Felbamate in the treatment of refractory epileptic spasms.

Several small case series provide conflicting impressions of the efficacy of felbamate for treatment of epileptic spasms. Using a large single-center cohort of children with epileptic spasms, we retrospectively evaluated the efficacy and safety of felbamate. We identified all patients with video-EEG confirmed epileptic spasms who were treated with felbamate at our center. We quantified felbamate exposure by calculating peak and weighted-average weight-based dose. Clinical response was defined as resolution of epileptic spasms for at least 28 days, beginning not more than 3 months after felbamate initiation. Electroclinical response was defined as clinical response accompanied by overnight video-EEG demonstrating freedom from epileptic spasms and hypsarrhythmia. Among a cohort of 476 infants, we identified 62 children who were treated with felbamate, of whom 58 had previously failed treatment with hormonal therapy or vigabatrin. Median peak and weighted-average felbamate dosages were 47 and 40 mg/kg/day, respectively. Five (8%) children were classified as clinical responders and two (3%) children were classified as electroclinical responders. Among 17 patients with latency from epileptic spasms onset to felbamate initiation of less than 12 months, we observed 4 (24%) clinical responders. This study suggests that felbamate may be efficacious for treatment of epileptic spasms and that further rigorous study is warranted.

Informations de copyright

Copyright © 2020 Elsevier B.V. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of Competing Interest Dr. Hussain has received research support from the Epilepsy Therapy Project, the Milken Family Foundation, the Hughes Family Foundation, the Elsie and Isaac Fogelman Endowment, Eisai, Lundbeck, Insys, GW Pharma, UCB Biopharma, Zogenix, and the NIH (R34MH089299). He has received honoraria for service on the scientific advisory boards of Mallinckrodt, Upsher-Smith Laboratories, Insys, UCB Biopharma, and Zogenix; as a consultant to UCB, Mallinckrodt, Insys, GW Pharma, West Therapeutic Development, Aquestive Therapeutics, Shennox, and Amzell; and on the speaker’s bureau for Mallinckrodt and GW Pharmaceuticals. Dr. Rajaraman has received research support from Marinus and the International CDKL5 Research Foundation. Dr. Shrey has received research support from the Lennox-Gastaut Syndrome Foundation, UCB Biopharma, and Mallinckrodt, as well as the CHOC Children’s Physician Subspecialty Faculty Tithe Fund and the CHOC Children’s Physician Scientist Scholars Program. Dr. Sankar serves on scientific advisory boards or serves on the speaker’s bureau for and has received honoraria and funding for travel from Eisai, UCB Pharma, Sunovion, Supernus, Greenwich Biosciences, LivaNova, and on the advisory boards for Aquestive Therapeutics, West Therapeutic Development, Insys Development Company, and Zogenix; receives royalties from the publication of Pellock’s Pediatric Epilepsy, 4th ed. (Demos Publishing, 2016) and Epilepsy: Mechanisms, Models, and Translational Perspectives (CRC Press, 2011); serves on speakers’ bureaus for and has received speaker honoraria from Eisai, UCB, GlaxoSmithKline, LivaNova, Supernus, and BioMarin. He has received research support from SK Life Sciences and Insys Development Company, Inc. The remaining authors report no conflicts of interest.