Mank Arenda, van Maurik Ingrid S, Bakker Els D, van de Glind Esther M M, Jönsson Linus, Kramberger Milica G, Novak Petr, Diaz Ana, Gove Dianne, Scheltens Philip, van der Flier Wiesje M, Visser Leonie N C
Alzheimer Center Amsterdam Department of Neurology VU University Medical Center Amsterdam UMC Amsterdam the Netherlands.
Department of Epidemiology and Data Science Amsterdam UMC, Vrije Universiteit Amsterdam Amsterdam the Netherlands.
Alzheimers Dement (N Y). 2021 Aug 20;7(1):e12189. doi: 10.1002/trc2.12189. eCollection 2021.
Prognostic studies in the context of Alzheimer's disease (AD) mainly predicted time to dementia. However, it is questionable whether onset of dementia is the most relevant outcome along the AD disease trajectory from the perspective of patients and their care partners. Therefore, we aimed to identify the most relevant outcomes from the viewpoint of patients and care partners.
We used a two-step, mixed-methods approach. As a first step we conducted four focus groups in the Netherlands to elicit a comprehensive list of outcomes considered important by patients ( = 12) and care partners ( = 14) in the prognosis of AD. The focus groups resulted in a list of 59 items, divided into five categories. Next, in an online European survey, we asked participants ( = 232; 99 patients, 133 care partners) to rate the importance of all 59 items (5-point Likert scale). As participants were likely to rate a large number of outcomes as "important" (4) or "very important" (5), we subsequently asked them to select the three items they considered most important.
The top-10 lists of items most frequently mentioned as "most important" by patients and care partners were merged into one core outcome list, comprising 13 items. Both patients and care partners selected outcomes from the category "cognition" most often, followed by items in the categories "functioning and dependency" and "physical health." No items from the category "behavior and neuropsychiatry" and "social environment" ended up in our core list of relevant outcomes.
We identified a core list of outcomes relevant to patients and care partner, and found that prognostic information related to cognitive decline, dependency, and physical health are considered most relevant by both patients and their care partners.
阿尔茨海默病（AD）背景下的预后研究主要预测痴呆发生时间。然而，从患者及其护理伙伴的角度来看，痴呆的发生是否是AD疾病轨迹中最相关的结局尚存在疑问。因此，我们旨在从患者和护理伙伴的角度确定最相关的结局。
我们采用了两步混合方法。第一步，我们在荷兰进行了四个焦点小组讨论，以得出患者（n = 12）和护理伙伴（n = 14）认为在AD预后中重要的全面结局清单。焦点小组讨论得出了一份包含59项内容的清单，分为五类。接下来，在一项欧洲在线调查中，我们要求参与者（n = 232；99名患者，133名护理伙伴）对所有59项内容的重要性进行评分（5点李克特量表）。由于参与者可能会将大量结局评为“重要”（4）或“非常重要”（5），随后我们要求他们选择三项他们认为最重要的内容。
患者和护理伙伴最常提及为“最重要”的前10项内容合并为一个核心结局清单，包括13项。患者和护理伙伴最常从“认知”类别中选择结局，其次是“功能与依赖”和“身体健康”类别中的内容。“行为与神经精神学”和“社会环境”类别中的内容均未进入我们的相关核心结局清单。
我们确定了一份与患者和护理伙伴相关的核心结局清单，并发现与认知能力下降、依赖和身体健康相关的预后信息被患者及其护理伙伴认为最相关。
