Effectiveness of Cenobamate in Pediatric Patients With Lennox-Gastaut Syndrome: A Retrospective Claims-based Analysis
Karen Keough1, Sean Stern2, Clarence Wade2, Mindl Weingarten2
1Pediatrix Child Neurology Consultants of Austin and Texas A&M University Health Science Center ‒ Round Rock, 2SK Life Science, Inc.
Objective:
To compare outcomes in pediatric patients with Lennox-Gastaut syndrome (LGS) taking cenobamate vs their previous antiseizure medications (ASMs) using data from a national claims database.
Background:
Small case series have reported benefit with off-label cenobamate in patients with drug-resistant pediatric developmental encephalopathies.
Design/Methods:
Patients ≤17 years old with LGS (ICD-10-CM G40.81*) taking ≥1 ASM between 5/1/2020-12/31/2022 were identified retrospectively from the HealthVerity Marketplace Private Source 20 database. Patients were required to have taken ≥2 prescription fills of cenobamate and have ≥180 days of medical and pharmacy enrollment before and after initiating cenobamate. Patients with a history of Dravet syndrome were excluded (n=1). Data were analyzed by line of therapy (LOT); a new LOT was identified as the dispensing of a new ASM after ≥30 days without a previous fill. Health utilization outcomes (eg, epilepsy-related inpatient days and emergency room [ER] visits) during the patient’s previous therapies were compared to outcomes when on cenobamate.
Results:
76 patients (41% female, mean age 13.4 years, age range 2-17 years; mean previous LOT=6.01) with LGS were included. The median line duration was 371.2 days with cenobamate vs 377.2 days on the previous ASM combinations. The rate of inpatient days per year improved from 3.94 to 3.36, and the rate of ER days per year improved from 1.19 to 0.66. Among patients without a previous history of status epilepticus, new status epilepticus occurred in 20% (3/15) during the previous ASM combinations compared to 7.7% (2/26) during cenobamate treatment. 35.5% of patients on cenobamate required a new LOT during follow-up.
Conclusions:
Cenobamate was associated with fewer inpatient days and ER visits and lower rates of status epilepticus compared to patients’ previous ASM combinations. Further studies examining the causal nature of these outcomes and studies evaluating cenobamate use in patients with LGS are warranted.