Objective: To assess risk attitudes of patients suffering from multiple system atrophy (MSA) and Parkinson’s disease (PD) on future disease-modifying therapies as well as related clinical trials.

Background: MSA is the most devastating α-synucleinopathy characterized by fast disease progression and poor prognosis with no effective therapy available [1]. Currently, disease modifying therapeutic strategies in MSA and PD are on the verge to be clinically tested. Until today, there is no data available on patients’ opinion on future therapeutic approaches, on safety and tolerability issues or their willingness to take risks which are associated with potential side effects when participating in clinical trials.

Method: 50 patients with MSA and 50 patients with PD were recruited between March 2019 and March 2021 by a network of 9 university medical centres in Germany. Eligible participants met consensus criteria for possible or probable MSA or MDS diagnostic criteria for the clinical diagnosis of PD, respectively. Risk attitudes were measured by paper standard gamble scenarios [2, 3]. Patients completed a 28-item questionnaire about their view on the design and barriers to participate in clinical trials. Patient’s own evaluation of their illness was assessed by the MSA Quality of Life Questionnaire [4, 5]. In addition, the clinical phenotype, disease stage, and comorbidities were recorded.

Results: Data of 50 MSA and 50 PD patients will be available at the end of March 2021. Correlations between the willingness to take trial- and therapy-associated risks, disease type and severity will be calculated. Data on the percentages of patients accepting fatal adverse events after taking drugs, death after surgery, and their opinion on the most bearable as well as most severe side effects of drugs and surgery will be shown. Possible barriers preventing MSA and PD patients from participating in clinical trials will be presented. Reasons for the patients’ risk attitudes will be further discussed.

Conclusion: Our results could help improving future study designs by taking the patients’ willingness for risk-taking, attitudes towards an intervention, and perceived barriers into account.

References: [1] Wenning GK, Geser F, Krismer F, Seppi K, Duerr S, Boesch S, Köllensperger M, Goebel G, Pfeiffer KP, Barone P, et al. 2013. The natural history of multiple system atrophy: A prospective European cohort study. Lancet Neurol 12: 264 – 274. [2] Green, C., Brazier, J. & Deverill, M. Valuing health-related quality of life. A review of health state valuation techniques. PharmacoEconomics 17, 151–165 (2000). [3] Littenberg, B., Partilo, S., Licata, A. & Kattan, M. W. Paper Standard Gamble: The Reliability of a Paper Questionnaire to Assess Utility. Med. Decis. Making 23, 480–488 (2003). [4] Schrag, A. et al. Measuring health-related quality of life in MSA: The MSA-QoL. Mov. Disord. 22, 2332–2338 (2007). [5] Krismer, F. et al. MSA-QoL: spezifisches Bewertungsinstrument zur Erfassung der Lebensqualität bei Multisystematrophie: Validierung der deutschsprachigen Übersetzung. Nervenarzt 84, 709–714 (2013).

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