Felbamate Treatment Persistence in Children with Epilepsy
Abstract number :
3.367
Submission category :
7. Anti-seizure Medications / 7C. Cohort Studies
Year :
2025
Submission ID :
615
Source :
www.aesnet.org
Presentation date :
12/8/2025 12:00:00 AM
Published date :
Authors :
Presenting Author: Dominique Cooper, MD – The Children's Hospital of Philadelphia
Sudha Kessler, MD, MSCE – The Children's Hospital of Philadelphia
Christina Bergqvist, MD – The Children's Hospital of Philadelphia
Mark Fitzgerald, MD, PhD – The Children's Hospital of Philadelphia
Eva Catenaccio, MD – The Children's Hospital of Philadelphia/University of Pennsylvania
Rationale: Felbamate (FBM) is approved for treatment of focal seizures and Lennox-Gastaut Syndrome, but its use has been limited by rare but severe idiosyncratic reactions. We aim to describe the contemporary use FBM in children with epilepsy. We aim to evaluate the effectiveness of FBM in pediatric patients using time to treatment failure as the outcome measure and to evaluate reasons for FBM discontinuation.
Methods: A retrospective cohort study was performed of patients under 21 years old at Children’s Hospital of Philadelphia receiving FBM from 2013 to 2023, with manual extraction of demographic and clinical data. Kaplan-Meier survival curves were generated for time to FBM failure, defined as discontinuation of FLB or addition of another antiseizure medication.
Results: The analysis cohort included 27 patients (15 females) with median follow-up of 45 months (range 0.4 - 94 months). Eight (30%) patients had focal epilepsy, 8 (30%) had generalized epilepsy, 9 (33%) had mixed epilepsy, and 2 patients (7%) had an undetermined epilepsy type. The majority of patients had Lennox-Gastaut Syndrome (73%). The mean age at FBM initiation was 10.3±4.6 years (range 1.8 – 17.8 years). At the time of FBM initiation, the median number of concomitant antiseizure medications (ASM) was 3 (range 1-6) and the median number of prior ASM was 5.5 (range 2-16). The median time to FBM failure was 12.5 months (95% CI 5.7 – 26.0 months). For all patients, the probability of remaining on FBM without the addition of another therapy was 52% at 12 months (95% CI 32-69%) and 34% at 24 months (95% CI 16-53%). Fifty-two percent of patients discontinued FBM during the observation period. The reason for discontinuation was lack of efficacy in 43%, lack of tolerability in 29%, and lack of tolerability and efficacy in 21%. One patient discontinued due to seizure freedom. Three patients (11.1%) developed leukopenia, one (3.7%) developed anemia and one patient (3.7%) developed transaminitis while on FBM; these adverse effects resolved with FBM discontinuation.
Conclusions: More than 50% of children with epilepsy who are treated with FBM will continue to take the treatment for a year or more without additional treatments added or treatment discontinuation. FBM is more often discontinued for lack efficacy than lack of tolerability or safety. In this cohort, FBM use was safe. Reversible adverse blood and hepatic toxicities occurred in a small proportion of patients and was not definitively proven to be caused by FBM.
Funding: N/A
Anti-seizure Medications