Content Validation of Clinician-Reported Items for a Severity Measure for CDKL5 Deficiency Disorder
Year: 2021
Type: article
Abstract: CDKL5 deficiency disorder (CDD) results in early-onset seizures and severe developmental impairments. A CDD clinical severity assessment (CCSA) was previously developed with clinician and parent-report items to capture information on a range of domains. Consistent with US Food and Drug Administration (FDA) guidelines, content validation is the first step in evaluating the psychometric properties of an outcome measure. The aim of this study was to validate the content of the clinician-reported items in the CCSA (CCSA-Clinician). Eight neurologists leading the USA CDD Center of Excellence clinics were interviewed using the "think aloud" technique to critique 26 clinician-reported items. Common themes were aggregated, and a literature search of related assessments informed item modifications. The clinicians then participated in 2 consensus meetings to review themes and finalize the items. A consensus was achieved for the content of the CCSA-Clinician. Eight of the original items were omitted, 11 items were added, and the remaining 18 items were revised. The final 29 items were classified into 2 domains: functioning and neurologic impairments. This study enabled refinement of the CCSA-Clinician and provided evidence for its content validity. This preliminary validation is essential before field testing and further validation, in order to advance the instrument toward clinical trial readiness.
Source: Journal of Child Neurology
Institutions: The Kids Research Institute Australia, St. Louis Children's Hospital, Washington University in St. Louis, Cleveland Clinic, Children's Hospital of Philadelphia, University of Pennsylvania, Baylor College of Medicine, Texas Children's Hospital, University of California, Los Angeles, Mattel Children's Hospital, Boston Children's Hospital, NYU Langone Health, New York University, The University of Western Australia, Children's Hospital Colorado, University of Colorado Anschutz, Curtin University
Language: English
Subfield: Education
Field: Social Sciences
Domain: Social Sciences
Open Access status: green
Funders: Children's Hospital Colorado, ACADIA Pharmaceuticals, International Foundation for CDKL5 Research, National Institutes of Health, National Health and Medical Research Council, National Institute of Neurological Disorders and Stroke, Eunice Kennedy Shriver National Institute of Child Health and Human Development
Awards: G1266746737, Child disability:understanding determinants and improving outcomes, Diagnosis and genotype-phenotype correlations in early life epilepsy and CDKL5 disorder, Multi-Site Validation of Biomarkers and Core Clinical Outcome Measures for Clinical Trials Readiness in CDKL5 Deficiency Disorder, Boston Children's Hospital/HMS Intellectual and Developmental Disabilities Research Center
OpenAlex ID: W3191112699