CADA-PRO, a patient questionnaire measuring key cognitive, motor, emotional and behavioral Outcomes in CADASIL

Di Folco, Cécile; Jabouley, Aude; Reyes, Sonia; Machado, Carla; Guey, Stéphanie; Hervé, Dominique; Fernandes, Fanny; Agossa, Joseph; Chabriat, Hugues; du Montcel, Sophie Tezenas · 2024 · Crossref

DOI: 10.1101/2024.05.30.24306664

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Summary

This study addresses the lack of patient-reported outcome measures (PROMs) specifically designed for Cerebral Small Vessel Disease (cSVD), particularly Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL). While cSVD significantly impacts cognitive, motor, emotional, and behavioral aspects of daily life, existing instruments were either focused on acute stroke deficits or lacked relevance to the chronic, progressive nature of cSVD. The authors aimed to develop and validate CADA-PRO, a questionnaire capturing these multidimensional consequences from the patient’s perspective to support future clinical trials. The research employed a two-stage design following COSMIN guidelines. In the development phase, 79 items were generated through consensus among patients, family representatives, and experts, then tested in 44 cSVD patients to assess feasibility and dimensionality. In the validation phase, a reduced item set was administered to 89 patients (43 with CADASIL, 46 with other cSVDs). Psychometric properties, including internal consistency, test-retest reliability, concurrent validity, and sensitivity to change, were evaluated. Concurrent validity was assessed by correlating CADA-PRO scores with clinical measures such as the modified Rankin scale, Hospital Anxiety and Depression scale (HAD), Starkstein Apathy Scale (SAS), and various neuropsychological tests. The final CADA-PRO scale comprises 18 items across four domains: daily activities, anxiety/depression, attention/executive functions, and motor difficulties. The instrument demonstrated high feasibility with low missing data and no significant floor or ceiling effects. It exhibited excellent internal consistency (Cronbach’s alpha = 0.95) and good test-retest reliability (ICC = 0.88). Concurrent validity results differed by diagnosis: in CADASIL patients, CADA-PRO scores correlated significantly with disability (Rankin scale), apathy (SAS), mood (HAD), and cognitive performance (Trail Making Test). In contrast, for patients with other cSVDs, scores correlated only with HAD and SAS, showing weak associations with cognitive or motor clinical markers. Sensitivity to change analysis over one year showed no significant score changes, consistent with the slow progression of the disease. The study concludes that CADA-PRO is a valid and reliable instrument for measuring patient-reported outcomes in CADASIL, effectively capturing the disease’s impact on daily life, mood, and cognition. However, its applicability to other forms of cSVD requires further refinement due to weaker correlations with clinical markers in those groups. This tool provides a standardized method for assessing clinically relevant outcomes in future therapeutic trials for CADASIL.

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• dbq psychometrics