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@ARTICLE{Seemann:280027,
      author       = {Seemann, Jens and Beyme, Theresa and John, Natalie and
                      Harmuth, Florian and Giese, Martin and Schöls, Ludger and
                      Timmann, Dagmar and Synofzik, Matthis and Ilg, Winfried},
      title        = {{C}apture of {L}ongitudinal {C}hange in {R}eal-{L}ife
                      {W}alking in {C}erebellar {A}taxia {I}ncreases {P}atient
                      {R}elevance and {E}ffect {S}ize.},
      journal      = {Movement disorders},
      volume       = {40},
      number       = {7},
      issn         = {0885-3185},
      address      = {New York, NY},
      publisher    = {Wiley},
      reportid     = {DZNE-2025-00871},
      pages        = {1343 - 1355},
      year         = {2025},
      abstract     = {With disease-modifying drugs for degenerative ataxias on
                      the horizon, ecologically valid measures of gait performance
                      that can detect patient-relevant changes in trial-like time
                      frames are highly warranted.In this 2-year longitudinal
                      study, we aimed to unravel ataxic gait measures sensitive to
                      longitudinal changes in patients' real lives using wearable
                      sensors.We assessed longitudinal gait changes of 26
                      participants with degenerative cerebellar disease (Scale for
                      the Assessment and Rating of Ataxia [SARA]: 9.4 ± 4.1) at
                      baseline, 1-year, and 2-year follow-up using three body-worn
                      inertial sensors in two conditions: (1) laboratory-based
                      walking (LBW); and (2) real-life walking (RLW). In RLW, a
                      context-sensitive analysis was performed by selecting
                      comparable walking bouts according to macroscopic gait
                      characteristics. Gait analysis focused on measures of
                      spatio-temporal variability, particularly stride length
                      variability, lateral step deviation, and a compound measure
                      of spatial variability (SPCmp).Gait variability measures
                      showed high test-retest reliability in both walking
                      conditions (intraclass correlation coefficient [ICC],
                      ≥0.82). Cross-sectional analyses revealed high
                      correlations of gait measures with ataxia severity (SARA,
                      effect size ρ ≥ 0.75); and with patients' subjective
                      balance confidence (Activity-specific Balance Confidence
                      scale [ABC]: ρ ≥ 0.71). Although SARA showed longitudinal
                      changes only after 2 years, the gait measure SPCmp revealed
                      changes after 1 year with high effect size (rprb = 0.80).
                      Sample size estimation for the gait measure SPCmp showed a
                      required cohort size of n = 42 participants (n = 38;
                      spinocerebellar ataxias [SCA]1/2/3 subgroup) to detect a
                      $50\%$ reduction in progression at 1 year with a
                      hypothetical intervention, compared to n = 147 for SARA at 2
                      years.Because of their ecological validity and larger effect
                      sizes, real-life gait characteristics represent promising
                      performance measures as outcomes for future treatment
                      trials. © 2025 The Author(s). Movement Disorders published
                      by Wiley Periodicals LLC on behalf of International
                      Parkinson and Movement Disorder Society.},
      keywords     = {Humans / Female / Male / Cerebellar Ataxia: physiopathology
                      / Cerebellar Ataxia: complications / Longitudinal Studies /
                      Middle Aged / Aged / Walking: physiology / Gait Analysis /
                      Gait Disorders, Neurologic: physiopathology / Gait
                      Disorders, Neurologic: etiology / Reproducibility of Results
                      / Disease Progression / Gait: physiology / biomarker (Other)
                      / cerebellar ataxia (Other) / digital health (Other) /
                      real‐life walking (Other) / wearable sensors (Other)},
      cin          = {AG Schöls / AG Gasser},
      ddc          = {610},
      cid          = {I:(DE-2719)5000005 / 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:40395207},
      pmc          = {pmc:PMC12273614},
      doi          = {10.1002/mds.30230},
      url          = {https://pub.dzne.de/record/280027},
}