Brain atrophy staging in spinocerebellar ataxia type 3 for clinical prognosis and trial enrichment.

ABSTRACT:

Background

Spinocerebellar ataxia type 3 (SCA3) is characterised by progressive brain atrophy, with regional volume loss detectable via MRI prior to clinical manifestation. We aimed to identify the previously unknown sequence of brain atrophy in SCA3 and evaluate whether this sequence can be translated into an atrophy staging framework to enable accurate clinical prognosis and trial enrichment.

Methods

We included data from 322 SCA3 mutation carriers, enrolled in observational studies conducted across Europe, the Americas, and Asia. Participants underwent follow-up assessments up to five years after baseline. The Subtype and Stage Inference machine learning algorithm was applied to estimate the most likely atrophy sequence(s) from baseline anatomical MRI. The Scale for the A

SUBMITTER: Baumeister H 

PROVIDER: S-EPMC12800623 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature