First long-term experience with the orphan drug rufinamide in children with myoclonic-astatic epilepsy (Doose syndrome)

详细信息    查看全文

• 作者：C. von St¨¹lpnagel ; G. Coppola ; P. Striano ; A. M¨¹ller ; M. Staudt ; G. Kluger
• 关键词：Myoclonic-astatic epilepsy ; Doose syndrome ; Children ; Long-term experience ; Rufinamide
• 刊名：European Journal of Paediatric Neurology
• 出版年：2012
• 出版时间：September, 2012
• 年：2012
• 卷：16
• 期：5
• 页码：459-463
• 全文大小：133 K

文摘

Introduction

We evaluated the long-term efficacy and tolerability of the orphan drug rufinamide (RUF) in children with pharmacoresistant myoclonic-astatic epilepsy (MAE, Doose syndrome).

Methods

This was a retrospective European multicenter study on eight patients who had started an intention-to-treat trial of RUF between July 2007 and June 2010. Clinical information was collected via questionnaire. Responder rate was defined as reduction of seizure frequency ¡Ý50 % in comparison to four weeks before starting RUF. Maximum follow-up was eighteen months.

Results

Responder rates were 7/8 patients after 3 months, 6/8 patients after 6 months and 5/8 patients after 12 months. RUF seemed particularly effective in the prevention of myoclonic-astatic seizures (comparable with drop attacks in Lennox-Gastaut-Syndrome, for which RUF is particularly effective). Some loss of efficacy was noticed in the long-term observation. Side-effects occurred in two patients. Seizure aggravation was not observed.

Conclusion

RUF seems to be a promising therapeutic option in children with MAE. Further studies are warranted to confirm these first observations.