Objective: To investigate the usefulness of a symptom assessment scale for multiple system atrophy (MSA) in Hokkaido, Japan from the data of the Hokkaido Rare-disease Consortium for MSA (HoRC-MSA).

Background: MSA is a refractory neurodegenerative disease, but novel treatments can be expected in the future. In clinical trials, a sufficient number of patients and appropriate outcome measures will be needed. We started a patient registry to investigate the natural history of MSA in preparation for clinical trials. Previous studies have suggested the possibility of a useful symptom assessment scale in the early stage of MSA.

Methods: We continued recruitment of MSA patients in Hokkaido after approval from the Institutional review board of Hokkaido University Hospital in November 2014. Postal surveys to medical facilities and MSA patients were conducted. Score changes and standardized response mean (SRM) of symptom assessment scales were evaluated.

Results: One hundred and fifty-seven patients agreed to participate in the study until October 31, 2017. Eighty-three patients were evaluated by clinical assessment scales. Annual score changes of Unified MSA Rating Scale (UMSARS) part 2 was 4 points. SRM of UMSARS part 2 was high. SRMs of items related to walking and standing were high in possible MSA.

Conclusions: A survey using HoRC-MSA provided novel epidemiological knowledge for MSA. Evaluation related to walking and standing was useful indicators in clinical trials.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/usefulness-of-a-simple-symptom-assessment-scale-for-multiple-system-atrophy-data-from-horc-msa-project/