Institute of Psychiatry, King's College London, London, UK.
Int J Geriatr Psychiatry. 2014 Jan;29(1):22-31. doi: 10.1002/gps.3958. Epub 2013 May 6.
There is great interest in conducting clinical trials of disease-modifying therapies in the prodromal (early, pre-dementia), asymptomatic stages of Alzheimer's disease. Diagnostic biomarker tests offer a means of identifying prodromal patients, but it is unclear how potential participants feel about their use. Deciding whether to take part in a clinical trial is a complex process in which eligible participants must balance risks and discomforts against uncertain benefits. We sought to explore the views of potential participants through qualitative research methods.
Focus groups with people with early memory problems, current and former family carers explored attitudes towards participating in clinical trials in the prodromal stages of the disease, using an example of anti-amyloid antibody-therapy (immunotherapy), which are currently in development.
Despite the complexities involved, almost all participants had a clear idea about whether they, personally, would like to take part. Many were highly motivated to obtain an unambiguous diagnosis, regardless of their desire to participate in a clinical trial. Participants expressed minimal concern regarding the risk of adverse events associated with immunotherapy, whereas certain tests and trial procedures provoked greater anxiety. People with memory problems were found to assess the study demands in relation to their own priorities and circumstances.
The priorities of patients might be different to clinicians and those who design and regulate clinical trials. Patient views can be used to inform the ethical debate around the disclosure of biomarker status, the design of clinical trials and the content of trial information.
人们对于在阿尔茨海默病的前驱期（早期、痴呆前）和无症状阶段开展针对疾病修饰疗法的临床试验有着浓厚的兴趣。诊断生物标志物检测为识别前驱期患者提供了一种手段，但目前尚不清楚潜在参与者对其使用的看法。是否参与临床试验是一个复杂的决策过程，合格的参与者必须权衡风险和不适与不确定的获益。我们通过定性研究方法来探索潜在参与者的观点。
我们开展了有早期记忆问题患者、现症和既往照料者参加的焦点小组讨论，采用正在开发的抗淀粉样蛋白抗体疗法（免疫疗法）这一事例，探讨了参与者对在疾病前驱期参加临床试验的态度。
尽管存在诸多复杂性，但几乎所有参与者都对自己是否愿意参加有明确的想法。许多人非常希望获得明确的诊断，而不论他们是否有兴趣参加临床试验。尽管免疫疗法相关的不良事件风险较低，但参与者对某些检测和试验程序的担忧较小。有记忆问题的人会根据自己的优先级和情况来评估研究的需求。
患者的优先事项可能与临床医生以及设计和监管临床试验的人员不同。患者的观点可以用于为围绕生物标志物状态的披露、临床试验的设计以及试验信息的内容的伦理辩论提供信息。
