Evidence map›Paper›PMID 39190322›Full record

ArticleEpilepsia2024

Psychometric evaluation of clinician- and caregiver-reported clinical severity assessments for individuals with CDKL5 deficiency disorder.

Jacinta M Saldaris, Peter Jacoby, Jenny Downs, Eric D Marsh, Helen Leonard, Elia Pestana-Knight, Rajsekar Rajaraman, Judith Weisenberg, Bernhard Suter, Heather E Olson and 5 more

Abstract readEvaluation Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Epilepsia, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Article
  2. Observational
  3. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Jacinta M SaldarisTelethon Kids Institute, Centre for Child Health Research, The University of Western Australia, Perth, Western Australia, Australia.ORCID https://orcid.org/0000-0003-4638-6589
Peter JacobyTelethon Kids Institute, Centre for Child Health Research, The University of Western Australia, Perth, Western Australia, Australia.ORCID https://orcid.org/0000-0001-5092-7697
Jenny DownsTelethon Kids Institute, Centre for Child Health Research, The University of Western Australia, Perth, Western Australia, Australia.ORCID https://orcid.org/0000-0001-7358-9037
Eric D MarshDepartments of Neurology and Pediatrics, Division of Child Neurology and University of Pennsylvania Perelman School of Medicine, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.ORCID https://orcid.org/0000-0003-3264-0902
Helen LeonardTelethon Kids Institute, Centre for Child Health Research, The University of Western Australia, Perth, Western Australia, Australia.ORCID https://orcid.org/0000-0001-6405-5834
Elia Pestana-KnightCleveland Clinic, Neurological Institute, Cleveland, Ohio, USA.ORCID https://orcid.org/0000-0001-9068-4167
Rajsekar RajaramanUCLA Mattel Children's Hospital, Los Angeles, California, USA.ORCID https://orcid.org/0000-0002-9685-7118
Judith WeisenbergSt. Louis Children's Hospital and Washington University School of Medicine, St. Louis, Missouri, USA.ORCID https://orcid.org/0000-0003-2031-7278
Bernhard SuterDepartment of Pediatrics & Neurology, Baylor College of Medicine, Houston, Texas, USA.ORCID https://orcid.org/0000-0002-0127-7322
Heather E OlsonDivision of Epilepsy and Clinical Neurophysiology and Epilepsy Genetics Program, Department of Neurology, Boston Children's Hospital, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0002-5385-0119
Dana PriceNYU Langone Health and Department of Neurology, New York University, New York City, New York, USA.ORCID https://orcid.org/0000-0003-3969-6394
William HongDivision of Epilepsy and Clinical Neurophysiology and Epilepsy Genetics Program, Department of Neurology, Boston Children's Hospital, Boston, Massachusetts, USA.ORCID https://orcid.org/0009-0003-4208-1857
Erin PrangeDepartments of Neurology and Pediatrics, Division of Child Neurology and University of Pennsylvania Perelman School of Medicine, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.ORCID https://orcid.org/0009-0002-2609-9187
Tim A BenkeDepartments of Pediatrics, Neurology and Pharmacology, University of Colorado School of Medicine and Children's Hospital Colorado, Aurora, Colorado, USA.ORCID https://orcid.org/0000-0002-6969-5061
Scott DemarestDepartments of Pediatrics and Neurology, Precision Medicine Institute, University of Colorado School of Medicine and Children's Hospital Colorado, Aurora, Colorado, USA.ORCID https://orcid.org/0000-0002-4544-3724

Funding

Genetic Analysis and Manipulation Core (GAEC)P50HD105351 · NICHD · BOSTON CHILDREN'S HOSPITAL · PI SCOTT Loren POMEROY, MUSTAFA SAHIN · 2021 to 2026
$9.4M
Multi-Site Validation of Biomarkers and Core Clinical Outcome Measures for Clinical Trials Readiness in CDKL5 Deficiency DisorderU01NS114312 · NINDS · UNIVERSITY OF COLORADO DENVER · PI BENKE, TIMOTHY A, DOWNS, JENNY · 2021 to 2025
$4.7M
International Foundation for CDKL5 ResearchNational Institutes of Health (NIH) U01NS114312NICHD NIH HHS P50 HD105351NINDS NIH HHS U01 NS114312
6 · The paper itself

Abstract

objectiveThe CDKL5 Clinical Severity Assessment (CCSA) is a comprehensive, content-validated measurement tool capturing the diverse challenges of cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD), a genetically caused developmental epileptic encephalopathy (DEE). The CCSA is divided into clinician-reported (CCSA-Clinician) and caregiver-reported (CCSA-Caregiver) assessments. The aim of this study was to evaluate the factor structure of these measures through confirmatory factor analysis (CFA) and evaluate their validity and reliability.

methodsParticipants were recruited from the International CDKL5 Clinical Research Network to take part in an in-clinic CCSA-Clinician evaluation (n = 148) and/or complete the CCSA-Caregiver questionnaire (n = 198). CFA was used to determine domains, and factor loadings and validity were assessed. For the CCSA-Clinician, inter-rater reliability was assessed by nine CDD experienced clinicians via 14 pre-recorded evaluations. Eight clinicians re-viewed and re-scored the videos after 4 weeks to evaluate intra-rater reliability. The CCSA-Caregiver was completed on a second occasion by 34 caregivers after 2-4 weeks to assess test-retest reliability.

resultsCFA resulted in three domains for the CCSA-Clinician (motor and movement, communication, vision) and four domains for the CCSA-Caregiver (seizures, behavior, alertness, feeding), with good item loadings across both measures. Structural statistics, internal consistency, discriminant validity, and reliability were satisfactory for both measures, and scores were consistent between known groups. SIGNIFICANCE: This study provides strong evidence that the CCSA measures are suitable to assess the clinical severity of individuals with CDD, supporting their use in clinical trials. Further evaluation of responsiveness to change in a longitudinal assessment is planned. Use may also be appropriate in similar DEEs but would require validation in those populations.

Indexed as

CaregiversEpileptic SyndromesPhysiciansPsychometricsSpasms, InfantileAdolescentChildChild BehaviorChild, PreschoolFemaleHumansInfantInfant, NewbornMaleReproducibility of ResultsSeverity of Illness Indexclinical outcome assessmentconfirmatory factor analysisdevelopmental epileptic encephalopathyreliabilityvalidity

Identifiers

PMID39190322
PMCPMC11495992

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Registered trials

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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.