{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,17]],"date-time":"2026-09-17T08:08:23Z","timestamp":1789632503381,"version":"build-2803163510"},"reference-count":34,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2024,8,27]],"date-time":"2024-08-27T00:00:00Z","timestamp":1724716800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/501100010091","name":"International Foundation for CDKL5 Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100010091","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Epilepsia"],"published-print":{"date-parts":[[2024,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>The CDKL5 Clinical Severity Assessment (CCSA) is a comprehensive, content\u2010validated measurement tool capturing the diverse challenges of cyclin\u2010dependent kinase\u2010like 5 (CDKL5) deficiency disorder (CDD), a genetically caused developmental epileptic encephalopathy (DEE). The CCSA is divided into clinician\u2010reported (CCSA\u2010Clinician) and caregiver\u2010reported (CCSA\u2010Caregiver) 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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Participants were recruited from the International CDKL5 Clinical Research Network to take part in an in\u2010clinic CCSA\u2010Clinician evaluation (<jats:italic>n<\/jats:italic>\u2009=\u2009148) and\/or complete the CCSA\u2010Caregiver questionnaire (<jats:italic>n<\/jats:italic>\u2009=\u2009198). CFA was used to determine domains, and factor loadings and validity were assessed. For the CCSA\u2010Clinician, inter\u2010rater reliability was assessed by nine CDD experienced clinicians via 14 pre\u2010recorded evaluations. Eight clinicians re\u2010viewed and re\u2010scored the videos after 4\u2009weeks to evaluate intra\u2010rater reliability. The CCSA\u2010Caregiver was completed on a second occasion by 34 caregivers after 2\u20134\u2009weeks to assess test\u2013retest reliability.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>CFA resulted in three domains for the CCSA\u2010Clinician (motor and movement, communication, vision) and four domains for the CCSA\u2010Caregiver (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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Significance<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec>","DOI":"10.1111\/epi.18094","type":"journal-article","created":{"date-parts":[[2024,8,27]],"date-time":"2024-08-27T15:29:45Z","timestamp":1724772585000},"page":"3064-3075","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Psychometric evaluation of clinician\u2010 and caregiver\u2010reported clinical severity assessments for individuals with <scp>CDKL5<\/scp> deficiency disorder"],"prefix":"10.1111","volume":"65","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4638-6589","authenticated-orcid":false,"given":"Jacinta M.","family":"Saldaris","sequence":"first","affiliation":[{"name":"Telethon Kids Institute, Centre for Child Health Research The University of Western Australia  Perth Western Australia Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5092-7697","authenticated-orcid":false,"given":"Peter","family":"Jacoby","sequence":"additional","affiliation":[{"name":"Telethon Kids Institute, Centre for Child Health Research The University of Western Australia  Perth Western Australia Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7358-9037","authenticated-orcid":false,"given":"Jenny","family":"Downs","sequence":"additional","affiliation":[{"name":"Telethon Kids Institute, Centre for Child Health Research The University of Western Australia  Perth Western Australia Australia"},{"name":"Curtin School of Allied Health Curtin University  Perth Western Australia Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3264-0902","authenticated-orcid":false,"given":"Eric D.","family":"Marsh","sequence":"additional","affiliation":[{"name":"Departments of Neurology and Pediatrics, Division of Child Neurology and University of Pennsylvania Perelman School of Medicine Children's Hospital of Philadelphia  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6405-5834","authenticated-orcid":false,"given":"Helen","family":"Leonard","sequence":"additional","affiliation":[{"name":"Telethon Kids Institute, Centre for Child Health Research The University of Western Australia  Perth Western Australia Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9068-4167","authenticated-orcid":false,"given":"Elia","family":"Pestana\u2010Knight","sequence":"additional","affiliation":[{"name":"Cleveland Clinic Neurological Institute  Cleveland Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9685-7118","authenticated-orcid":false,"given":"Rajsekar","family":"Rajaraman","sequence":"additional","affiliation":[{"name":"UCLA Mattel Children's Hospital  Los Angeles California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2031-7278","authenticated-orcid":false,"given":"Judith","family":"Weisenberg","sequence":"additional","affiliation":[{"name":"St. Louis Children's Hospital and Washington University School of Medicine  St. Louis Missouri USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0127-7322","authenticated-orcid":false,"given":"Bernhard","family":"Suter","sequence":"additional","affiliation":[{"name":"Department of Pediatrics &amp; Neurology Baylor College of Medicine  Houston Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5385-0119","authenticated-orcid":false,"given":"Heather E.","family":"Olson","sequence":"additional","affiliation":[{"name":"Division of Epilepsy and Clinical Neurophysiology and Epilepsy Genetics Program, Department of Neurology Boston Children's Hospital  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3969-6394","authenticated-orcid":false,"given":"Dana","family":"Price","sequence":"additional","affiliation":[{"name":"NYU Langone Health and Department of Neurology New York University  New York City New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0003-4208-1857","authenticated-orcid":false,"given":"William","family":"Hong","sequence":"additional","affiliation":[{"name":"Division of Epilepsy and Clinical Neurophysiology and Epilepsy Genetics Program, Department of Neurology Boston Children's Hospital  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-2609-9187","authenticated-orcid":false,"given":"Erin","family":"Prange","sequence":"additional","affiliation":[{"name":"Departments of Neurology and Pediatrics, Division of Child Neurology and University of Pennsylvania Perelman School of Medicine Children's Hospital of Philadelphia  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6969-5061","authenticated-orcid":false,"given":"Tim A.","family":"Benke","sequence":"additional","affiliation":[{"name":"Departments of Pediatrics, Neurology and Pharmacology University of Colorado School of Medicine and Children's Hospital Colorado  Aurora Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4544-3724","authenticated-orcid":false,"given":"Scott","family":"Demarest","sequence":"additional","affiliation":[{"name":"Departments of Pediatrics and Neurology, Precision Medicine Institute University of Colorado School of Medicine and Children's Hospital Colorado  Aurora Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,8,27]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2019.02.015"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejpn.2021.04.007"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awz195"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.37851"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41431-022-01163-1"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1097\/GIM.0b013e31820605f5"},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/cge.13862"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2022.120498"},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(22)00035-7"},{"key":"e_1_2_13_11_1","volume-title":"Guidance for industry: patient\u2010reported outcome measures: use in medical product development to support labeling claims","author":"US Department of Health and Human Services Food and Drug Administration (FDA). 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