Authors :
Presenting Author: Katherine Labiner, MD – Child Neurology Consultants of Austin, part of Pediatrix Medical Group
Sean Stern, MS – SK Life Science, Inc.; Clarence T. Wade, MBA – SK Life Science, Inc.; Mindl Weingarten, PharmD – SK Life Science, Inc.
Rationale:
Cenobamate is an antiseizure medication (ASM) approved in the United States (XCOPRI®) and Europe (ONTOZRY®) as a treatment for adults with focal seizures. Several small retrospective studies have reported benefit with off-label cenobamate in pediatric patients with drug-resistant epilepsy. This retrospective observational analysis used data from a national claims database to compare outcomes in pediatric patients with intractable focal seizures taking cenobamate versus their prior ASMs. Methods:
Patients ≤17 years old with a diagnosis of intractable focal seizures (ICD-10-CM G40.219*) taking at least 1 ASM between 1/1/2020 through 12/31/2021 were identified from the HealthVerity Marketplace Private Source 2020 database. Patients were required to have ≥12 months of medical and pharmacy enrollment before initiating cenobamate. Patients’ data were analyzed by line of therapy, whereby a new line of therapy was identified as the dispensing of a new ASM after ≥30 days without a previous fill. Patients who received a sequence of ASMs during follow-up would contribute multiple lines of therapy to the analysis. We compared outcomes using a “self-control” approach, whereby outcomes during the patient’s previous line of therapy were compared to outcomes when on cenobamate. Outcomes included: retention, rate of epilepsy-related inpatient (IP) visit days, rate of epilepsy-related emergency (ER) visit days, epilepsy monitoring unit hours, new status epilepticus, and new ASM therapy.Results:
A total of 247 patients with intractable focal seizures were exposed to cenobamate. Mean line of therapy at cenobamate initiation was 6.7. The median duration of follow-up was 213 days (range 30-882 days). All assessed outcomes improved with cenobamate vs the previous ASMs. The median duration of retention was 447 days with cenobamate vs 357 days on the previous ASM. The rate of epilepsy-related IP days improved from 4.48 per 100 days to 1.43 per 100 days, and the rate of epilepsy-related ER visits improved from 0.34 per 100 days to 0.15 per 100 days. The number of hours in the epilepsy monitoring unit decreased from an average of 26.0 to 19.5 per patient. Among patients without a previous history of status epilepticus, new status epilepticus occurred in 18.5% (17/92) during the previous line of therapy compared to 13.3% (10/75) during cenobamate treatment. Overall, 29.6% of patients on cenobamate required a new ASM to be added.Conclusions:
Cenobamate was associated with notable improvement in outcomes in pediatric patients with intractable focal epilepsy. Further studies evaluating the use of cenobamate in pediatric patients with intractable focal seizures are warranted.Funding:
Funded by SK Life Science, Inc.