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Willingness to participate in natural history studies amongst patients with sporadic inclusion body myositis (sIBM): A comparison between the United States and Australia

Mathew, V., Habib, A., Goyal, N., Goldberg, R., Needham, M. and Mozaffar, T. (2019) Willingness to participate in natural history studies amongst patients with sporadic inclusion body myositis (sIBM): A comparison between the United States and Australia. Neurology, 92 (15S).

Abstract

Objective: To gauge patient attitudes toward natural history studies, particularly ones that involve invasive procedures, like muscle biopsies, in sporadic inclusion body myositis across two countries with distinct healthcare models.

Background: Defining natural history of sporadic inclusion body myositis (sIBM) is vital for designing effective clinical trials. Given the recent identification of serological markers in patients with this disease, it is important to understand if natural history and disease behavior is variable based on serological status. We studied sIBM patients in the USA and Australia to assess their attitudes towards natural history studies.

Design/Methods: An 8-item survey was developed on Google Forms and disseminated through The Myositis Association for the two regions studied. The items measured satisfaction with current sIBM therapeutics, perception towards natural history studies, and willingness to complete on-site visits and various procedures.

Results: A total of 646 (539 USA, 107 AUS) sIBM patients responded to the survey. Approximately 91% of respondents (89% USA, 93% AUS) were willing to partake in a natural history study with biannual visits involving physical assessments, questionnaires, and serum collections for DNA analyses. The more involved procedures such as the muscle biopsy and DNA testing were, on average, accepted by 75% and 97% of respondents, respectively. Self-pay for study-related expenses (e.g., meals, parking) deterred 26% of all respondents from participating in the proposed study.

Conclusions: Our survey demonstrated an unmet need in sIBM independent of geographic variation. Additionally, the difference in responses received from American and Australian sIBM patients lead us to consider the impact of the combined universal and private insurance platform utilized in Australia on the higher satisfaction with treatment options scale. Regardless, the vast majority of all respondents were willing to participate in a complex natural history studies because they are unsatisfied with current options.

Item Type: Journal Article
Publisher: American Academy of Neurology
Copyright: © 2019 American Academy of Neurology
Publishers Website: https://n.neurology.org/content/92/15_Supplement/P...
URI: http://researchrepository.murdoch.edu.au/id/eprint/50157
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