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Table of Contents

Table of Contents

Abstract
01. Presentation Overview
Presentation
01. Introduction02. Patient Features03. Image Processing Methods04. MRPI05. Gray Matter Density06. Regional Volumes07. Sample Size Analysis08. Diffusion MRI Processing09. Diffusion MRI Measures10. Diffusion MRI Sample Sizes11. Summary

Given that PSP is a rare disease, minimizing the number of participants in clinical trials is critical. The aim of this work was to identify the brain regions that require the lowest sample sizes to detect potential therapeutic effects for PSP clinical trials.   

By using both the exploratory voxelwise analysis and regional volumetric measurements of brain atrophy, we have shown:

  • The brain regions-of-interest that require the lowest sample sizes to detect potential therapeutic effects for PSP clinical trials.   
  • Subcortical regional volumes illustrate the rapid progression of PSP with statistically significant changes in atrophy as early as 6 months from baseline visit.
  • Assessment of the regional volume changes over time using a neuroimaging processing/analysis pipeline purpose-built for clinical trials can generate smaller sample size requirements than the planimetric biomarkers MRPI and MRPI 2.0 for monitoring disease progression.
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This work provides a thorough analysis of MRI data from the 4RTNI 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 PSP clinical trials.

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