Objective: To investigate epidemiological aspects of multiple system atrophy (MSA) in Hokkaido, Japan from the data of the Hokkaido Rare-disease Consortium for MSA (HoRC-MSA).

Background: MSA is a progressive neurodegenerative disease, but novel treatments can be expected in the future. We started a patient registry to investigate the natural history of MSA in preparation for clinical trials. We analyzed epidemiological data accumulated for about 4 years.

Method: We have been continuing recruitment of MSA patients in Hokkaido after approval from the Institutional review board of our hospital in November 2014. Postal surveys to medical facilities and MSA patients were conducted. Amassed data were analyzed after being anonymous linked.

Results: One hundred and seventy-four patients agreed to participate in the study until January 31, 2019. Among them, 165 patients were assessed their activity of daily living and diagnostic criteria. The average age at the time of inclusion was 66.4 ± 11.0 years old. The number of MSA-C cases was about twice as much as MSA-P cases, and 57% of those patients had difficulty walking. As of Feb 2019, 26 participants have been followed for 4 years. Clinical assessment scales were also evaluated only for ambulatory patients. Unified MSA rating scale part 2 score increased 2 to 5 points / year. Although 97 participants were examined at the first time, only 6 patients could continue for 4 years.

Conclusion: HoRC-MSA is useful for investigating natural history of MSA and establishing infrastructure of clinical trials for MSA. Long-term follow-up of MSA is accompanied by difficulties, so some ingenuity is required to successfully continue the cohort study.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/epidemiological-study-of-multiple-system-atrophy-data-from-horc-msa-project-2014-2019/