阿尔茨海默病临床试验的临床意义。欧盟-美国 CTAD 工作组报告

D. Angioni, J. Cummings, C. J. Lansdall, L. Middleton, C. Sampaio, S. Gauthier, S. Cohen, R. C. Petersen, D. M. Rentz, A. M. Wessels, S. B. Hendrix, F. Jessen, M. C. Carrillo, R. S. Doody, M. Irizarry, J. S. Andrews, B. Vellas, P. Aisen, Sandrine Andrieu, Randall Bateman, Richard Batrla, Joanne Bell, Oskar Bosson, Sasha Bozeat, Dawn Brooks, Samantha Budd Haeberlein, Teresa Buracchio, Min Cho, Matthew Choung, Gavin Cook, Darrin Crisitello, Fernando Dangond, Susan de Santi, Ellen Dennehy, Shobha Dhadda, Harjeet Dhillon, Billy Dunn, Michael Egan, Fiona Elwood, Sven Eriksson, Tom Fagan, Howard Fillit, Per-Ola Freskgard, Diana Gallagher, Gopi Gangi, Carlos Granda, David Greeley, Anna-Kaija Gronblad, Harald Hampel, Paul Hawthorne, David Henley, Joe Herring, Steve Hersch, Bill Holt, Takeshi Iwatsubo, Daryl Jones, Anja Kahl, Gene Kinney, Hartmuth Kolb, Lynn Kramer, Luka Kulic, Sanjay Kumar, Lars Lannfelt, John Lawson, Valérie Legrand, Rachel Lenington, Frank Longo, Brandy Matthews, D..
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引用次数: 0

摘要

继数十年的失败之后,最近三项抗淀粉样蛋白单克隆抗体 III 期试验的积极结果正在改变改变阿尔茨海默病治疗方法的格局。事实上,所有三项试验都达到了主要终点。然而,在这些试验中测出的获益的绝对规模引发了一场争论,即在临床结果评估中观察到的评分变化是否代表患者获得了有临床意义的获益。目前迫切需要一个基于证据的结论,以便在临床实践中为新兴疗法的审批、报销和最终管理提供决策依据。欧盟-美国 CTAD 工作组在波士顿召开会议,以解决这一重要问题。会议讨论并总结了目前用于解释 AD 临床试验结果临床意义的最新知识,包括患者和研究合作伙伴对什么是有临床意义的观点。为应对挑战,提出了多种方法。对临床意义的理解仍存在差距,只有长期纵向研究才能解决这一问题。
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Clinical Meaningfulness in Alzheimer’s Disease Clinical Trials. A Report from the EU-US CTAD Task Force

Recent positive results of three phase III anti-amyloid monoclonal antibody trials are transforming the landscape of disease-modifying therapeutics for Alzheimer’s disease, following several decades of failures. Indeed, all three trials have met their primary endpoints. However, the absolute size of the benefit measured in these trials has generated a debate on whether the change scores observed on clinical outcome assessments represent a clinically meaningful benefit to patients. An evidence-based conclusion is urgently required to inform decision-making related to the approval, reimbursement, and ultimately, the management of emerging therapies in clinical practice. The EU-US CTAD Task Force met in Boston to address this important question. The current state-of-the-art knowledge for interpreting clinical meaningfulness of AD clinical trial results, including the point of view of patients and study partners on what is clinically meaningful, was discussed and is summarized here. A combination of methodologies to address the challenges emerged. There remain gaps in the understanding of clinical meaningfulness that only long-term longitudinal studies will be able to address.

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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.
期刊最新文献
Association between Cognitive Reserve Indicator and Chronic Disease-Free Survival: A Large Community-Based Longitudinal Study Phase 1 Studies of the Anti-Tau Monoclonal Antibody JNJ-63733657 in Healthy Participants and Participants with Alzheimer’s Disease Roles of TREM2 in the Pathological Mechanism and the Therapeutic Strategies of Alzheimer’s Disease Development and Validation the Mobile Toolbox (MTB) Spelling Test Correlates of Subjective Cognitive Decline in Black American Men
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