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@ARTICLE{Bernhardt:271338,
      author       = {Bernhardt, Alexander Maximilian and Oeller, Marc and
                      Friedrich, Isabel and Kocakavuk, Emre and Nachman, Eliana
                      and Peikert, Kevin and Roderigo, Malte and Rossmann, Andreas
                      and Schröter, Tabea and Wilhelm, Lea Olivia and Prell, Tino
                      and van Riesen, Christoph and Nieweler, Johanna and
                      Katzdobler, Sabrina and Weiler, Markus and Jacobi, Heike and
                      Warnecke, Tobias and Claus, Inga and Palleis, Carla and
                      Breimann, Stephan and Falkenburger, Björn and Brandt,
                      Moritz and Hermann, Andreas and Rumpf, Jost-Julian and
                      Claßen, Joseph and Höglinger, Günter and Gandor, Florin
                      and Levin, Johannes and Giese, Armin and Janzen, Annette and
                      Oertel, Wolfgang Hermann},
      title        = {{R}isk willingness in multiple system atrophy and
                      {P}arkinson's disease understanding patient preferences.},
      journal      = {npj Parkinson's Disease},
      volume       = {10},
      number       = {1},
      issn         = {2373-8057},
      address      = {London [u.a.]},
      publisher    = {Nature Publ. Group},
      reportid     = {DZNE-2024-01039},
      pages        = {158},
      year         = {2024},
      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.},
      cin          = {Clinical Research (Munich) / AG Fischer / AG Haass / AG
                      Falkenburger / AG Hermann / AG Levin},
      ddc          = {610},
      cid          = {I:(DE-2719)1111015 / I:(DE-2719)1410002 /
                      I:(DE-2719)1110007 / I:(DE-2719)1710012 / I:(DE-2719)1511100
                      / I:(DE-2719)1111016},
      pnm          = {353 - Clinical and Health Care Research (POF4-353) / 352 -
                      Disease Mechanisms (POF4-352)},
      pid          = {G:(DE-HGF)POF4-353 / G:(DE-HGF)POF4-352},
      typ          = {PUB:(DE-HGF)16},
      pmc          = {pmc:PMC11327309},
      pubmed       = {pmid:39147806},
      doi          = {10.1038/s41531-024-00764-5},
      url          = {https://pub.dzne.de/record/271338},
}