Documenting the psychometric properties of the scale for the assessment and rating of ataxia to advance trial readiness of Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay.

Background: The Scale for the Assessment and Rating of Ataxia (SARA) is a commonly used scale measuring the severity of cerebellar ataxia and is a candidate for outcome measurement in foreseeable clinical trials in Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS). Documenting its psychometric properties in this population will accelerate clinical trial readiness. The objectives of this study were to document the content and construct validity, the internal consistency, and to explore the 2-year responsiveness and the 4-year interpretability of the SARA in ARSACS.
Methods: The first phase of the study consisted of an international Delphi survey to document the content validity. The second phase consisted of a methodological study from the secondary analysis of a longitudinal study to document the construct validity in 69 participants. Responsiveness to change and interpretability of the SARA was explored among a sub-sample of participants (n = 32 and n = 16, respectively).
Results: The SARA demonstrates adequate content validity with possible influence of pyramidal and/or neuropathic involvement. It demonstrates excellent construct validity (r _s  = 0.77-0.95) and internal consistency (Cronbach's α = 0.89). The responsiveness to change was not significant, and the interpretation of change score increased by 1.9 ± 2.5 falling below the minimal detectable change threshold of 3.06.
Conclusions: The SARA has shown evidences of adequate content validity and excellent construct validity in ARSACS. Responsiveness to change and interpretability will need to be further documented among a larger sample over a longer period of time.
(Copyright © 2020. Published by Elsevier B.V.)