Clinical Meaningfulness in Alzheimer's Disease Clinical Trials. A Report from the EU-US CTAD Task Force

被引：5

作者：

Angioni, D. ^{[1
]}

Cummings, J. ^{[2
]}

Lansdall, C. J. ^{[3
]}

Middleton, L. ^{[4
]}

Sampaio, C. ^{[5
]}

Gauthier, S. ^{[6
,7
]}

Cohen, S. ^{[8
]}

Petersen, R. C. ^{[9
]}

Rentz, D. M. ^{[10
,11
]}

Wessels, A. M. ^{[12
]}

Hendrix, S. B. ^{[13
]}

Jessen, F. ^{[14
,15
]}

Carrillo, M. C. ^{[16
,17
]}

Doody, R. S. ^{[18
,19
]}

Irizarry, M. ^{[20
]}

Andrews, J. S. ^{[21
]}

Vellas, B. ^{[1
]}

Aisen, P. ^{[22
]}

机构：

[1] Toulouse Univ Hosp, Inst Hosp Univ HealthAge, Alzheimers Dis Res & Clin Ctr, Toulouse, France

[2] Univ Nevada Las Vegas UNLV, Chambers Grundy Ctr Transformat Neurosci, Sch Integrated Hlth Sci, Dept Brain Hlth, Las Vegas, NV USA

[3] F Hoffmann La Roche Ltd, Basel, Switzerland

[4] Imperial Coll London, Sch Publ Hlth, Ageing Epidemiol AGE Res Unit, London, England

[5] CHDI Fdn, CHDI Management, Princeton, NJ USA

[6] McGill Univ, McGill Ctr Studies Aging, Dept Neurol & Neurosurg, Montreal, PQ, Canada

[7] McGill Univ, McGill Ctr Studies Aging, Dept Psychiat, Montreal, PQ, Canada

[8] Toronto Memory Program, Toronto, ON, Canada

[9] Mayo Clin, Dept Neurol, Rochester, MN USA

[10] Harvard Med Sch, Brigham & Womens Hosp, Ctr Alzheimer Res & Treatment, Boston, MA USA

[11] Harvard Med Sch, Massachusetts Gen Hosp, Dept Neurol, Boston, MA USA

[12] Eli Lilly & Co, Indianapolis, IN USA

[13] Hendrix Pentara Corp, Salt Lake City, UT USA

[14] German Ctr Neurodegenerat Dis DZNE, Bonn, Germany

[15] Univ Cologne, Med Fac, Dept Psychiat, Cologne, Germany

[16] Univ Cologne, Excellence Cluster Cellular Stress Responses Aging, Cologne, Germany

[17] Alzheimers Assoc, Chicago, IL USA

[18] Bayor Coll Med, Houston, TX USA

[19] Genentech & Hoffman LaRoche, Basel, Switzerland

[20] Eisai Inc, Nutley, NJ USA

[21] Takeda Pharmaceut, Cambridge, MA USA

[22] USC Alzheimers Therapeut Res Inst, San Diego, CA USA

来源：

JPAD-JOURNAL OF PREVENTION OF ALZHEIMERS DISEASE | 2024年 / 11卷 / 05期

关键词：

RCTs; clinical outcome assessments; CDR; iADRS; QUALITY-OF-LIFE; TIME;

D O I：

10.14283/jpad.2024.112

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

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.

引用

页码：1219 / 1227

页数：9