Psychometric evaluation of clinician- and caregiver-reported clinical severity assessments for individuals with CDKL5 deficiency disorder.
Jacinta M SaldarisPeter JacobyJennepher Anne DownsEric D MarshHelen Margaret LeonardElia M Pestana KnightRajsekar RajaramanJudith WeisenbergBernhard SuterHeather E OlsonDana PriceWilliam HongErin PrangeTimothy A BenkeScott T DemarestPublished in: Epilepsia (2024)
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.