Given that the FTD variants have a complex overlapping set of clinical and anatomical phenotypes, identifying disease specific regions and minimizing the number of participants with disease subtypes in clinical trials is critical. An aim of this work was to identify the brain regions that require the lowest sample sizes to detect potential therapeutic effects for FTD clinical trials.
By using both the exploratory voxel- & vertex-wise analysie and regional volumetric measurements of brain atrophy, we have shown:
- The spatial & temporal complexity associated with brain atrophy in the FTD variants.
- The brain regions that require the lowest sample sizes to detect potential therapeutic effects for FTD clinical trials.
- The rapid progression of the svPPA population, where the hippocampus and temporal cortex have statistically significant changes in atrophy as early as 6 months from baseline.
This work provides a detailed analysis of MRI data from the FTLDNI study. Our results show that progressive, regional brain atrophy can be effectively assessed using quantitative analysis of MR images with reasonable sample sizes over a relatively short timeframe. As such, these imaging biomarkers appear to be promising for assessment of the efficacy of disease-modifying therapeutics in FTD clinical trials.