Friedreich’s ataxia Common Data Element Review: Updates to the National Institute of Neurological Disorders and Stroke Friedreich’s ataxia (FA) Common Data Element Recommendations: 2018 FA Working Group Review

Publication Type
Year of Publication
Edun, Ilar; Gay, Katelyn; Sheikh, Muniza; Esterlitz, Joy; Lynch, David; Pandolfo, Massimo; Gubitz, Amelie; Lungu, Codrin; Mendoza-Puccini, M. Carolina; NINDS Friedreich's ataxia CDE Oversight Committee and Working Groups
Updates to the National Institute of Neurological Disorders and Stroke (NINDS) Friedreich’s Ataxia (FA) Common Data Element (CDE) Recommendations
Objective: Through the development of Friedreich’s Ataxia (FA)-specific common data elements (CDEs), the National Institute of Neurological Disorders and Stroke (NINDS) CDE project strives to improve data collection by increasing efficiency to reduce study start-up time, improving data quality, and facilitating data sharing/meta-analyses. The CDEs are dynamic and are updated as needed with new scientific findings. Background: The FA CDEs were developed in 2011 by an international group of subject matter experts. Recommendations were stratified by classifications: Core (required), Supplemental-Highly Recommended (strongly encouraged based on study type), Supplemental (commonly collected, but not required), and Exploratory (reasonable to fill in gaps, but require further validation). An FA Oversight Committee (OC) was subsequently formed to review user feedback and updates to the CDEs based on research advancements. The OC convened in January 2018 and determined that a comprehensive review was needed to update the FA CDEs. Design/Methods: Domain-specific subgroups were convened to review the current FA CDEs: Ataxia and Performance Measures; Biomarkers; Cardiac and Clinical Outcomes; and Imaging. Subgroup members focused on ensuring the accuracy of current classifications, providing any new elements/measures to be included, and determining a context for use for Supplemental – Highly Recommended CDEs. Results: Updates to the FA CDEs will include classification revisions and a new Brain MRI case report form. The FA OC and subgroups will review the draft recommendations prior to having a public review on the NINDS CDE website. Updates to the FA CDEs will be posted once comments and reviews have been incorporated in 2019. Conclusions: NINDS encourages the use of CDEs for all clinical research in neuroscience. To ensure that the FA CDEs are a dynamic and useful resource, the recommendations are updated periodically based on the current state of science and user input.