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The purpose of this pilot study was to analyze treatment pathways of pediatric epilepsy using the common data model (CDM) based on electronic health record (EHR) data. We also aimed to reveal whether CDM analysis was feasible and applicable to epilepsy research. We analyzed the treatment pathways of pediatric epilepsy patients from our institute who underwent antiseizure medication (ASM) treatment for at least 2 years, using the Observational Medical Outcomes Partnership (OMOP)-CDM. Subgroup analysis was performed for generalized or focal epilepsy, varying age of epilepsy onset, and specific epilepsy syndromes. Changes in annual prescription patterns were also analyzed to reveal the different trends. We also calculated the proportion of drug-resistant epilepsy by applying the definition of seizure persistence after application of two ASMs for a sufficient period of time (more than 6 months). We identified 1,192 patients who underwent treatment for more than 2 years (mean ± standard deviation: 6.5 ± 3.2 years). In our pediatric epilepsy cohort, we identified 313 different treatment pathways. Drug resistance, calculated as the application of more than three ASMs during the first 2 years of treatment, was 23.8%. Treatment pathways and ASM resistance differed between subgroups of generalized vs. focal epilepsy, different onset age of epilepsy, and specific epilepsy syndromes. The frequency of ASM prescription was similar between onset groups of different ages; however, phenobarbital was frequently used in children with epilepsy onset < 4 years. Ninety-one of 344 cases of generalized epilepsy and 187 of 835 cases of focal epilepsy were classified as medically intractable epilepsy. The percentage of drug resistance was markedly different depending on the specific electro-clinical epilepsy syndrome [79.0% for Lennox-Gastaut syndrome (LGS), 7.1% for childhood absence epilepsy (CAE), and 9.0% for benign epilepsy with centrotemporal spikes (BECTS)]. We could visualize the annual trend and changes of ASM prescription for pediatric epilepsy in our institute from 2004 to 2017. We revealed that CDM analysis was feasible and applicable for epilepsy research. The strengths and limitations of CDM analysis should be carefully considered when planning the analysis, result extraction, and interpretation of results.
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http://dx.doi.org/10.3389/fneur.2020.00409 | DOI Listing |
J Clin Invest
September 2025
Department of Clinical and Biomedical Sciences, Faculty of Health and Life Sciences, University of Exeter, Exeter, United Kingdom.
Understanding the genetic causes of diseases affecting pancreatic β cells and neurons can give insights into pathways essential for both cell types. Microcephaly, epilepsy and diabetes syndrome (MEDS) is a congenital disorder with two known aetiological genes, IER3IP1 and YIPF5. Both genes encode proteins involved in endoplasmic reticulum (ER) to Golgi trafficking.
View Article and Find Full Text PDFQual Life Res
September 2025
The Kids Research Institute Australia, The University of Western Australia, P.O. Box 855, West Perth, WA, 6872, Australia.
Purpose: CDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy. Greater understanding of the smallest meaningful improvements for individuals with CDD in clinical trials and practice is needed for a person-centred approach to treatment efficacy. This study explored how parent/caregivers of people with CDD understood meaningful improvements and described change for priority functional domains including communication, gross motor, fine motor, feeding.
View Article and Find Full Text PDFJ Behav Med
September 2025
Behavioral Medicine and Clinical Psychology, Cincinnati Children's Hospital Medical Center, Department of Pediatrics, University of Cincinnati College of Medicine, 3333 Burnet Avenue, MLC 7039, Cincinnati, OH, 45229, USA.
Randomized pretest, posttest, follow-up (RPPF) designs are widely used in longitudinal behavioral intervention research to evaluate the efficacy of treatments over time. These designs typically involve random assignment of participants to treatment and control conditions, with assessments conducted at baseline, immediately post-intervention, and during the follow-up period. Researchers primarily focus on determining whether the intervention is more effective than the control condition at post-treatment and whether these effects are sustained or change over time.
View Article and Find Full Text PDFEpilepsia
September 2025
Department of Pharmacology and Neuroscience, Creighton University School of Medicine, Omaha, Nebraska, USA.
The rate of sudden unexpected death in epilepsy (SUDEP) is ~1 per 1000 patients each year. Terminal events reportedly involve repeated and prolonged apnea, suggesting a failure to autoresuscitate. To better understand the mechanisms and identify novel therapeutics, standardized tests to screen for autoresuscitation efficacy are needed in preclinical SUDEP.
View Article and Find Full Text PDFPediatr Crit Care Med
September 2025
Department of Anesthesiology and Critical Care, Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.
Objective: To develop a set of pediatric neurocritical care (PNCC) entrustable professional activities (EPAs) for pediatric critical care medicine (PCCM).
Design: Survey and Delphi methodology in a panel of experts from the Pediatric Neurocritical Care Research Group (PNCRG) and the Education in Pediatric Intensive Care (EPIC) Research Collaborative.
Setting: Interprofessional local focus group, national focus group, and subsequent national multi-institutional, multidisciplinary expert panel in the United States.