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CADA-PRO: A Patient Questionnaire Measuring Key Cognitive, Motor, Emotional, and Behavioral Outcomes in CADASIL
Cécile Di Folco1, Aude Jabouley2, Sonia Reyes2
1ARAMIS, Sorbonne Université, Paris Brain Institute (ICM Institut du Cerveau), Institut national de recherche en sciences et technologies du numérique (INRIA), Institut national de la santé et de la recherche médicale (INSERM), Assistance Publique - Hôpitaux de Paris, Groupe Hospitalier Sorbonne Université, Paris, France (C.D.F., J.A., S.T.d.M.).
Insights
A new patient-reported outcome scale was developed to measure the impact of cerebral small vessel disease (cSVD), including CADASIL, on daily life. This validated tool aids in assessing patient experiences in clinical trials.
Area of Science:
- Neurology
- Clinical Psychology
- Medical Informatics
Background:
- Cerebral small vessel disease (cSVD), including CADASIL, significantly impacts patients' cognitive, motor, emotional, and behavioral functions.
- Currently, no patient-reported outcome (PRO) instrument exists to capture these diverse impacts of cSVD.
- This study addresses the need for a validated PRO tool for cSVD patients.
Purpose of the Study:
- To develop and validate a patient-reported questionnaire for assessing the impact of cSVD on daily life.
- To create a reliable and sensitive instrument for use in clinical research and patient care.
Main Methods:
- A 79-item scale was generated through expert and patient consensus.
- Feasibility, internal consistency, and dimensionality were assessed in an initial patient sample.
- A reduced 18-item scale was validated in a larger cohort for reliability, validity, and sensitivity to change.
Main Results:
- The final 18-item CADASIL Patient-Reported Outcome scale effectively covers depression/anxiety, attention/executive functions, motor, and daily activities.
- The scale demonstrated low missing data, no significant floor/ceiling effects, and excellent internal consistency (Cronbach alpha=0.95) and test-retest reliability (ICC=0.88).
- Scores correlated well with established clinical measures in CADASIL patients, showing promise for assessing treatment effects.
Conclusions:
- The CADASIL Patient-Reported Outcome scale is a promising, innovative instrument for measuring PROs in cSVD clinical trials.
- The scale is fully validated for patients with CADASIL, offering a patient-centered outcome measure.
- Further refinement is recommended for its application in broader cSVD populations.
Background:
Cerebral small vessel disease (cSVD) of ischemic type, either sporadic or genetic, as cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL), can impact the quality of daily life on various cognitive, motor, emotional, or behavioral aspects. No instrument has been developed to measure these outcomes from the patient's perspective. We thus aimed to develop and validate a patient-reported questionnaire.
Methods:
In a development study, 79 items were generated by consensus between patients, family representatives, and cSVD experts. A first sample of patients allowed assessing the feasibility (missing data, floor and ceiling effect, and acceptability), internal consistency, and dimensionality of a first set of items. Thereafter, in a validation study, we tested a reduced version of the item set in a larger sample to assess the feasibility, internal consistency, dimensionality, test-retest reliability, concurrent validity, and sensitivity to change.
Results:
The scale was developed in 44 patients with cSVD and validated in a second sample of 89 individuals (including 43 patients with CADASIL and 46 with another cSVD). The final CADASIL Patient-Reported Outcome scale comprised 18 items covering 4 categories of consequences (depression/anxiety, attention/executive functions, motor, and daily activities) of the disease. The proportion of missing data was low, and no item displayed a major floor or ceiling effect. Both the internal consistency and test-retest reliability were good (Cronbach alpha=0.95, intraclass correlation coefficient=0.88). In patients with CADASIL, CADASIL Patient-Reported Outcome scores correlated with the modified Rankin Scale, Starkstein Apathy Scale, Hospital Anxiety and Depression scale, Working Memory Index, and trail making test times. In patients with other cSVDs, CADASIL Patient-Reported Outcome correlated only with Hospital Anxiety and Depression scale and Starkstein Apathy Scale.
Conclusions:
The CADASIL Patient-Reported Outcome may be an innovative instrument for measuring patient-reported outcomes in future cSVD trials. Full validation was obtained for its use in patients with CADASIL, but further improvement is needed for its application in other cSVDs.
