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@ARTICLE{Buchholz:259692,
      author       = {Buchholz, Maresa and Weber, Niklas and Borel, Stephanie and
                      Sayah, Sabrina and Xie, Feng and Schulz, Jörg B and Reetz,
                      Kathrin and Boesch, Sylvia and Klopstock, Thomas and Karin,
                      Ivan and Schöls, Ludger and Grobe-Einsler, Marcus and
                      Klockgether, Thomas and Davies, Elin Haf and Schmeder,
                      Madeleine and Nadke, Andreas and Michalowsky, Bernhard},
      title        = {{P}atient-reported, health economic and psychosocial
                      outcomes in patients with {F}riedreich ataxia ({PROFA}):
                      protocol of an observational study using momentary data
                      assessments via mobile health app.},
      journal      = {BMJ open},
      volume       = {13},
      number       = {8},
      issn         = {2044-6055},
      address      = {London},
      publisher    = {BMJ Publishing Group},
      reportid     = {DZNE-2023-00764},
      pages        = {e075736},
      year         = {2023},
      abstract     = {Friedreich ataxia (FA) is the most common hereditary ataxia
                      in Europe, characterised by progressively worsening movement
                      and speech impairments with a typical onset before the age
                      of 25 years. The symptoms affect the patients'
                      health-related quality of life (HRQoL) and psychosocial
                      health. FA leads to an increasing need for care, associated
                      with an economic burden. Little is known about the impact of
                      FA on daily lives and HRQoL. To fill that gap, we will
                      assess patient-reported, psychosocial and economic outcomes
                      using momentary data assessment via a mobile health
                      application (app).The PROFA Study is a prospective
                      observational study. Patients with FA (n=200) will be
                      recruited at six European study centres (Germany, France and
                      Austria). We will interview patients at baseline in the
                      study centre and subsequently assess the patients' health at
                      home via mobile health app. Patients will self-report ataxia
                      severity, HRQoL, speech and hearing disabilities, coping
                      strategies and well-being, health services usage, adverse
                      health events and productivity losses due to informal care
                      on a daily to monthly basis on the app for 6 months. Our
                      study aims to (1) validate measurements of HRQoL and
                      psychosocial health, (2) assess the usability of the mobile
                      health app, and (3) use descriptive and multivariate
                      statistics to analyse patient-reported and economic outcomes
                      and the interaction effects between these outcomes. Insights
                      into the app's usability could be used for future studies
                      using momentary data assessments to measure outcomes of
                      patients with FA.Ethical approval has been obtained from the
                      Ethics Committee of the University Medicine of Greifswald,
                      (BB096/22a, 26 October 2022) and from all local ethics
                      committees of the participating study sites. Findings of the
                      study will be published in peer-reviewed journals, presented
                      at relevant international/national congresses and
                      disseminated to German and French Patient Advocacy
                      Organizations.ClinicalTrials.gov Registry (NCT05943002);
                      Pre-results.},
      keywords     = {Humans / Adult / Friedreich Ataxia / Quality of Life /
                      Mobile Applications / Telemedicine: methods / Patient
                      Reported Outcome Measures / Observational Studies as Topic /
                      Friedreich ataxia (Other) / Rare diseases (Other) / health
                      and informal care (Other) / health economics (Other) /
                      m-health app assessment (Other) / patient-reported outcomes
                      (Other) / speech and hearing disabilities (Other)},
      cin          = {AG Hoffmann / AG Thyrian / AG Klockgether},
      ddc          = {610},
      cid          = {I:(DE-2719)1510600 / I:(DE-2719)1510800 /
                      I:(DE-2719)1011001},
      pnm          = {353 - Clinical and Health Care Research (POF4-353)},
      pid          = {G:(DE-HGF)POF4-353},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:37527887},
      pmc          = {pmc:PMC10394552},
      doi          = {10.1136/bmjopen-2023-075736},
      url          = {https://pub.dzne.de/record/259692},
}