Abstracts

Rationale: We report about the add-on therapy of the orphan-drug rufinamide (RUF), which is licensed for Lennox-Gastaut-Syndrome in six patients with difficult-to-treat Doose-Syndrome (= myoclonic-astatic epilepsy, MAE). Methods: Patients and Methods: Six Patients (4 male; age 4 to 20 years, mean age 6 2/12 years) with MAE were included in a retrospective evaluation of treatment with RUF. All patients had difficult-to-treat epilepsy- average anticonvulsive pre-treatment was nine anticonvulsive drugs (AED) (range 2- 14 AED; mean 7), together with mild to severe mental retardation (five mild; one severe). RUF was gradually introduced as add-on therapy. Responder were defined as a reduction of seizure frequency >50% in comparison to four weeks before starting the therapy with RUF and a lasting effect for at least three month (RS). Results: Responders were five of six patients (83.3%), one patient became seizure-free, four patients showed a 75 % reduction of seizure frequency. The best effect of RUF was noticed for drop-attacks, generalized tonic-clonic seizures and tonic seizures. After RUF treatment for six month we had four responders out of the six patients (66.6%); after 12 month four patients of five (80%) still showed a 75% reduction of seizure frequency and after 18 month one patient out of three (33%) still taking RUF reported a 80 % reduction of his main seizure types drop-attacks and myoclonic seizures. Side-effects (SE) occurred in 40 % and were mainly decreased appetite and sleepiness which got less after the patient had become accustomed to the medication; no aggravation of seizures was seen. Conclusions: In a small number of patients with refractory MAE we observed a high efficacy with RUF (an orphan drug for LGS). Some loss of efficacy was noticed in the long-term treatment. Further multicenter studies a warranted to confirm our first observations.