Authors :
Presenting Author: Karen Keough, MD – Pediatrix CNCA and Texas A&M University Health Science Center
Sean Stern, MS – SK Life Science, Inc.; Clarence T. Wade, MBA – SK Life Science, Inc.; Mindl Weingarten, PharmD – SK Life Science, Inc.
Rationale:
Cenobamate (XCOPRI®) is an antiseizure medication (ASM) approved in the United States and Europe (ONTOZRY®) as a treatment for adults with focal seizures. Several small case series have reported benefit with off-label cenobamate in patients with drug-resistant pediatric developmental encephalopathies. This retrospective observational analysis used data from a national claims database to compare outcomes in pediatric patients with Lennox-Gastaut syndrome (LGS) taking cenobamate versus their previous line of therapy.
Methods:
Patients ≤17 years old with a diagnosis of LGS (ICD-10-CM G40.81*) taking at least 1 script of cenobamate between 1/1/2020-12/31/2022 were identified from the HealthVerity Marketplace Private Source 20 database.brPatients 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 therapies were compared to outcomes when on cenobamate. Outcomes included: retention, rate of epilepsy-related inpatient visit days, rate of epilepsy-related ER visit days, new status epilepticus, and new line of therapy.
Results:
A total of 125 patients (57.6% female, mean age 13.6 years, age range 2-17 years) with LGS were exposed to cenobamate. Patients starting cenobamate had received an average of 6.5 previous lines of therapy. The median (min, max) duration of follow-up was 213 days (30, 790 days). All assessed outcomes improved with the addition of cenobamate vs the previous combination of therapies. The median duration of retention was 434 days with cenobamate versus 344 days on the previous combination of therapies. The rate of inpatient days improved from 4.5 per 100 days to 1.8 per 100 days, and the rate of ER visits improved from 0.3 per 100 days to 0.2 per 100 days. Among patients without a previous history of status epilepticus, new status epilepticus occurred in 20.6% (7/34) during the previous combination of therapies compared to 7.7% (2/26) during cenobamate treatment. Overall, 28.8% of patients on cenobamate required a new line of therapy.
Conclusions: In this claims-based analysis, cenobamate was associated with longer retention, fewer IP days and ER visits, and lower rates of status epilepticus compared to patients’ previous combination of therapies. Further studies examining the causal nature of cenobamate’s association on these outcomes and studies evaluating the use of cenobamate in patients with LGS are warranted.
Funding:
Funded by SK Life Science, Inc.