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Prof Matilde Inglese: Cerebellar volume as an outcome measure in therapeutic trials of primary progressive multiple sclerosis

Prof. Matilde Inglese

In this video, Prof Inglese discusses preliminary findings from a study on primary progressive MS. Her lab is attempting to identify new MRI markers that identify tissue changes over a relatively short period of time in patients with primary progressive MS. Currently, the number of gadolinium-enhancing lesions and the number of new T2-lesions are useful MRI markers for screening experimental treatments, but these markers are not sensitive enough in patients with primary progressive MS who are characterized by a lower accrual of T2 lesions.

The study cohort is a group of 26 patients with primary progressive MS. Clinical examination and MRI data was collected at baseline and after 12 months follow up. MRI markers assessed include number of T2 lesions, T1 lesion load, brain grey-matter & white matter volume, volume of the cerebellum and volume of the spinal cord. At 12 months, the cerebellum and spinal cord volumes had changed significantly, but only the volume of the cerebellum was significant from baseline at 12 months in patients who had sustained disability progression versus those who had not.

These results suggest that cerebellum volume measurements are sensitive enough to potentially act as a surrogate MRI marker for screening new treatments on a short-term basis in patients with primary progressive MS. Next steps are to reproduce the study in a larger cohort, and improve the software for measuring cerebellum volume (i.e., currently requires manual measurements). 

Professor Matilde Inglese, Neurology, Radiology and Neuroscience, Mount Sinai School of Medicine NY USA

This video was recorded at ECTRIMS 2016, September 14-17 2016, London, United Kingdom.