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@ARTICLE{Klockgether:258692,
      author       = {Klockgether, Thomas and Synofzik, Matthis and COAs, AGI
                      working group on},
      collaboration = {Registries},
      othercontributors = {Alhusaini, Saud and Anheim, Mathieu and Antonijevic, Irina
                          and Ashizawa, Tee and Bataller, Luis and Berard, Mélanie
                          and Bertini, Enrico and Boesch, Sylvia and Braga-Neto, Pedro
                          and Cassou, Emanuel and Chan, Edwin and Chuang, Rosalind and
                          Collins, Abbie and Damásio, Joana and Donis, Karina and
                          Duquette, Antoine and Durães, João and Durr, Alexandra and
                          Evans, Rebecca and Faber, Jennifer and Farmer, Jennifer and
                          Gennarino, Vincenzo and Graessner, Holm and Grobe-Einsler,
                          Marcus and Hanagasie, Hasmet and Heidari, Morteza and
                          Houlden, Henry and Indelicato, Elisabetta and Ishikawa,
                          Kinya and Jacobi, Heike and Jardim, Laura and Kisanuki, Yaz
                          and Kopishinskaia, Svetlana and L Italien, Gilbert and Maas,
                          Roderick and Mancuso, Michelangelo and Mariotti, Caterina
                          and Ibrahim, Norlinah Mohamed and Nachbauer, Wolfgang and
                          Nemeth, Andrea and Ng, Yi Shiau and Obieglo, Katja and
                          Onodera, Osamu and Opal, Puneet and de Almeida, Luis Pereira
                          and Perlman, Susan and Primiano, Guido and Renaud, Mathilde
                          and Rosenthal, Liana and Saccà, Francesco and Sattar, Zahid
                          and Schmitz-Hübsch, Tanja and Schöls, Ludger and
                          Schüle-Freyer, Rebecca and Seeberger, Lauren and Silvestri,
                          Gabriella and Sobanska, Anna and Soong, Bin-Weng and
                          Srivastava, Achal Kumar and Stoyas, Colleen and du Montcel,
                          Sophie Tezenas and Thieme, Andreas and Timmann, Dagmar and
                          Tocoian, Adina and Traschuetz, Andreas and van de
                          Warrenburg, Bart and Ziegler, Wolfram},
      title        = {{C}onsensus {R}ecommendations for {C}linical {O}utcome
                      {A}ssessments and {R}egistry {D}evelopment in {A}taxias:
                      {A}taxia {G}lobal {I}nitiative ({AGI}) {W}orking {G}roup
                      {E}xpert {G}uidance.},
      journal      = {The Cerebellum},
      volume       = {23},
      number       = {3},
      issn         = {1473-4222},
      address      = {London},
      publisher    = {Dunitz},
      reportid     = {DZNE-2023-00665},
      pages        = {924 - 930},
      year         = {2024},
      abstract     = {To accelerate and facilitate clinical trials, the Ataxia
                      Global Initiative (AGI) was established as a worldwide
                      research platform for trial readiness in ataxias. One of
                      AGI's major goals is the harmonization and standardization
                      of outcome assessments. Clinical outcome assessments (COAs)
                      that describe or reflect how a patient feels or functions
                      are indispensable for clinical trials, but similarly
                      important for observational studies and in routine patient
                      care. The AGI working group on COAs has defined a set of
                      data including a graded catalog of COAs that are recommended
                      as a standard for future assessment and sharing of clinical
                      data and joint clinical studies. Two datasets were defined:
                      a mandatory dataset (minimal dataset) that can ideally be
                      obtained during a routine clinical consultation and a more
                      demanding extended dataset that is useful for research
                      purposes. In the future, the currently most widely used
                      clinician-reported outcome measure (ClinRO) in ataxia, the
                      scale for the assessment and rating of ataxia (SARA), should
                      be developed into a generally accepted instrument that can
                      be used in upcoming clinical trials. Furthermore, there is
                      an urgent need (i) to obtain more data on ataxia-specific,
                      patient-reported outcome measures (PROs), (ii) to
                      demonstrate and optimize sensitivity to change of many COAs,
                      and (iii) to establish methods and evidence of anchoring
                      change in COAs in patient meaningfulness, e.g., by
                      determining patient-derived minimally meaningful thresholds
                      of change.},
      subtyp        = {Review Article},
      keywords     = {Humans / Ataxia: therapy / Ataxia: diagnosis / Outcome
                      Assessment, Health Care: standards / Outcome Assessment,
                      Health Care: methods / Registries: standards / Consensus /
                      Clinical Trials as Topic: standards / Clinical Trials as
                      Topic: methods / Activities of daily living (ADL) (Other) /
                      Ataxia (Other) / Clinical outcome assessment (COA) (Other) /
                      Scale for the assessment and rating of ataxia (SARA) (Other)
                      / Standardization (Other)},
      cin          = {AG Gasser / AG Klockgether},
      ddc          = {610},
      cid          = {I:(DE-2719)1210000 / I:(DE-2719)1011001},
      pnm          = {353 - Clinical and Health Care Research (POF4-353)},
      pid          = {G:(DE-HGF)POF4-353},
      typ          = {PUB:(DE-HGF)16},
      pmc          = {pmc:PMC11102398},
      pubmed       = {pmid:37020147},
      doi          = {10.1007/s12311-023-01547-z},
      url          = {https://pub.dzne.de/record/258692},
}