International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA
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Le résumé fourni par la source
INTRODUCTION: The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials. METHODS: Clinical outcome assessments for the top 10 priority domains identified in the Voice of the Patient Report for Rett syndrome were compiled and available psychometric data were extracted. The clinical outcome assessments measured clinical severity, functional abilities, comorbidities and quality of life, and electrophysiological biomarkers. An international and multidisciplinary panel of 29 experts with clinical, research, psychometric, biostatistical, industry and lived experience was identified through International Rett Syndrome Foundation networks, to discuss validation of the clinical outcome assessments, gaps and next steps, during a workshop and in a follow-up questionnaire. The identified gaps and limitations were coded using inductive content analysis. RESULTS: Variable validation profiles across 26 clinical outcome assessments of clinical severity, functional abilities, and comorbidities were discussed. Reliability, validity, and responsiveness profiles were mostly incomplete; there were limited content validation data, particularly parent-informed relevance, comprehensiveness and comprehensibility of items; and no data on meaningful change or cross-cultural validity. The panel identified needs for standardised administration protocols and systematic validation programmes. CONCLUSION: A pipeline of collaborative clinical outcome assessment development and validation research in Rett syndrome can now be designed, aiming to have fit-for-purpose measures that can evaluate meaningful change, to serve future clinical trials and clinical practice.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA
- Date Crossref
- 21/12/2024
- Éditeur
- Springer Science and Business Media LLC
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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The Kids Research Institute Australia pays non établi dans la noticeOrganisation à but non lucratif
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The University of Western Australia The Kids Research Institute Australia pays non établi dans la noticeUniversité ou école supérieure
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Curtin University pays non établi dans la noticeUniversité ou école supérieure
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International Rett syndrome Foundation pays non établi dans la noticeOrganisation à but non lucratif
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Rome Foundation pays non établi dans la noticeOrganisation à but non lucratif
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Boston Children's Hospital Department of Neurology pays non établi dans la noticeÉtablissement de santé
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Emory University Department of Human Genetics pays non établi dans la noticeUniversité ou école supérieure
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Duke University Duke Center for Autism and Brain Development pays non établi dans la noticeUniversité ou école supérieure
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Center for Autism and Related Disorders pays non établi dans la noticeInstitution
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RTI International pays non établi dans la noticeOrganisation à but non lucratif
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University of Reading pays non établi dans la noticeUniversité ou école supérieure
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Children's Hospital of Philadelphia pays non établi dans la noticeOrganisme public
The Kids Research Institute Australia, The Kids Research Institute Australia — The University of Western Australia et Curtin University, avec 9 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.