Dev Med Child Neurol. 2025 Sep 19. doi: 10.1111/dmcn.16506. Online ahead of print.
ABSTRACT
AIM: To assess the psychometric characteristics of the Infantile Neuroaxonal Dystrophy Rating Scale (INAD-RS).
METHOD: We retrospectively analysed interim data from the INAD Natural History Study using clinicians’ ratings (n = 39) of patients with infantile neuroaxonal dystrophy (INAD). Data were analysed to explore the internal consistency, test-retest reliability, known group validity, and longitudinal changes of the INAD-RS.
RESULTS: The analysis identified good internal consistency, convergent validity, and test-retest reliability across scale domains assessing gross motor, fine motor, bulbar, ocular, and temporo-frontal functions, whereas the domain of autonomic nervous system function contributed weakly to the overall INAD-RS score. Furthermore, preliminary evidence suggested that INAD-RS total scores discriminated among clinical phenotypes and was sensitive to change over time.
INTERPRETATION: The psychometric properties of the INAD-RS showed construct validity and reliability for five out of six domains (autonomic nervous system excluded). The scale is a promising instrument for evaluating children with INAD in clinical research and clinical practice. Although promising, further evidence is needed to refine the scale and support its suitability for assessing disease progression in clinical trials and clinical practice.
PMID:40974106 | DOI:10.1111/dmcn.16506
