scholarly journals Sample size estimation for clinical trials with tau‐PET SUVR as the primary outcome in dominantly inherited Alzheimer’s disease

2021 ◽  
Vol 17 (S9) ◽  
Author(s):  
Guoqiao Wang ◽  
Yan Li ◽  
Chengjie Xiong ◽  
Tammie L.S. Benzinger ◽  
Brian A. Gordon ◽  
...  
2017 ◽  
Vol 56 (1) ◽  
pp. 75-88 ◽  
Author(s):  
Motonobu Fujishima ◽  
Atsushi Kawaguchi ◽  
Norihide Maikusa ◽  
Ryozo Kuwano ◽  
Takeshi Iwatsubo ◽  
...  

2011 ◽  
Vol 24 (5) ◽  
pp. 689-697 ◽  
Author(s):  
P. A. Thompson ◽  
D. E. Wright ◽  
C. E. Counsell ◽  
J. Zajicek

ABSTRACTBackground: The social and economic burden of Alzheimer's disease (AD) and its increasing prevalence has led to much work on new treatment strategies and clinical trials. The search for surrogate markers of disease progression continues but traditional parallel group trial designs that use well-established, but often insensitive, clinical outcome measures predominate.Methods: We performed a systematic search across the Cochrane Library and PubMed abstracts published between January 2004 and August 2009. Information regarding the clinical trial methodology, outcome measures, intervention type and primary statistical analysis techniques was extracted and categorized, according to a standard protocol.Results: We identified 149 papers describing results from clinical trials in AD containing sufficient detail for our purposes. The largest proportion (38%) presented results of trials based on tests of cognition as the primary outcome measure. The primary analysis in most papers (85%) was a univariate significance test of a single primary outcome measure.Conclusions: The majority of trials reported a comparison of baseline and end-point assessment over relatively short patient follow-up periods, using univariate statistical methods to compare differences between intervention and control groups in the primary analysis. There is considerable scope to introduce newer statistical methods and trial designs in treatment evaluations in AD.


2012 ◽  
Vol 9 (1S) ◽  
pp. S45-S55 ◽  
Author(s):  
Jesse M. Cedarbaum ◽  
Mark Jaros ◽  
Chito Hernandez ◽  
Nicola Coley ◽  
Sandrine Andrieu ◽  
...  

2014 ◽  
Vol 24 (2) ◽  
pp. 254-271 ◽  
Author(s):  
Yuh-Jenn Wu ◽  
Te-Sheng Tan ◽  
Shein-Chung Chow ◽  
Chin-Fu Hsiao

2020 ◽  
Vol 21 (S21) ◽  
Author(s):  
Taeho Jo ◽  
◽  
Kwangsik Nho ◽  
Shannon L. Risacher ◽  
Andrew J. Saykin

Abstract Background Alzheimer’s disease (AD) is the most common type of dementia, typically characterized by memory loss followed by progressive cognitive decline and functional impairment. Many clinical trials of potential therapies for AD have failed, and there is currently no approved disease-modifying treatment. Biomarkers for early detection and mechanistic understanding of disease course are critical for drug development and clinical trials. Amyloid has been the focus of most biomarker research. Here, we developed a deep learning-based framework to identify informative features for AD classification using tau positron emission tomography (PET) scans. Results The 3D convolutional neural network (CNN)-based classification model of AD from cognitively normal (CN) yielded an average accuracy of 90.8% based on five-fold cross-validation. The LRP model identified the brain regions in tau PET images that contributed most to the AD classification from CN. The top identified regions included the hippocampus, parahippocampus, thalamus, and fusiform. The layer-wise relevance propagation (LRP) results were consistent with those from the voxel-wise analysis in SPM12, showing significant focal AD associated regional tau deposition in the bilateral temporal lobes including the entorhinal cortex. The AD probability scores calculated by the classifier were correlated with brain tau deposition in the medial temporal lobe in MCI participants (r = 0.43 for early MCI and r = 0.49 for late MCI). Conclusion A deep learning framework combining 3D CNN and LRP algorithms can be used with tau PET images to identify informative features for AD classification and may have application for early detection during prodromal stages of AD.


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