ArticleJournal of child neurology2021
Content Validation of Clinician-Reported Items for a Severity Measure for CDKL5 Deficiency Disorder.
Article in Journal of child neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05558371 (Multi-Site Validation of Biomarkers and Core Clinical Outcome Measures for Clinical Trials Readiness in CDKL5 Deficiency Disorder), which is not on this map. Cited by 19 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Multi-Site Validation of Biomarkers and Core Clinical Outcome Measures for Clinical Trials Readiness in CDKL5 Deficiency Disorder
Who cites it
19 citing papers in PubMed, 27 citations in OpenAlex.
- Exploring medical food supplementation as an adjunct strategy for microbiota restoration and seizure reduction in CDKL5 deficiency disorder.Epilepsia open · 2026Article
- Thinking-aloud methodology applied to health questionnaire development and validation: a scoping review.BMC medical research methodology · 2026Article
- Cortical Visual Impairment Across a Range of Neurodevelopmental Disorders: Clinical Characterization, Diagnostic Tool Evaluation, and Association with Developmental Outcomes.Journal of child neurology · 2026Article
- Development and initial validation of the Communication Inventory Disability - Observer Reported (CID-OR): a measure of communication in CDKL5 deficiency disorder.Journal of patient-reported outcomes · 2025Article
- Beyond Seizures as an Outcome Measure: A Global Severity Scoring System for CDKL5 Deficiency Disorder.Brain and behavior · 2025Observational
- Deletions in the CDKL5 5' untranslated region lead to CDKL5 deficiency disorder.American journal of medical genetics. Part A · 2025Article
- Adult Phenotype ofNeurology. Genetics · 2024Article
- The long odyssey for the DEE-CDKL5 diagnosis: A call for action.Epilepsia open · 2024Article
- Psychometric evaluation of clinician- and caregiver-reported clinical severity assessments for individuals with CDKL5 deficiency disorder.Epilepsia · 2024Article
- Bipolar Disorder in a female with CDKL5 Deficiency Disorder: A Case Report.Research square · 2024Article
- Gut microbiota profile in CDKL5 deficiency disorder patients.Scientific reports · 2024Article
- CDKL5 Deficiency Disorder: Some Lessons Learned 20 Years After the First Description.American journal on intellectual and developmental disabilities · 2024Article
- Adult Phenotype ofNeurology. Genetics · 2023Article
- Delineating clinical and developmental outcomes in STXBP1-related disorders.Brain : a journal of neurology · 2023Article
- The development, content and response process validation of a caregiver-reported severity measure for CDKL5 deficiency disorder.Epilepsy research · 2023Article
- Top caregiver concerns in Rett syndrome and related disorders: data from the US natural history study.Journal of neurodevelopmental disorders · 2023Article
- CDKL5 deficiency disorder: clinical features, diagnosis, and management.The Lancet. Neurology · 2022Review
- International Consensus Recommendations for the Assessment and Management of Individuals With CDKL5 Deficiency Disorder.Frontiers in neurology · 2022Article
- Natural History Studies and Clinical Trial Readiness for Genetic Developmental and Epileptic Encephalopathies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2021Review
Corrections and comments
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Authors and funding
15 authors at 9 institutions in 2 countries.
Funding
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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.