TY  - JOUR
AU  - Klockgether, Thomas
AU  - Synofzik, Matthis
AU  - COAs, AGI working group on
TI  - Consensus Recommendations for Clinical Outcome Assessments and Registry Development in Ataxias: Ataxia Global Initiative (AGI) Working Group Expert Guidance.
JO  - The Cerebellum
VL  - 23
IS  - 3
SN  - 1473-4222
CY  - London
PB  - Dunitz
M1  - DZNE-2023-00665
SP  - 924 - 930
PY  - 2024
AB  - 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.
KW  - Humans
KW  - Ataxia: therapy
KW  - Ataxia: diagnosis
KW  - Outcome Assessment, Health Care: standards
KW  - Outcome Assessment, Health Care: methods
KW  - Registries: standards
KW  - Consensus
KW  - Clinical Trials as Topic: standards
KW  - Clinical Trials as Topic: methods
KW  - Activities of daily living (ADL) (Other)
KW  - Ataxia (Other)
KW  - Clinical outcome assessment (COA) (Other)
KW  - Scale for the assessment and rating of ataxia (SARA) (Other)
KW  - Standardization (Other)
LB  - PUB:(DE-HGF)16
C2  - pmc:PMC11102398
C6  - pmid:37020147
DO  - DOI:10.1007/s12311-023-01547-z
UR  - https://pub.dzne.de/record/258692
ER  -