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: 10.1161/strokeaha.124.047692

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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 large-stroke deficits or lacked relevance to the specific complaints of cSVD patients. The authors aimed to develop and validate CADA-PRO, a questionnaire capturing the multidimensional consequences of the disease from the patient’s perspective, to support future clinical trials and regulatory assessments. The research employed a two-stage design involving a development study and a validation study. In the development phase, 79 items were generated through consensus among patients, family representatives, psychologists, and clinicians. These items were tested on 44 patients with various forms of cSVD to assess feasibility, internal consistency, and dimensionality. Based on these results, a reduced set of items was selected for the validation study, which included 89 participants (43 with CADASIL and 46 with other cSVDs). The validation process assessed feasibility, test-retest reliability, concurrent validity, and sensitivity to change over one year. Psychometric properties were evaluated using Exploratory Factor Analysis, Confirmatory Multidimensional Item Response Theory, Cronbach’s alpha, and Intraclass Correlation Coefficients. Concurrent validity was established by correlating CADA-PRO scores with clinical assessments, including the Hospital Anxiety and Depression scale (HAD), Starkstein Apathy Scale (SAS), modified Rankin scale, and various neuropsychological tests. The final CADA-PRO scale comprises 18 items organized into four domains: daily activities, anxiety/depression, attention/executive functions, and motor difficulties. The instrument demonstrated excellent psychometric properties, with high internal consistency (Cronbach’s alpha = 0.95) and good test-retest reliability (ICC = 0.88). Feasibility was high, with low missing data rates and no significant floor or ceiling effects. Concurrent validity analysis revealed that CADA-PRO scores correlated significantly with clinical measures in CADASIL patients, including disability (Rankin score), mood (HAD, SAS), and cognitive performance (Working Memory Index, Trail Making Test). In contrast, for patients with other cSVDs, correlations were limited primarily to mood scales (HAD and SAS). Sensitivity to change analysis showed no significant difference in scores over one year, consistent with the slow progression of the disease in this cohort. The study concludes that CADA-PRO is a robust, validated instrument for measuring patient-perceived outcomes in CADASIL, capturing emotional, behavioral, and functional impacts that are distinct from pure cognitive impairment. The findings highlight that mood disturbances and apathy are critical drivers of quality of life in these patients. While fully validated for CADASIL, the tool requires further refinement for broader application to other cSVD types. The authors recommend CADA-PRO as a suitable secondary endpoint for future clinical trials targeting disease-modifying therapies in CADASIL, emphasizing the importance of incorporating patient-reported outcomes to ensure treatments address clinically relevant issues.

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• dbq psychometrics