Abstracts

Rationale: Because few studies on the long-term safety and efficacy of rufinamide (Inovelon®) in patients with Lennox-Gastaut syndrome (LGS) have been reported, we evaluated these characteristics of rufinamide using Japanese real-world data. Methods: Rufinamide, marketed in Japan since 2013, is an anti-epileptic drug (AED) that was granted orphan drug status as an adjunctive therapy for tonic-atonic seizures associated with LGS. This ongoing, long-term, nationwide prospective observational study followed all patients treated with rufinamide for a maximum of 2 years in clinical practice. Patient characteristics, side effects, frequency of tonic-atonic seizure, and overall symptom improvement were summarized descriptively. Side effects were further summarized by the timing of occurrence after rufinamide administration: within 6 months, >6 months–1 year, and >1 year. Results: Case report forms for 704 patients were collected from 178 institutions as of January 2017. Of these, 691 patients were included in the safety analysis set and 490 patients were included in the efficacy analysis set (comprising LGS patients only). In the safety analysis set, 85.5% were patients with LGS, and 14.5% were non-LGS. Women accounted for 43.1%. Mean (±standard deviation) age was 18.3±11.4 years, weight was 39.0±19.5 kg, and duration since diagnosis of epilepsy was 14.4±11.9 years. For adjunctive therapy, 51.4% of patients concomitantly received Conclusions: The results of this study in a real-world setting were comparable with those from clinical trials in terms of the safety and efficacy of rufinamide. Because limited research on LGS has been reported, our study may contribute to the understanding of this condition in Japan. Funding: This study was funded by Eisai Co., Ltd.