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@ARTICLE{Hermle:282538,
      author       = {Hermle, Dominik and Schubert, Robin and Barallon, Pascal
                      and Ilg, Winfried and Schüle-Freyer, Rebecca and Reilmann,
                      Ralf and Synofzik, Matthis and Traschütz, Andreas},
      title        = {{D}igital {O}utcomes of {U}pper {L}imb {A}taxia {C}apture
                      {M}eaningful {L}ongitudinal {C}hange and {T}reatment
                      {R}esponse},
      journal      = {Movement disorders},
      volume       = {40},
      number       = {11},
      issn         = {0885-3185},
      address      = {New York, NY},
      publisher    = {Wiley},
      reportid     = {DZNE-2025-01301},
      pages        = {2486 - 2496},
      year         = {2025},
      abstract     = {Digital-motor outcomes promise better responsiveness than
                      clinician-reported outcomes in ataxia trials. However, their
                      patient meaningfulness and sensitivity to change remain to
                      be demonstrated, particularly in the upper limb
                      domain.Validation of quantitative motor (Q-Motor) assessment
                      for upper limb ataxia against patient-reported outcomes and
                      regarding sensitivity to both longitudinal and
                      treatment-induced change, the latter in n-of-1 treatment
                      settings.Single-center longitudinal assessment of finger
                      tapping, diadochokinesia, grip-lift, spiral drawing, and
                      target reaching in (1) 36 cross-genotype ataxia patients and
                      20 controls, validating digital measures for correlations
                      with patient-reported outcome measure (PROM)-ataxia, 2-weeks
                      test-retest reliability, and sensitivity to change within a
                      trial-relevant 1-year follow-up, anchored in Patient Global
                      Impression of Change (PGI-C); and (2) two patients with
                      spinocerebellar ataxia type 27B (SCA27B) on versus off
                      treatment with 4-aminopyridine.Twenty-four digital measures
                      correlated with the PROM-ataxia upper-limb composite (|ρ| =
                      0.4-0.7) and had excellent test-retest reliability (ICC =
                      0.91-0.99). Correlations to individual PROM-ataxia items
                      were specific for functional impairment the respective
                      measure was hypothesized to capture. Speed of finger tapping
                      and diadochokinesia, and smoothness of target reaching
                      (spectral arc length of movement in three dimensions
                      [SPARC3D]) captured 1-year progression in ataxia patients
                      (|rprb| = 0.38-0.51), and specifically in patients with
                      worsening PGI-C. Estimated sample sizes to detect
                      longitudinal change were lower for digital than clinical
                      outcomes (SPARC3D: n = 33, Scale for the Assessment and
                      Rating of Ataxia (SARA): n = 79, nine-hole peg-test: n =
                      214). Speed of diadochokinesia, stability of grip-lift, and
                      variability of target reaching captured treatment responses
                      to 4-aminopyridine in SCA27B, exceeding minimal detectable
                      and minimal important change.Digital upper limb measures
                      capture patient-meaningful 1-year longitudinal and
                      treatment-induced change, and are therefore promising
                      outcomes for upcoming ataxia trials. © 2025 The Author(s).
                      Movement Disorders published by Wiley Periodicals LLC on
                      behalf of International Parkinson and Movement Disorder
                      Society.},
      keywords     = {Humans / Male / Female / Middle Aged / Upper Extremity:
                      physiopathology / Longitudinal Studies / Adult / Aged /
                      Patient Reported Outcome Measures / Ataxia: physiopathology
                      / Ataxia: drug therapy / Reproducibility of Results /
                      Treatment Outcome / Spinocerebellar Ataxias: physiopathology
                      / Spinocerebellar Ataxias: drug therapy / Fingers:
                      physiopathology / ataxia; clinical outcome assessment;
                      digital motor outcome; patient meaningfulness; sensitivity
                      to change (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:40888004},
      pmc          = {pmc:PMC12661631},
      doi          = {10.1002/mds.70012},
      url          = {https://pub.dzne.de/record/282538},
}