Risk willingness in multiple system atrophy and Parkinson’s disease understanding patient preferences
Year: 2024
Type: article
Study design: Observational Study
Abstract: Disease-modifying therapeutics in the α-synucleinopathies multiple system atrophy (MSA) and Parkinson's Disease (PD) are in early phases of clinical testing. Involving patients' preferences including therapy-associated risk willingness in initial stages of therapy development has been increasingly pursued in regulatory approval processes. In our study with 49 MSA and 38 PD patients, therapy-associated risk willingness was quantified using validated standard gamble scenarios for varying severities of potential drug or surgical side effects. Demonstrating a non-gaussian distribution, risk willingness varied markedly within, and between groups. MSA patients accepted a median 1% risk [interquartile range: 0.001-25%] of sudden death for a 99% [interquartile range: 99.999-75%] chance of cure, while PD patients reported a median 0.055% risk [interquartile range: 0.001-5%]. Contrary to our hypothesis, a considerable proportion of MSA patients, despite their substantially impaired quality of life, were not willing to accept increased therapy-associated risks. Satisfaction with life situation, emotional, and nonmotor disease burden were associated with MSA patients' risk willingness in contrast to PD patients, for whom age, and disease duration were associated factors. An individual approach towards MSA and PD patients is crucial as direct inference from disease (stage) to therapy-associated risk willingness is not feasible. Such studies may be considered by regulatory agencies in their approval processes assisting with the weighting of safety aspects in a patient-centric manner. A systematic quantitative assessment of patients' risk willingness and associated features may assist physicians in conducting individual consultations with patients who have MSA or PD by facilitating communication of risks and benefits of a treatment option.
Source: npj Parkinson s Disease
Authors: Alexander Maximilian Bernhardt, Marc Oeller, Isabel Friedrich, Emre Kocakavuk, Eliana Nachman, Kevin Peikert, Malte Roderigo, Andreas Rossmann, Tabea Schröter, Lea Olivia Wilhelm, Tino Prell, Christoph van Riesen, Johanna Nieweler, Sabrina Katzdobler, Markus Weiler, Heike Jacobi, Tobias Warnecke, Inga Claus, Carla Palleis, Stephan Breimann, Björn Falkenburger, Moritz Brandt, Andreas Hermann, Jost-Julian Rumpf, Joseph Claßen, Günter Höglinger, Florin Gandor, Johannes Levin, Armin Giese, Annette Janzen, Wolfgang Hermann Oertel
Institutions: German Center for Neurodegenerative Diseases, Ludwig-Maximilians-Universität München, Max Planck Institute of Biochemistry, University Hospital Leipzig, Leipzig University, Essen University Hospital, VIB-KU Leuven Center for Brain & Disease Research, KU Leuven, Universitätsmedizin Rostock, University of Rostock, University of Münster, University Hospital Münster, Jena University Hospital, MSB Medical School Berlin, University Hospital in Halle, Universitätsmedizin Göttingen, Heidelberg University, University Hospital Heidelberg, Klinikum Osnabrück, Munich Cluster for Systems Neurology, Technical University of Munich, German Center for Neurodegenerative Diseases (DZNE) Dresden, Technische Universität Dresden, Neurologisches Fachkrankenhaus für Bewegungsstörungen/Parkinson, Otto-von-Guericke-Universität Magdeburg, Institut für Neuropathologie, Philipps University of Marburg, Helmholtz Munich
Language: English
Subfield: Neurology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $3,590
OpenAlex ID: W4401614108