of disease effects, not limited to cognitive functioning. In particular, assessing the functional impact of cognitive impairment is recommended by regulatory authorities, but there is no consensus on the optimal approach for outcome assessment in dementia research. Our aim was to describe the outcome measures used in dementia and mild cognitive impairment (MCI) intervention studies, with particular interest in those evaluating patient-centred outcomes of functional performance and quality of life.
We performed a focused review of the literature with multiple embedded checks of internal and external validity. We used the Cochrane Dementia and Cognitive Improvement Group's register of dementia studies, ALOIS. ALOIS was searched to obtain records of all registered dementia and MCI intervention studies over a 10-year period (2004-2014). We included both published and unpublished materials. Outcomes were categorised as cognitive, functional, quality of life, mood, behaviour, global/disease severity and institutionalisation.
From an initial return of 3271 records, we included a total of 805 records, including 676 dementia trial records and 129 MCI trial records. Of these, 78 % (630) originated from peer-reviewed publications and 60 % (487) reported results of pharmacological interventions. Cognitive outcomes were reported in 70 % (563), in contrast with 29 % (237) reporting measures of functional performance and only 13 % (102) reporting quality of life measures. We identified significant heterogeneity in the tools used to capture these outcomes, with frequent use of non-standardised tests.
This focus on cognitive performance questions the extent to which intervention studies for dementia are evaluating outcome measures which are relevant to individual patients and their carers. The heterogeneity in measures, use of bespoke tools and poor descriptions of test strategy all support the need for a more standardised approach to the conduct and reporting of outcomes assessments.
在一项研究中，为了全面评估干预措施的影响，结局指标应反映疾病的实际体验。在痴呆症研究中，这就需要使用能够涵盖疾病影响范围的结局指标，而不仅限于认知功能。特别是，监管机构建议评估认知障碍的功能影响，但在痴呆症研究的结局评估最佳方法上尚未达成共识。我们的目的是描述痴呆症和轻度认知障碍（MCI）干预研究中使用的结局指标，尤其关注那些评估以患者为中心的功能表现和生活质量结局的指标。
我们对文献进行了重点综述，并对内部和外部效度进行了多次嵌入式检查。我们使用了Cochrane痴呆与认知改善小组的痴呆症研究登记册ALOIS。对ALOIS进行检索，以获取10年期间（2004 - 2014年）所有已注册的痴呆症和MCI干预研究的记录。我们纳入了已发表和未发表的材料。结局分为认知、功能、生活质量、情绪、行为、整体/疾病严重程度和机构化。
从最初返回的3271条记录中，我们共纳入了805条记录，包括676条痴呆症试验记录和129条MCI试验记录。其中，78%（630条）来自同行评审出版物，60%（487条）报告了药物干预的结果。70%（563条）报告了认知结局，相比之下，29%（237条）报告了功能表现指标，只有13%（102条）报告了生活质量指标。我们发现用于获取这些结局的工具存在显著异质性，经常使用非标准化测试。
对认知表现的关注质疑了痴呆症干预研究在评估与个体患者及其护理人员相关的结局指标方面的程度。测量方法的异质性、定制工具的使用以及测试策略描述不佳均支持在结局评估的实施和报告中需要采用更标准化的方法。
