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@ARTICLE{Willemse:282584,
      author       = {Willemse, Ilse H J and Mellone, Sabato and Tacconi, Carlo
                      and Santorelli, Filippo M and Ricca, Ivana and Satolli, Sara
                      and Klebe, Stephan and Jeschonneck, Nicole and Ilg, Winfried
                      and Schüle-Freyer, Rebecca and Synofzik, Matthis and
                      Nonnekes, Jorik and van de Warrenburg, Bart P C},
      title        = {{F}easibility of a smartphone application for remote use in
                      spastic ataxias: an 8-week long-term {PROSPAX} study.},
      journal      = {Journal of neurology},
      volume       = {273},
      number       = {1},
      issn         = {0367-004X},
      address      = {[Darmstadt]},
      publisher    = {Steinkopff},
      reportid     = {DZNE-2025-01344},
      pages        = {3},
      year         = {2025},
      abstract     = {Digital biomarkers show significant potential and are
                      emerging as outcome measures in clinical trials in spastic
                      ataxias (SPAX). Research on the performances of digital
                      biomarkers in home environments remains limited.To evaluate
                      feasibility, user adherence, time-of-day and learning
                      effects, and consistency across settings of the SPAX-app for
                      home monitoring in clinical trials. The app contains four
                      tasks assessing gait, standing balance, and finger and hand
                      movements, along with a patient-reported outcome measure
                      (PROM).We conducted an 8-week long-term study in 38 SPAX
                      patients and 10 healthy controls. Subjects first answered
                      the SPAX-app PROM and performed tasks during one in-clinic
                      session. Severity of ataxia and spasticity were assessed by
                      clinician-reported outcome measures (ClinROs). Subjects then
                      performed the tasks and answered the PROM twice a week in
                      the morning and evening at home.The SPAX-app showed high
                      feasibility for finger and hand movement tasks $(92\%),$ but
                      limited feasibility for gait $(47\%)$ and stance $(63\%)$
                      tasks. Among those able to perform the tasks, adherence was
                      high $(81\%).$ A significant difference (p < 0.01) was found
                      between morning and evening assessments by PROMs in SPAX.
                      Results remained stable across the day and repeated
                      assessments, except for the inter-onset interval in finger
                      tapping (p = 0.002 and p = 0.020, respectively). Strong
                      correlations were observed between tasks performed at home
                      and in the clinic. Motor outcome measures correlated well
                      with ClinROs, but not with PROMs.The SPAX-app is a feasible
                      tool for remote assessment in SPAX, showing high feasibility
                      for finger and hand movement tasks, but limited feasibility
                      for gait and stance tasks at home. Time-of-day and learning
                      effects were minimal. Longer-term studies are needed to
                      assess clinical relevance.},
      keywords     = {Humans / Male / Female / Feasibility Studies / Middle Aged
                      / Adult / Smartphone / Mobile Applications / Muscle
                      Spasticity: diagnosis / Muscle Spasticity: physiopathology /
                      Aged / Patient Reported Outcome Measures / Longitudinal
                      Studies / Postural Balance: physiology / Young Adult /
                      Digital biomarkers (Other) / Long-term study (Other) /
                      Remote monitoring (Other) / Smartphone application (Other) /
                      Spastic ataxia (Other)},
      cin          = {AG Gasser},
      ddc          = {610},
      cid          = {I:(DE-2719)1210000},
      pnm          = {353 - Clinical and Health Care Research (POF4-353)},
      pid          = {G:(DE-HGF)POF4-353},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:41342937},
      doi          = {10.1007/s00415-025-13514-1},
      url          = {https://pub.dzne.de/record/282584},
}