Abstracts

Rationale: To date, studies evaluating rufinamide as add-on therapy in children with refractory epilepsy have been limited by small numbers and short follow-up periods. Accordingly, we retrospectively evaluated the use of rufinamide in a large cohort of epilepsy patients from a single pediatric center with long-term follow-up. Methods: We included all patients taking rufinamide for refractory epilepsy in a retrospective review of medical records from November 2008 to March 2013. Follow-up was divided into 2 categories: <12 months and 12 months. Response to treatment was defined as a reduction in seizure frequency by 50% compared to baseline prior to introduction of rufinamide. Results: Three-hundred patients (age: median 9.1 years, interquartile range 4-14.6 years, range 0.4-29.6 years) were included [Table 1]. Median follow-up was 9 months (range 1 -37 months). The mean starting dose of rufinamide was 8.8 mg/kg/day (range 0.9-52.6), with a mean target dose of 39.5 mg/kg/day (range 1.8-135). At baseline 46 (15.3%) children were taking one other antiepileptic drug (AED), 76 (25.3%) two, 91 (30.3%) three and 81 (27%) more than three other AED. Epilepsy etiology was classified as genetic (23.7%), structural/metabolic (41%), and unknown cause (35.3%). Overall, rufinamide treatment led to a seizure reduction of 59.2% (interquartile range 0-92.2). Overall, response to treatment with rufinamide was more frequent in the group of patients with genetic etiology (66.2% responders) compared to patients with structural/metabolic etiology (45.5%) or unknown cause (57.5%; Fisher s exact test: p = 0.017). In the first year of follow-up [Table 2], rufinamide treatment led to a response in 158/293 (53.9%) children (14% seizure free). Discontinuation was observed in 110/300 (36.7%) patients: in 63 (21%) due to unsatisfactory response, in 47 (15.7%) due to side effects, and due to both in 18 (6%) cases. Overall, side effects were observed in 79 (26.3%) patients. Furthermore, 39 (13%) had incomplete data at follow-up and 17 (5.7%) showed no compliance. For the remaining 134 patients that were on rufinamide after the first year, 74/134 (55.2%) continued as responders, 18 (13.4%) were de novo responders, 35 (26.1%) relapsed (7 [5.2%] had follow up only after the first year). After one year of treatment [Table 2], 95/134 (70.9%) showed a response to rufinamide treatment (16.4% seizure free). Overall, the most common reported adverse events with rufinamide treatment include sleepiness in 21/47 (44.7%), vomiting in 17/47 (36.2%), mood changes in 12/47 (25.5%) and loss of appetite in 8/47 (17%). Conclusions: Our experience in a large cohort suggests that rufinamide provides good seizure reduction as an adjunctive treatment in refractory pediatric epilepsy. In addition, it may provide efficacy after the first year. Side effects were observed in 26% of the patients but lead to discontinuation only in a small group of patients.