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@ARTICLE{Oender:257585,
      author       = {Oender, Demet and Faber, Jennifer and Wilke, Carlo and
                      Schaprian, Tamara and Lakghomi, Asadeh and Mengel, David and
                      Schöls, Ludger and Traschuetz, Andreas and Fleszar, Zofia
                      and Dufke, Claudia and Vielhaber, Stefan and Machts, Judith
                      and Giordano, Ilaria and Grobe-Einsler, Marcus and
                      Klopstock, Thomas and Stendel, Claudia and Boesch, Sylvia
                      and Nachbauer, Wolfgang and Timmann-Braun, Dagmar and
                      Thieme, Andreas Gustafsson and Kamm, Christoph and Dudesek,
                      Ales and Tallaksen, Chantal and Wedding, Iselin and Filla,
                      Alessandro and Schmid, Matthias and Synofzik, Matthis and
                      Klockgether, Thomas},
      title        = {{E}volution of {C}linical {O}utcome {M}easures and
                      {B}iomarkers in {S}poradic {A}dult-{O}nset {D}egenerative
                      {A}taxia.},
      journal      = {Movement disorders},
      volume       = {38},
      number       = {4},
      issn         = {0885-3185},
      address      = {New York, NY},
      publisher    = {Wiley},
      reportid     = {DZNE-2023-00461},
      pages        = {654 - 664},
      year         = {2023},
      abstract     = {Sporadic adult-onset ataxias without known genetic or
                      acquired cause are subdivided into multiple system atrophy
                      of cerebellar type (MSA-C) and sporadic adult-onset ataxia
                      of unknown etiology (SAOA).To study the differential
                      evolution of both conditions including plasma neurofilament
                      light chain (NfL) levels and magnetic resonance imaging
                      (MRI) markers.SPORTAX is a prospective registry of sporadic
                      ataxia patients with an onset >40 years. Scale for the
                      Assessment and Rating of Ataxia was the primary outcome
                      measure. In subgroups, blood samples were taken and MRIs
                      performed. Plasma NfL was measured via a single molecule
                      assay. Regional brain volumes were automatically measured.
                      To assess signal changes, we defined the pons and middle
                      cerebellar peduncle abnormality score (PMAS). Using
                      mixed-effects models, we analyzed changes on a time scale
                      starting with ataxia onset.Of 404 patients without genetic
                      diagnosis, 130 met criteria of probable MSA-C at baseline
                      and 26 during follow-up suggesting clinical conversion to
                      MSA-C. The remaining 248 were classified as SAOA. At
                      baseline, NfL, cerebellar white matter (CWM) and pons
                      volume, and PMAS separated MSA-C from SAOA. NfL decreased in
                      MSA-C and did not change in SAOA. CWM and pons volume
                      decreased faster, whereas PMAS increased faster in MSA-C. In
                      MSA-C, pons volume had highest sensitivity to change, and
                      PMAS was a predictor of faster progression. Fulfillment of
                      possible MSA criteria, NfL and PMAS were risk factors, CWM
                      and pons volume protective factors for conversion to
                      MSA-C.This study provides detailed information on
                      differential evolution and prognostic relevance of
                      biomarkers in MSA-C and SAOA. © 2023 The Authors. Movement
                      Disorders published by Wiley Periodicals LLC on behalf of
                      International Parkinson and Movement Disorder Society.},
      keywords     = {Humans / Adult / Cerebellar Ataxia: diagnosis / Ataxia:
                      genetics / Cerebellum / Multiple System Atrophy: diagnosis /
                      Biomarkers / multiple system atrophy (Other) / natural
                      history (Other) / neurofilament light chain (Other) /
                      sporadic ataxia (Other) / volumetric MRI (Other) /
                      Biomarkers (NLM Chemicals)},
      cin          = {AG Klockgether / Clinical Research Platform (CRP) / Patient
                      Studies Bonn / AG Gasser / AG Schmid Bonn / AG Schöls /
                      Clinical Research (Munich) / AG Höglinger 1 / AG Düzel},
      ddc          = {610},
      cid          = {I:(DE-2719)1011001 / I:(DE-2719)1011401 /
                      I:(DE-2719)1011101 / I:(DE-2719)1210000 / I:(DE-2719)1013028
                      / I:(DE-2719)5000005 / I:(DE-2719)1111015 /
                      I:(DE-2719)1110002 / I:(DE-2719)5000006},
      pnm          = {353 - Clinical and Health Care Research (POF4-353)},
      pid          = {G:(DE-HGF)POF4-353},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:36695111},
      doi          = {10.1002/mds.29324},
      url          = {https://pub.dzne.de/record/257585},
}