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@ARTICLE{Hermle:282538,
      author       = {Hermle, Dominik and Schubert, Robin and Barallon, Pascal
                      and Ilg, Winfried and Schüle, 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     = {BackgroundDigital-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.ObjectivesValidation
                      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.MethodsSingle-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.ResultsTwenty-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.ConclusionDigital 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.},
      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},
      doi          = {10.1002/mds.70012},
      url          = {https://pub.dzne.de/record/282538},
}