Centre for Dementia Prevention, University of Edinburgh, 9a Edinburgh BioQuarter, 9 Little France Road, Edinburgh, EH16 4UX, UK.
Division of Psychiatry, Centre for Clinical Brain Sciences, University of Edinburgh, Edinburgh, UK.
Alzheimers Res Ther. 2017 Oct 26;9(1):85. doi: 10.1186/s13195-017-0312-4.
This commentary discusses the implications of disease-modifying treatments for Alzheimer's disease which seem likely to appear in the next few years and results from a meeting of British experts in neurodegenerative diseases in Edinburgh. The availability of such treatments would help change public and professional attitudes and accelerate engagement with the prodromal and preclinical populations who might benefit from them. However, this would require an updated understanding of Alzheimer's disease, namely the important distinction between Alzheimer's disease and Alzheimer's dementia.
Since treatments are likely to be most effective in the early stages, identification of clinically relevant brain changes (for example, amyloid burden using imaging or cerebrospinal fluid biomarkers) will be crucial. While current biomarkers could be useful in identifying eligibility for new therapies, trial data are not available to aid decisions about stopping or continuing treatment in clinical practice. Therefore, effective monitoring of safety and effectiveness when these treatments are introduced into clinical practice will be necessary to inform wide-scale use. Equity of access is key but there is a tension between universal access for everyone with a diagnosis of Alzheimer's disease and specifying an eligible population most likely to respond. We propose the resources necessary for an optimal care pathway as well as the necessary education and training for primary and secondary care.
The majority of current services in the UK and elsewhere would not be able to accommodate the specialist investigations required to select patients and prescribe these therapies. Therefore, a stepped approach would be necessary: from innovating sentinel clinical-academic centres that already have capacity to deliver the necessary phase IV trials, through early adoption in a hub and spoke model, to nationwide adoption for true equity of access. The optimism generated by recent and anticipated developments in the understanding and treatment of Alzheimer's disease presents a great opportunity to innovate and adapt our services to incorporate the next exciting development in the field of dementia.
本文讨论了在未来几年内可能出现的针对阿尔茨海默病的疾病修饰治疗的影响，该文源自于在爱丁堡举行的英国神经退行性疾病专家会议。此类治疗方法的出现将有助于改变公众和专业人士的态度，并促使人们更早地关注那些可能从中受益的前驱期和临床前期人群。然而，这需要对阿尔茨海默病有一个更新的认识，即明确区分阿尔茨海默病和阿尔茨海默痴呆。
由于治疗方法可能在早期阶段最有效，因此识别临床上相关的大脑变化（例如，使用成像或脑脊液生物标志物来评估淀粉样蛋白负担）将至关重要。虽然目前的生物标志物可能有助于确定新疗法的资格，但尚无试验数据可用于辅助临床实践中关于停止或继续治疗的决策。因此，在将这些治疗方法引入临床实践时，必须有效地监测安全性和有效性，以便为广泛使用提供信息。公平获得治疗机会是关键，但在为所有被诊断为阿尔茨海默病的人提供普遍获得治疗的机会与指定最有可能对治疗有反应的合格人群之间存在紧张关系。我们提出了优化护理途径所需的资源，以及为初级和二级保健提供必要的教育和培训。
英国和其他地方的大多数当前服务都无法进行选择患者和开这些疗法所需的专科检查。因此，需要采取分阶段的方法：从已经有能力开展必要的 IV 期试验的创新型临床学术中心开始，通过在枢纽和辐射模型中的早期采用，最终实现全国范围内真正公平地获得治疗的机会。最近和预期的在阿尔茨海默病的理解和治疗方面的发展所带来的乐观情绪为我们提供了一个很好的创新机会，可以调整我们的服务，以纳入痴呆领域的下一个令人兴奋的发展。
