Inglese M, Petracca M, Mormina E, Achiron A, Straus-Farber R, Miron S, Fabian M, Krieger S, Miller A, Lublin F, Sormani MP. Cerebellar volume as imaging outcome in progressive multiple sclerosis. PLoS One. 2017 Apr 24;12(4):e0176519.
BACKGROUND AND PURPOSE: To assess whether cerebellar volumes changes could represent a sensitive outcome measure in primary-progressive MS.
MATERIAL AND METHODS: Changes in cerebellar volumes over one-year follow-up, estimated in 26 primary-progressive MS patients and 20 controls with Freesurfer longitudinal pipeline, were assessed using Wilcoxon test and tested for their correlation with disability worsening by a logistic regression. Clinical worsening was defined as EDSS score increase or change of >20% for 25-foot walk test or 9-hole peg test scores at follow-up. Sample sizes for given treatment effects and power were calculated. The findings were validated in an independent cohort of 20 primary-progressive MS patients.
RESULTS: Significant changes were detected in brain T1 lesion volume (p<0.01), cerebellar T2 and T1 lesion volume (p<0.01 and p<0.05), cerebellar volume, cerebellar cortex volume, and cerebellar WM volume (p<0.001). Only cerebellar volume and cerebellar cortex volume percentage change were significantly reduced in clinically progressed patients when compared to patients who did not progress (p<0.01; respectively AUC of 0.91 and 0.96). Cerebellar volume percentage changes were consistent in the exploration and validation cohorts (cerebellar volume -1.90±1.11% vs -1.47±2.30%; cerebellar cortex volume -1.68±1.41% vs -1.56±2.23%). Based on our results the numbers of patients required to detect a 30% effect are 81 per arm for cerebellar volume and 162 per arm for cerebellar cortex volume (90% power, type 1 error alpha = 0.05).
CONCLUSIONS: Our results suggest a role for cerebellar cortex volume and cerebellar volume as potential short-term imaging metrics to monitor treatment effect in primary-progressive MS clinical trials.
Speeding up trials is the quickest way to get treatments to people. This study suggests that the volume of the cerebellum, which is an region controlling movement and balance, shrinks
Labels: progressive MS