A two-year clinical follow-up study in 80 CADASIL subjects: progression patterns and implications for clinical trials

Background and Purpose: CADASIL is a monogenic form of small vessel disease leading to stroke and dementia. The goal of this study was to explore the natural course of the disease, to check for prognostic variables, and to provide sample size estimates for future therapeutic trials. Methods: Eighty CADASIL subjects were followed prospectively over a mean period of 26.3+1.1 months. Standardized scales on disability (Rankin), activities of daily living (Barthel index), neurological outcome (NIH Stroke Scale) and cognitive performance (SIDAM and Mattis Dementia Rating Scale) were assessed at baseline and at follow-…