MétaCan
Menu
Back to cohort
Record W4362604309 · doi:10.1007/s12311-023-01547-z

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

2023· review· en· W4362604309 on OpenAlexaff
Thomas Klockgether, Matthis Synofzik, Saud Alhusaini, Mathieu Anheim, Irina Antonijevic, Tee Ashizawa, Luís Bataller, Mélanie Berard, Enrico Bertini, Sylvia Boesch, Pedro Braga‐Neto, Emanuel Cassou, Edwin H.W. Chan, Rosalind Chuang, Abbie Collins, Joana Damásio, Karina Carvalho Donis, Antoine Duquette, João Durães, Alexandra Dürr, Rebecca Evans, Jennifer Faber, Jennifer Farmer, Vincenzo A. Gennarino, Holm Graeßner, Marcus Grobe‐Einsler, Hasmet Hanagasie, Morteza Heidari, Henry Houlden, Elisabetta Indelicato, Kinya Ishikawa, Heike Jacobi, Laura Bannach Jardim, Yaz Y. Kisanuki, Svetlana Kopishinskaia, Gilbert L ́Italien, Roderick P.P.W.M. Maas, Michelangelo Mancuso, Caterina Mariotti, Norlinah Mohamed Ibrahim, Wolfgang Nachbauer, Andrea H. Németh, Yi Shiau Ng, Katja Obieglo, Osamu Onodera, Puneet Opal, Luís Pereira de Almeida, Susan Perlman, Guido Primiano, M. Renaud, Liana S. Rosenthal, Francesco Saccà, Zahid Sattar, Tanja Schmitz‐Hübsch, Lüdger Schöls, Rebecca Schüle, Lauren Seeberger, Gabriella Silvestri, Anna Sobańska, Bin‐Weng Soong, Achal Kumar Srivastava, Colleen Stoyas, Sophie Tézenas du Montcel, Andreas Thieme, Dagmar Timmann, Adina Tocoian, Andreas Traschütz, Bart van de Warrenburg, Wolfram Ziegler

Bibliographic record

VenueThe Cerebellum · 2023
Typereview
Languageen
FieldNeuroscience
TopicGenetic Neurodegenerative Diseases
Canadian institutionsCentre Hospitalier de l’Université de Montréal
FundersRheinische Friedrich-Wilhelms-Universität BonnNational Institute of Neurological Disorders and StrokeAcademy of Medical SciencesWellcome Trust
KeywordsClinical trialObservational studyStandardizationAtaxiaOutcome (game theory)MedicineRating scalePsychologyMedical physicsComputer sciencePsychiatryPathology

Abstract

fetched live from OpenAlex

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.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.001
metaresearch head score (Gemma)0.003
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMeta-epidemiology (narrow)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Not applicable · Consensus signal: none
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.965
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0010.003
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0010.000
Bibliometrics0.0000.001
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0010.001
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.510
GPT teacher head0.508
Teacher spread0.001 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

Study designNot applicable
Domainnot available
GenreReview

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations20
Published2023
Admission routes1
Has abstractyes

Explore more

Same venueThe CerebellumSame topicGenetic Neurodegenerative DiseasesFrench-language works237,207