Journal Article (Review Article)

DZNE-2023-00665

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Consensus Recommendations for Clinical Outcome Assessments and Registry Development in Ataxias: Ataxia Global Initiative (AGI) Working Group Expert Guidance.

Klockgether, T. (First author)DZNE* ; Synofzik, M. (Last author)DZNE* ; COAs, A. w. g. o. ; Registries (Collaboration Author) ; Alhusaini, S. (Contributor) ; Anheim, M. (Contributor) ; Antonijevic, I. (Contributor) ; Ashizawa, T. (Contributor) ; Bataller, L. (Contributor) ; Berard, M. (Contributor) ; Bertini, E. (Contributor) ; Boesch, S. (Contributor) ; Braga-Neto, P. (Contributor) ; Cassou, E. (Contributor) ; Chan, E. (Contributor) ; Chuang, R. (Contributor) ; Collins, A. (Contributor) ; Damásio, J. (Contributor) ; Donis, K. (Contributor) ; Duquette, A. (Contributor) ; Durães, J. (Contributor) ; Durr, A. (Contributor) ; Evans, R. (Contributor) ; Faber, J. (Contributor)Extern* ; Farmer, J. (Contributor) ; Gennarino, V. (Contributor) ; Graessner, H. (Contributor) ; Grobe-Einsler, M. (Contributor)Extern* ; Hanagasie, H. (Contributor) ; Heidari, M. (Contributor) ; Houlden, H. (Contributor) ; Indelicato, E. (Contributor) ; Ishikawa, K. (Contributor) ; Jacobi, H. (Contributor) ; Jardim, L. (Contributor) ; Kisanuki, Y. (Contributor) ; Kopishinskaia, S. (Contributor) ; L Italien, G. (Contributor) ; Maas, R. (Contributor) ; Mancuso, M. (Contributor) ; Mariotti, C. (Contributor) ; Ibrahim, N. M. (Contributor) ; Nachbauer, W. (Contributor) ; Nemeth, A. (Contributor) ; Ng, Y. S. (Contributor) ; Obieglo, K. (Contributor) ; Onodera, O. (Contributor) ; Opal, P. (Contributor) ; de Almeida, L. P. (Contributor) ; Perlman, S. (Contributor) ; Primiano, G. (Contributor) ; Renaud, M. (Contributor) ; Rosenthal, L. (Contributor) ; Saccà, F. (Contributor) ; Sattar, Z. (Contributor) ; Schmitz-Hübsch, T. (Contributor) ; Schöls, L. (Contributor)Extern* ; Schüle-Freyer, R. (Contributor)Extern* ; Seeberger, L. (Contributor) ; Silvestri, G. (Contributor) ; Sobanska, A. (Contributor) ; Soong, B.-W. (Contributor) ; Srivastava, A. K. (Contributor) ; Stoyas, C. (Contributor) ; du Montcel, S. T. (Contributor) ; Thieme, A. (Contributor) ; Timmann, D. (Contributor) ; Tocoian, A. (Contributor) ; Traschuetz, A. (Contributor)Extern* ; van de Warrenburg, B. (Contributor) ; Ziegler, W. (Contributor)

2024
Dunitz London

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Please use a persistent id in citations: doi:10.1007/s12311-023-01547-z

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.

Keyword(s): Humans (MeSH) ; Ataxia: therapy (MeSH) ; Ataxia: diagnosis (MeSH) ; Outcome Assessment, Health Care: standards (MeSH) ; Outcome Assessment, Health Care: methods (MeSH) ; Registries: standards (MeSH) ; Consensus (MeSH) ; Clinical Trials as Topic: standards (MeSH) ; Clinical Trials as Topic: methods (MeSH) ; Activities of daily living (ADL) ; Ataxia ; Clinical outcome assessment (COA) ; Scale for the assessment and rating of ataxia (SARA) ; Standardization

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Contributing Institute(s):

1. Parkinson Genetics (AG Gasser)
2. Patient Studies (AG Klockgether)

Research Program(s):

1. 353 - Clinical and Health Care Research (POF4-353) (POF4-353)

Appears in the scientific report 2023

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Database coverage:
; ; ; Clarivate Analytics Master Journal List ; DEAL Springer ; Ebsco Academic Search ; Essential Science Indicators ; IF < 5 ; JCR ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

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