Safety and retention rate of rufinamide in 300 patients: A single pediatric epilepsy center experience

Sigride Thome-Souza*, Navah E. Kadish, Sriram Ramgopal, Iván Sánchez Fernández, Ann M. Bergin, Jeffrey Bolton, Chellamani Harini, Mark Libenson, Heather Olson, Jurriaan Peters, Annapurna Poduri, Alexander Rotenberg, Masanori Takeoka, Sanjeev V. Kothare, Kush Kapur, Blaise F.D. Bourgeois, Tobias Loddenkemper

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

16 Scopus citations


Summary Objective Reports of studies evaluating rufinamide as an add-on therapy in children and adolescents with refractory epilepsy are restricted to a few publications. Prospective multicenter studies including children and adults have yielded important information about several types of epilepsies and syndromes. We evaluated the use of rufinamide in a single pediatric center with a large cohort and long-term follow-up period. Methods We retrospectively included patients taking rufinamide from November 2008 to March 2013. Response was defined by a seizure reduction of ≥50% compared to baseline. Results Three hundred patients with a median age of 9.1 years (range 0.4-29.6 years) were reviewed. Median follow-up was 9 months (range 1-37 months). Epilepsy etiology was classified as genetic (23.7%), structural/metabolic (41%), and unknown cause (35.3%). Overall, rufinamide treatment led to a median seizure frequency reduction of 59.2% from responders to baseline. Seizure reduction was greater in patients with genetic etiology compared to structural/metabolic (66.2% vs. 45.5% responders, p = 0.005). Rufinamide was discontinued in 110 (36.7%) of 300 patients: 63 (21%) due to unsatisfactory response, 47 (15.7%) due to side effects, and in 18 (6%) of those due to both. Most common adverse effects were sleepiness, vomiting, mood changes, nausea, and loss of appetite. Median time to loss of efficacy was 11.6 months (range 3-28 months). Significance Rufinamide provides satisfactory seizure reduction as an adjunctive treatment in refractory epilepsy. Results need to be interpreted in the setting of data acquisition, including inherent biases of retrospective studies. Patients with a known genetic etiology may have better responses than patients with structural/metabolic etiology.

Original languageEnglish (US)
Pages (from-to)1235-1244
Number of pages10
Issue number8
StatePublished - Aug 2014


  • Epilepsy
  • Follow-up
  • Pediatric
  • Refractory epilepsy
  • Retention rate
  • Rufinamide

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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