Aguilar-Amat Prior Maria José, Alonso Singer Pablo, Oliva Navarro Javier, Olivie Garcia Laura, Machio Castello Maria, Beltran-Corbellini Álvaro, Sánchez-Larsen Álvaro, Parejo-Carbonell Beatriz, de Toledo-Heras Maria, Vieira Campos Alba, Gonzalez-Villar Esther, Mercedes-Alvarez Blanca, Escobar-Montalvo Juan Manuel
Epilepsy Program. Department of Neurology. Hospital Universitario La Paz. Universidad Autonoma de Madrid Researcher, IdiPAZ´s Neurology and Cerebrovascular Diseases Research Group, Hospital La Paz Institute for Health Research, IdiPAZ, Spain.
Epilepsy Program. Department of Neurology. Hospital Universitario La Paz. Universidad Autonoma de Madrid Researcher, IdiPAZ´s Neurology and Cerebrovascular Diseases Research Group, Hospital La Paz Institute for Health Research, IdiPAZ, Spain.
Seizure. 2025 Sep;131:84-89. doi: 10.1016/j.seizure.2025.05.011. Epub 2025 May 27.
Lennox-Gastaut syndrome (LGS) is a rare treatment-resistant epilepsy classed as developmental and epileptic encephalopathy (DEE). In this study we investigated the effectiveness and safety of cenobamate (CNB) as adjunctive therapy in adults with LGS under real-world conditions.
We conducted a retrospective analysis of clinical data collected from patients diagnosed with LGS who were prescribed CNB in 8 different sites. Data was sourced from patient clinical records. Effectiveness was evaluated by seizure type (total seizures, focal onset seizures, generalized tonic-clonic seizures [GTCS], drop seizures and atypical absence) and included ≥50 %, ≥75 %, ≥90 % responder rate and seizure freedom rate at 3, 6 and 12month visits. Changes in the number of co-antiseizure medication (co-ASM) were also analyzed. Safety/tolerability was monitored by documenting the incidence of adverse events (AE) and AEs leading to discontinuation.
18 patients with LGS were included in the analysis (34.4 % women, mean age 25.2 years, and median number of seizures per month 195 (IQR: 12-1416)). The median of number of prior ASMs and concomitant ASMs were 12 (IQR: 8-16) and 3 (IQR: 2-6) respectively. Median CNB dosages/day was 200 mg (IQR: 50-350) at 3, 6 and 12 months. At 3, 6, and 12 months, 94.4 %, 94.4 % and 83.3 % of participants were retained on CNB treatment, respectively. At the last available visit, the seizure freedom rate was 12.5 %, ≥50 %, ≥75 %, ≥90 % responder rates were 46.2 %, 23.1 %, and 0 %, respectively. The number of co-ASMs was reduced in 36 % of patients. The percentage of patients with AEs and AEs leading to discontinuation was 0 %. The most frequent AEs were somnolence and bradypsychia or dizziness.
In this study, CNB demonstrated high effectiveness and good tolerability in patients with LGS when administered in adjuvancy in real-world practice after the failure of multiple ASMs. AEs were frequent but mostly mild-to-moderate.
