阿尔茨海默病治疗亟待进步--欧洲阿尔茨海默病联合会(EADC)研究人员的立场声明

Frank Jessen, M. G. Kramberger, D. Angioni, D. Aarsland, M. Balasa, K. Bennys, M. Boada, M. Boban, A. Chincarini, L. Exalto, A. Felbecker, K. Fliessbach, G. B. Frisoni, A. J. Garza-Martínez, T. Grimmer, B. Hanseeuw, J. Hort, A. Ivanoiu, S. Klöppel, L. Krajcovicova, B. McGuinness, P. Mecocci, A. de Mendonca, A. Nous, P.-J. Ousset, C. Paquet, R. Perneczky, O. Peters, M. Tabuas-Pereira, F. Piazza, D. Plantone, M. Riverol, A. Ruiz, G. Sacco, I. Santana, N. Scarmeas, E. Solje, E. Stefanova, S. Sutovsky, W. van der Flier, T. Welsh, A. Wimo, B. Winblad, L. Frölich, S. Engelborghs
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引用次数: 0

摘要

β-淀粉样蛋白靶向抗体代表了第一代有效的阿尔茨海默病(AD)因果治疗方法,可以说是历史性的研究里程碑。然而,其效果大小、副作用、实施挑战和成本引发了对其整体价值的争论。在这份立场声明中,欧洲阿尔茨海默病联盟(EADC)的临床医生们讨论了现在将这些新疗法引入临床治疗的重要意义。鉴于阿尔茨海默病的复杂性,分子单靶点治疗不太可能取得比目前的β-淀粉样蛋白靶向抗体更大的疗效。更大的疗效很可能只能通过不断优化分子方法、患者选择和组合疗法来逐步实现。要想在这方面取得成功,药物开发必须参考创新疗法在现实世界中的使用情况,因为要充分了解新型疗法的方方面面,除了临床试验之外,还需要现实世界中的治疗经验和数据。关于正在讨论的抗体,我们认为其效果是有意义的,潜在的副作用是可控的。我们假定,由于资格标准狭窄和获得治疗的障碍,最终接受治疗的患者人数仅占所有早期AD患者的一小部分。我们强烈支持在临床实践中对选定的患者使用这些新化合物,并在登记册中记录治疗情况。我们认为这是推进 AD 治疗领域的关键一步,也是为神经退行性疾病分子靶向治疗这一新领域建立医疗保健系统的关键一步。
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Progress in the Treatment of Alzheimer’s Disease Is Needed – Position Statement of European Alzheimer’s Disease Consortium (EADC) Investigators

β-amyloid-targeting antibodies represent the first generation of effective causal treatment of Alzheimer’s disease (AD) and can be considered historical research milestones. Their effect sizes, side effects, implementation challenges and costs, however, have stimulated debates about their overall value. In this position statement academic clinicians of the European Alzheimer’s Disease Consortium (EADC) discuss the critical relevance of introducing these new treatments in clinical care now. Given the complexity of AD it is unlikely that molecular single-target treatments will achieve substantially larger effects than those seen with current β-amyloid-targeting antibodies. Larger effects will most likely only be achieved incrementally by continuous optimization of molecular approaches, patient selection and combinations therapies. To be successful in this regard, drug development must be informed by the use of innovative treatments in real world practice, because full understanding of all facets of novel treatments requires experience and data of real-world care beyond those of clinical trials. Regarding the antibodies under discussion we consider their effects meaningful and potential side effects manageable. We assume that the number of eventually treated patient will only be a fraction of all early AD patients due to narrow eligibility criteria and barriers of access. We strongly endorse the use of these new compound in clinical practice in selected patients with treatment documentation in registries. We understand this as a critical step in advancing the field of AD treatment, and in shaping the health care systems for the new area of molecular-targeted treatment of neurodegenerative diseases.

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来源期刊
The Journal of Prevention of Alzheimer's Disease
The Journal of Prevention of Alzheimer's Disease Medicine-Psychiatry and Mental Health
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9.20
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期刊介绍: The JPAD Journal of Prevention of Alzheimer’Disease will publish reviews, original research articles and short reports to improve our knowledge in the field of Alzheimer prevention including: neurosciences, biomarkers, imaging, epidemiology, public health, physical cognitive exercise, nutrition, risk and protective factors, drug development, trials design, and heath economic outcomes.JPAD will publish also the meeting abstracts from Clinical Trial on Alzheimer Disease (CTAD) and will be distributed both in paper and online version worldwide.We hope that JPAD with your contribution will play a role in the development of Alzheimer prevention.
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