Publication Date
11-1-2023
Journal
Epilepsy Research
DOI
10.1016/j.eplepsyres.2023.107231
PMID
37751639
PMCID
PMC10760432
PubMedCentral® Posted Date
1-2-2024
PubMedCentral® Full Text Version
Author MSS
Published Open-Access
yes
Keywords
Child, Humans, Infant, Child, Preschool, Adolescent, Young Adult, Adult, Caregivers, Spasms, Infantile, Epileptic Syndromes, Surveys and Questionnaires, Protein Serine-Threonine Kinases, CDKL5 deficiency disorder, caregiver-report, validation, outcome measure, severity, developmental attainment
Abstract
BACKGROUND: CDKL5 Deficiency Disorder (CDD) is a severe X-linked developmental and epileptic encephalopathy. Existing developmental outcome measures have floor effects and cannot capture incremental changes in symptoms. We modified the caregiver portion of a CDD clinical severity assessment (CCSA) and assessed content and response-process validity.
METHODS: We conducted cognitive interviews with 15 parent caregivers of 1-39-year-old children with CDD. Caregivers discussed their understanding and concerns regarding appropriateness of both questions and answer options. Item wording and questionnaire structure were adjusted iteratively to ensure questions were understood as intended.
RESULTS: The CCSA was refined during three rounds of cognitive interviews into two measures: (1) the CDD Developmental Questionnaire - Caregiver (CDQ-Caregiver) focused on developmental skills, and (2) the CDD Clinical Severity Assessment - Caregiver (CCSA-Caregiver) focused on symptom severity. Branching logic was used to ensure questions were age and skill appropriate. Initial pilot data (n = 11) suggested no floor effects.
CONCLUSIONS: This study modified the caregiver portion of the initial CCSA and provided evidence for its content and response process validity.