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@ARTICLE{Buchholz:279169,
      author       = {Buchholz, Maresa and Pfaff, Michelle and Iskandar, Audrey
                      and Reetz, Kathrin and Schulz, Jörg B and Grobe-Einsler,
                      Marcus and Klockgether, Thomas and Michalowsky, Bernhard},
      collaboration = {Group, EFACTS Study},
      othercontributors = {Boesch, Sylvia and Nachbauer, Wolfgang and Eigentler,
                          Andreas and Indelicato, Elisabetta and Amprosi, Matthias and
                          Rai, Myriam and Pandolfo, Massimo and Mariotti, Caterina and
                          Nanetti, Lorenzo and Gellera, Cinzia and Mongelli, Alessia
                          and Castaldo, Anna and Fichera, Mario and Bertini, Enrico
                          and Vasco, Gessica and Durr, Alexandra and Ewenczyk, Claire
                          and Biet, Marie and Monin, Marie Lorraine and Holtbernd,
                          Florian and Brcina, Nikolina and Hohenfeld, Christian and
                          Didszun, Claire and Dogan, Imis and Giordano, Ilaria and
                          Klopstock, Thomas and Stendel, Claudia and Radelfahr,
                          Florentine and Bischoff, Almut and Schöls, Ludger and
                          Hayer, Stefanie and Koutsis, Georgios and Breza, Marianthi
                          and Palau, Francesc and O'Callaghan, Mar and de Rivera
                          Garrido, Francisco Javier Rodríguez and Rummey, Christian
                          and Giunti, Paola and Parkinson, Michael H and Thomas-Black,
                          Gilbert and Manso, Katarina and Solanky, Nita and Labrum,
                          Robyn},
      title        = {{H}ealth-{R}elated {Q}uality of {L}ife in {P}atients with
                      {F}riedreich {A}taxia {U}sing {M}obility {A}ssistive
                      {T}echnologies: {L}imited {F}it of the {EQ}-5{D}-3{L}
                      {M}obility {D}imension.},
      journal      = {Neurology and Therapy},
      volume       = {14},
      number       = {1},
      issn         = {2193-6536},
      address      = {Heidelberg [u.a.]},
      publisher    = {Springer},
      reportid     = {DZNE-2025-00697},
      pages        = {379 - 398},
      year         = {2025},
      abstract     = {Friedreich Ataxia (FA) is a multisystem neurodegenerative
                      disease. Affected individuals rely on mobility assistive
                      technologies (MAT) (e.g. wheelchairs) and require long-term
                      treatments and care. To analyse the patients' health-related
                      quality of life (HRQoL), the EuroQol 5 Dimension 3 Level
                      survey (EQ-5D-3L)-a widely used and recommended generic
                      measure-is used in clinical and in health economic studies.
                      Concerns about using the instrument in mobility-impaired
                      individuals who might have difficulties finding appropriate
                      response options for mobility-related items led us to
                      investigate how the 3L dimensions perform in patients with
                      FA using or not using MAT.Using longitudinal data from 607
                      patients with FA of the EFACTS study (from baseline to the
                      3-year follow-up), we analysed the acceptability,
                      distribution properties, validity, and responsiveness of the
                      EQ-5D-3L, focusing on the mobility item. Analyses were
                      stratified for patients without and with different
                      MAT-usage.We identified that n = 177 patients used no MAT, n
                      = 299 a wheelchair and n = 131 walking aids. The mobility
                      item non-response was highest in wheelchair users $(6.8\%)$
                      and lowest in patients without MAT. Walking aid users showed
                      the least variability, all selecting the mid-response option
                      'some problems' for mobility. The mobility item correlated
                      moderately with disease severity (rsp = 0.35) and the
                      activities of daily living scale (rsp = 0.36) in wheelchair
                      users. No correlation exists for walking aid users. The
                      strongest health changes occurred for wheelchair users, the
                      weakest for walking aid users. The mobility dimensions
                      showed the highest amount of no changes.The EQ-5D-3L's
                      mobility item has limitations in MAT users, particularly in
                      walking aid users, due to a tendency towards mid-responses.
                      These limitations may affect the efficacy and
                      (cost)effectiveness conclusions drawn from interventions and
                      clinical trials with mobility-impaired individuals. Further
                      research is needed to explore the understanding and
                      interpretation of responses of the EQ-5D in patients with FA
                      with mobility support.ClinicalTrials.gov identifier
                      NCT02069509.},
      keywords     = {EQ-5D-3L (Other) / Friedreich Ataxia (Other) /
                      Health-related quality of life (Other) / Mobility assistive
                      technology (Other)},
      cin          = {AG Michalowsky / AG Thyrian / AG Hoffmann / Clinical
                      Research (Bonn) / Patient Studies (Bonn)},
      ddc          = {610},
      cid          = {I:(DE-2719)5000067 / I:(DE-2719)1510800 /
                      I:(DE-2719)1510600 / I:(DE-2719)1011001 /
                      I:(DE-2719)1011101},
      pnm          = {353 - Clinical and Health Care Research (POF4-353)},
      pid          = {G:(DE-HGF)POF4-353},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:39738982},
      pmc          = {pmc:PMC11762039},
      doi          = {10.1007/s40120-024-00694-7},
      url          = {https://pub.dzne.de/record/279169},
}