Data-driven regions of interest for longitudinal change in three variants of frontotemporal lobar degeneration
Open accessAbstract
Introduction: Longitudinal imaging of neurodegenerative disorders is a potentially powerful biomarker for use in clinical trials. In Alzheimer's disease, studies have demonstrated that empirically derived regions of interest (ROI s) can provide more reliable measurement of disease progression compared with anatomically defined ROI s. Methods: We set out to derive ROI s with optimal effect size for quantifying longitudinal change in a hypothetical clinical trial by comparing atrophy rates in 44 patients with behavioral variant of frontotemporal dementia (bv FTD), 30 with the semantic variant primary progressive aphasia (sv PPA), and 26 with the nonfluent variant PPA (nfv PPA) to atrophy in 97 cognitively healthy controls. Results: The regions identified for each variant were generally what would be expected from prior studies of frontotemporal lobar degeneration (FTLD). Sample size estimates for detecting a 40% reduction in annual rate of ROI atrophy varied substantially across groups, being 103 per arm in bv FTD, 31 in nfv PPA, and 10 in sv PPA, but in all groups were less than those estimated for a priori ROI s and clinical measures. The variability in location of peak regions of atrophy across individuals was highest in bv FTD and lowest in sv PPA, likely relating to the differences in effect size. Conclusions: These findings suggest that, while cross‐validated maps of change can improve sensitivity to change in FTLD compared with a priori regions, the reliability of these maps differs considerably across syndromes. Future studies can utilize these maps to design clinical trials, and should try to identify factors accounting for the variability in patterns of atrophy across individuals, particularly those with bv FTD.
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ROI-based studies identified the medial and lateral frontal lobes, insula, striatum and bilateral temporo-parietal regions (1 year) as important for longitudinal change in bvFTD.
“the medial and lateral frontal lobes, insula, striatum and bilateral temporo-parietal regions (1 year) (Binney et al., 2017)”
From The pursuit for markers of disease progression in behavioral variant frontotemporal dementia: a scoping review to optimize outcome measures for clinical trials (2024) · cites this paper as Binney et al., 2017
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- Authors
- Binney, R. J., Pankov, A., Marx, G., He, X., McKenna, F., Staffaroni, A. M., Kornak, J., Attygalle, S., Boxer, A. L., Schuff, N., Gorno‐Tempini, M., Weiner, M. W., Kramer, J. H., Miller, B. L., & Rosen, H. J.
- Affiliations
- University of California, San Francisco (US)
- Corresponding author
- Rosen, Howard J.
- ORCID
- Venue
- Brain and Behavior
- Publisher
- Wiley
- Published
- 2017-03-23
- Volume
- 7
- Issue
- 4
- Pages
- e00675-e00675
- ISSN
- 2157-9032 (ISSN-L)
- 2162-3279
- PMC
- PMC5390848
- OpenAlex
- W2601677619
- Cited by
- 21
- References
- 51
- Citations by year
- 2025: 1 · 2024: 1 · 2023: 4 · 2022: 1 · 2021: 3 · 2020: 2 · 2019: 6 · 2018: 1 · 2017: 2
- FWCI
- 2.47
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Locations
- Brain and Behavior (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Funders
- National Science Foundation (1144247)
- National Institute of General Medical Sciences (T32GM067547)
- National Institutes of Health (AG045333, AG032306, U54NS092089, R01AG038791, AG019724, AG023501)
- Topics
- Health Sciences › Medicine › Psychiatry and Mental health › Dementia and Cognitive Impairment Research
- Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Keywords
- frontotemporal lobar degeneration, behavioral variant frontotemporal dementia, semantic dementia, region of interest, brain atrophy, longitudinal change, neuroimaging biomarkers, clinical trial design, sample size determination, effect size
- MeSH terms
- Aged, Atrophy, Female, Humans, Longitudinal Studies, Magnetic Resonance Imaging, Male, Mental Status Schedule, Middle Aged, Neuropsychological Tests, Retrospective Studies, Disease Progression, Aphasia, Primary Progressive, Frontotemporal Lobar Degeneration
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