Objective: To evaluate multiple system atrophy (MSA) disease progression on a panel of candidate MSA biomarkers pre-selected for their putative involvement in the pathophysiology of the disease.

Background: A better characterisation of the natural history of MSA over 6 and 12 months on surrogate biomarkers is needed to better understand the disease, optimise future trial designs in terms of patient selection, sample size and trial duration, and improve our ability to measure the therapeutic effects of novel treatments.

Method: This is a French, multicentre, 1-year prospective longitudinal, non-drug study in 41 multiple system atrophy (MSA) patients and 20 matched healthy controls, examined at baseline, 6 months and 12 months. A panel of candidate MSA biomarkers derived using magnetic resonance imaging (MRI), dopamine transporter single-photon emission computed tomography (DaT-SPECT), blood and cerebrospinal fluid samples was evaluated in conjunction with a selection of well-validated clinical scales.

Results: 26 patients fulfilled consensus criteria for MSA-P and 15 for MSA-C. 8 patients accepted to have a lumbar puncture. Mean age of patients at baseline was 63.0 (SD 7.5) years. The average time since first symptom onset was 41.8 (SD 14.9) months. Patients had a total UMSARS (UMSARS1+2) score of 36.9 (SD 9.7) at baseline and 42.8 (SD 10.4) at 6 months. Preliminary descriptive results indicate changes in a number of biomarkers in the expected direction which are already observed at 6 months, although their significance remains to be confirmed. Results at 6 and 12 months derived from inferential statistics will be presented.

Conclusion: This is the first study to evaluate potential biomarkers of MSA disease progression over a six-month period.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/natural-history-and-disease-progression-biomarkers-of-multiple-system-atrophy-the-aspire-msa-study/