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Examining amyloid reduction as a surrogate endpoint through latent class analysis using clinical trial data for dominantly inherited Alzheimer's disease

Title: Examining amyloid reduction as a surrogate endpoint through latent class analysis using clinical trial data for dominantly inherited Alzheimer's disease
Authors: Wang, G; Li, Y; Xiong, C; Benzinger, TLS; Gordon, BA; Hassenstab, J; Aschenbrenner, AJ; McDade, E; Clifford, DB; Libre-Guerra, JJ; Shi, X; Mummery, CJ; van Dyck, CH; Lah, JJ; Honig, LS; Day, G; Ringman, JM; Brooks, WS; Fox, NC; Suzuki, K; Levin, J; Jucker, M; Delmar, P; Bittner, T; Bateman, RJ
Source: Alzheimer's and Dementia (2024) (In press).
Publisher Information: Wiley
Publication Year: 2024
Collection: University College London: UCL Discovery
Subject Terms: Dominantly Inherited Alzheimer Network; autosomal dominant Alzheimer's disease; gantenerumab; latent class analysis; solanezumab; surrogate biomarker
Description: INTRODUCTION: Increasing evidence suggests that amyloid reduction could serve as a plausible surrogate endpoint for clinical and cognitive efficacy. The double-blind phase 3 DIAN-TU-001 trial tested clinical and cognitive declines with increasing doses of solanezumab or gantenerumab. METHODS: We used latent class (LC) analysis on data from the Dominantly Inherited Alzheimer Network Trials Unit 001 trial to test amyloid positron emission tomography (PET) reduction as a potential surrogate biomarker. RESULTS: LC analysis categorized participants into three classes: amyloid no change, amyloid reduction, and amyloid growth, based on longitudinal amyloid Pittsburgh compound B PET standardized uptake value ratio data. The amyloid-no-change class was at an earlier disease stage for amyloid amounts and dementia. Despite similar baseline characteristics, the amyloid-reduction class exhibited reductions in the annual decline rates compared to the amyloid-growth class across multiple biomarker, clinical, and cognitive outcomes. DISCUSSION: LC analysis indicates that amyloid reduction is associated with improved clinical outcomes and supports its use as a surrogate biomarker in clinical trials. Highlights: We used latent class (LC) analysis to test amyloid reduction as a surrogate biomarker. Despite similar baseline characteristics, the amyloid-reduction class exhibited remarkably better outcomes compared to the amyloid-growth class across multiple measures. LC analysis proves valuable in testing amyloid reduction as a surrogate biomarker in clinical trials lacking significant treatment effects.
Document Type: article in journal/newspaper
File Description: application/pdf
Language: English
Relation: https://discovery.ucl.ac.uk/id/eprint/10189432/1/Examining%20amyloid%20reduction%20as%20a%20surrogate%20endpoint%20through%20latent%20class%20analysis.pdf; https://discovery.ucl.ac.uk/id/eprint/10189432/
Availability: https://discovery.ucl.ac.uk/id/eprint/10189432/1/Examining%20amyloid%20reduction%20as%20a%20surrogate%20endpoint%20through%20latent%20class%20analysis.pdf; https://discovery.ucl.ac.uk/id/eprint/10189432/
Rights: open
Accession Number: edsbas.1AD3FA76
Database: BASE