Why a clinical trial is as good as its outcome measure: A framework for the selection and use of cognitive outcome measures for clinical trials of Alzheimer's disease.
Alzheimer's disease
clinical outcome assessments
clinical trial
cognition
preclinical
prodromal
Journal
Alzheimer's & dementia : the journal of the Alzheimer's Association
ISSN: 1552-5279
Titre abrégé: Alzheimers Dement
Pays: United States
ID NLM: 101231978
Informations de publication
Date de publication:
02 2023
02 2023
Historique:
revised:
29
06
2022
received:
25
02
2022
accepted:
22
07
2022
pmc-release:
01
02
2024
pubmed:
11
9
2022
medline:
18
2
2023
entrez:
10
9
2022
Statut:
ppublish
Résumé
A crucial aspect of any clinical trial is using the right outcome measure to assess treatment efficacy. Compared to the rapidly evolved understanding and measurement of pathophysiology in preclinical and early symptomatic stages of Alzheimer's disease (AD), relatively less progress has been made in the evolution of clinical outcome assessments (COAs) for those stages. The current paper aims to provide a benchmark for the design and evaluation of COAs for use in early AD trials. We discuss lessons learned on capturing cognitive changes in predementia stages of AD, including challenges when validating novel COAs for those early stages and necessary evidence for their implementation in clinical trials. Moving forward, we propose a multi-step framework to advance the use of more effective COAs to assess clinically meaningful changes in early AD, which will hopefully contribute to the much-needed consensus around more appropriate outcome measures to assess clinical efficacy of putative treatments. HIGHLIGHTS: We discuss lessons learned on capturing cognitive changes in predementia stages of AD. We propose a framework for the design and evaluation of performance based cognitive tests for use in early AD trials. We provide recommendations to facilitate the implementation of more effective cognitive outcome measures in AD trials.
Identifiants
pubmed: 36086926
doi: 10.1002/alz.12773
pmc: PMC9931632
mid: NIHMS1843856
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
708-720Subventions
Organisme : NIA NIH HHS
ID : P20 AG068053
Pays : United States
Organisme : NIA NIH HHS
ID : R35 AG071476
Pays : United States
Organisme : NINDS NIH HHS
ID : U01 NS093334
Pays : United States
Organisme : NIA NIH HHS
ID : P30 AG072980
Pays : United States
Organisme : NIGMS NIH HHS
ID : P20 GM109025
Pays : United States
Organisme : NIA NIH HHS
ID : R01 AG053798
Pays : United States
Organisme : NIA NIH HHS
ID : P30 AG019610
Pays : United States
Organisme : NIA NIH HHS
ID : P30 AG062421
Pays : United States
Organisme : NIA NIH HHS
ID : P01 AG036694
Pays : United States
Informations de copyright
© 2022 The Authors. Alzheimer's & Dementia published by Wiley Periodicals LLC on behalf of Alzheimer's Association.
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