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Consensus Recommendations for Clinical Outcome Assessments and Registry Development in Ataxias: Ataxia Global Initiative (AGI) Working Group Expert Guidance

Authors: Klockgether, Thomas; Synofzik, Matthis; AGI working group on COAs and Registries; Alhusaini, Saud; Anheim, Mathieu; Antonijevic, Irina; Ashizawa, Tee; +63 Authors

Consensus Recommendations for Clinical Outcome Assessments and Registry Development in Ataxias: Ataxia Global Initiative (AGI) Working Group Expert Guidance

Abstract

AbstractTo 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.

Countries
Italy, Italy, Germany, Italy, Portugal, United Kingdom, Italy, United States
Keywords

570, Consensus, Outcome Assessment, 610, Review, Clinical outcome assessment (COA) ; Activities of daily living (ADL) ; Ataxia ; Ataxia/diagnosis [MeSH] ; Registries/standards [MeSH] ; Humans [MeSH] ; Scale for the assessment and rating of ataxia (SARA) ; Outcome Assessment, Health Care/standards [MeSH] ; Clinical Trials as Topic/standards [MeSH] ; Ataxia/therapy [MeSH] ; Outcome Assessment, Health Care/methods [MeSH] ; Clinical Trials as Topic/methods [MeSH] ; Review ; Standardization ; Consensus [MeSH], methods [Clinical Trials as Topic], Outcome Assessment, Health Care, Humans, Registries, Clinical Trials as Topic, diagnosis [Ataxia], therapy [Ataxia], Standardization, Health Care, methods [Outcome Assessment, Health Care], Clinical outcome assessment (COA), standards [Clinical Trials as Topic], Scale for the assessment and rating of ataxia (SARA), Activities of daily living (ADL); Ataxia; Clinical outcome assessment (COA); Scale for the assessment and rating of ataxia (SARA); Standardization, Ataxia, standards [Outcome Assessment, Health Care], standards [Registries], Activities of daily living (ADL), ddc: ddc:610

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    popularity
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    Top 10%
    influence
    This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
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    This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
17
Top 10%
Average
Top 10%
Green
hybrid