Journal Article
Consensus Recommendations for Clinical Outcome Assessments and Registry Development in Ataxias: Ataxia Global Initiative (AGI) Working Group Expert Guidance
Thomas Klockgether; Matthis Synofzik; Saud Alhusaini; Mathieu Anheim; Irina Antonijevic; Tee Ashizawa; Luis Bataller; Mélanie Berard; Enrico Bertini; Sylvia Boesch; Pedro Braga-Neto; Emanuel Cassou; Edwin Chan; Rosalind Chuang; Abbie Collins; Joana Damásio; Karina Donis; Antoine Duquette; João Durães; Alexandra Durr; Rebecca Evans; Jennifer Faber; Jennifer Farmer; Vincenzo Gennarino; Holm Graessner; Marcus Grobe-Einsler; Hasmet Hanagasie; Morteza Heidari; Henry Houlden; Elisabetta Indelicato; Kinya Ishikawa; Heike Jacobi; Laura Jardim; Yaz Kisanuki; Svetlana Kopishinskaia; Gilbert L´Italien; Roderick Maas; Michelangelo Mancuso; Caterina Mariotti; Norlinah Mohamed Ibrahim; Wolfgang Nachbauer; Andrea Nemeth; Yi Shiau Ng; Katja Obieglo; Osamu Onodera; Puneet Opal; Luis Pereira de Almeida; Susan Perlman; Guido Primiano; Mathilde Renaud; Liana Rosenthal; Francesco Saccà; Zahid Sattar; Tanja Schmitz-Hübsch; Ludger Schöls; Rebecca Schüle; Lauren Seeberger; Gabriella Silvestri; Anna Sobanska; Bin-Weng Soong; Achal Kumar Srivastava; Colleen Stoyas; Sophie Tezenas du Montcel; Andreas Thieme; Dagmar Timmann; Adina Tocoian; Andreas Traschütz; Bart van de Warrenburg; Wolfram Ziegler
The Cerebellum · Vol. 23, Issue 3 · pp. 924-930 · 2023
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.