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

Klockgether, Thomas; du Montcel, Sophie Tezenas; Bertini, Enrico; Tocoian, Adina; Hanagasie, Hasmet; Schüle-Freyer, Rebecca; Ng, Yi Shiau; Cassou, Emanuel; Rosenthal, Liana; Evans, Rebecca; Berard, Mélanie; Opal, Puneet; Stoyas, Colleen; Houlden, Henry; Chan, Edwin; Obieglo, Katja; Gennarino, Vincenzo; Registries; Grobe-Einsler, Marcus; Farmer, Jennifer; van de Warrenburg, Bart; Bataller, Luis; Maas, Roderick; Antonijevic, Irina; Schmitz-Hübsch, Tanja; Sattar, Zahid; Donis, Karina; Synofzik, Matthis; Nachbauer, Wolfgang; Chuang, Rosalind; Primiano, Guido; de Almeida, Luis Pereira; Ishikawa, Kinya; Traschuetz, Andreas; Kisanuki, Yaz; Timmann, Dagmar; Heidari, Morteza; Kopishinskaia, Svetlana; Onodera, Osamu; Srivastava, Achal Kumar; L Italien, Gilbert; Durr, Alexandra; Saccà, Francesco; Perlman, Susan; Ashizawa, Tee; Boesch, Sylvia; Durães, João; Duquette, Antoine; Anheim, Mathieu; Schöls, Ludger; Thieme, Andreas; Ziegler, Wolfram; Jacobi, Heike; Collins, Abbie; Damásio, Joana; Jardim, Laura; Indelicato, Elisabetta; Braga-Neto, Pedro; Mariotti, Caterina; Faber, Jennifer; Nemeth, Andrea; Graessner, Holm; COAs, AGI working group on; Mancuso, Michelangelo; Renaud, Mathilde; Soong, Bin-Weng; Ibrahim, Norlinah Mohamed; Silvestri, Gabriella; Sobanska, Anna; Alhusaini, Saud; Seeberger, Lauren

doi:10.1007/s12311-023-01547-z

Items
Marc 21

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024	7	_	\|a pmid:37020147 \|2 pmid
024	7	_	\|a 1473-4222 \|2 ISSN
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037	_	_	\|a DZNE-2023-00665
041	_	_	\|a English
082	_	_	\|a 610
100	1	_	\|a Klockgether, Thomas \|0 P:(DE-2719)2810314 \|b 0 \|e First author
245	_	_	\|a Consensus Recommendations for Clinical Outcome Assessments and Registry Development in Ataxias: Ataxia Global Initiative (AGI) Working Group Expert Guidance.
260	_	_	\|a London \|c 2024 \|b Dunitz
336	7	_	\|a article \|2 DRIVER
336	7	_	\|a Output Types/Journal article \|2 DataCite
336	7	_	\|a Journal Article \|b journal \|m journal \|0 PUB:(DE-HGF)16 \|s 1716276944_21512 \|2 PUB:(DE-HGF) \|x Review Article
336	7	_	\|a ARTICLE \|2 BibTeX
336	7	_	\|a JOURNAL_ARTICLE \|2 ORCID
336	7	_	\|a Journal Article \|0 0 \|2 EndNote
520	_	_	\|a 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.
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650	_	7	\|a Activities of daily living (ADL) \|2 Other
650	_	7	\|a Ataxia \|2 Other
650	_	7	\|a Clinical outcome assessment (COA) \|2 Other
650	_	7	\|a Scale for the assessment and rating of ataxia (SARA) \|2 Other
650	_	7	\|a Standardization \|2 Other
650	_	2	\|a Humans \|2 MeSH
650	_	2	\|a Ataxia: therapy \|2 MeSH
650	_	2	\|a Ataxia: diagnosis \|2 MeSH
650	_	2	\|a Outcome Assessment, Health Care: standards \|2 MeSH
650	_	2	\|a Outcome Assessment, Health Care: methods \|2 MeSH
650	_	2	\|a Registries: standards \|2 MeSH
650	_	2	\|a Consensus \|2 MeSH
650	_	2	\|a Clinical Trials as Topic: standards \|2 MeSH
650	_	2	\|a Clinical Trials as Topic: methods \|2 MeSH
700	1	_	\|a Synofzik, Matthis \|0 P:(DE-2719)2811275 \|b 1 \|e Last author
700	1	_	\|a COAs, AGI working group on \|b 2
700	1	_	\|a Registries \|b 3 \|e Collaboration Author
700	1	_	\|a Alhusaini, Saud \|b 4 \|e Contributor
700	1	_	\|a Anheim, Mathieu \|b 5 \|e Contributor
700	1	_	\|a Antonijevic, Irina \|b 6 \|e Contributor
700	1	_	\|a Ashizawa, Tee \|b 7 \|e Contributor
700	1	_	\|a Bataller, Luis \|b 8 \|e Contributor
700	1	_	\|a Berard, Mélanie \|b 9 \|e Contributor
700	1	_	\|a Bertini, Enrico \|b 10 \|e Contributor
700	1	_	\|a Boesch, Sylvia \|b 11 \|e Contributor
700	1	_	\|a Braga-Neto, Pedro \|b 12 \|e Contributor
700	1	_	\|a Cassou, Emanuel \|b 13 \|e Contributor
700	1	_	\|a Chan, Edwin \|b 14 \|e Contributor
700	1	_	\|a Chuang, Rosalind \|b 15 \|e Contributor
700	1	_	\|a Collins, Abbie \|b 16 \|e Contributor
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700	1	_	\|a Durr, Alexandra \|0 P:(DE-HGF)0 \|b 21 \|e Contributor
700	1	_	\|a Evans, Rebecca \|b 22 \|e Contributor
700	1	_	\|a Faber, Jennifer \|0 P:(DE-2719)2811327 \|b 23 \|e Contributor \|u dzne
700	1	_	\|a Farmer, Jennifer \|b 24 \|e Contributor
700	1	_	\|a Gennarino, Vincenzo \|b 25 \|e Contributor
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700	1	_	\|a Jacobi, Heike \|0 P:(DE-HGF)0 \|b 33 \|e Contributor
700	1	_	\|a Jardim, Laura \|b 34 \|e Contributor
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700	1	_	\|a Kopishinskaia, Svetlana \|b 36 \|e Contributor
700	1	_	\|a L Italien, Gilbert \|b 37 \|e Contributor
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700	1	_	\|a Perlman, Susan \|b 49 \|e Contributor
700	1	_	\|a Primiano, Guido \|b 50 \|e Contributor
700	1	_	\|a Renaud, Mathilde \|b 51 \|e Contributor
700	1	_	\|a Rosenthal, Liana \|b 52 \|e Contributor
700	1	_	\|a Saccà, Francesco \|b 53 \|e Contributor
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700	1	_	\|a Silvestri, Gabriella \|b 59 \|e Contributor
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Library	Collection	CLSMajor	CLSMinor	Language	Author

Marc 21

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