Efficacy of levetiracetam in the treatment of drug-resistant Rett syndrome

Nicola Specchio, Martina Balestri, Pasquale Striano, Maria Roberta Cilio, Rosaria Nardello, Santina Patanè, Maria Luisa Margiotta, Giuseppe D'Orsi, Salvatore Striano, Silvia Russo, Luigi Maria Specchio, Raffaella Cusmai, Lucia Fusco, Federico Vigevano

Research output: Contribution to journalArticlepeer-review


Rett syndrome (RTT) is a progressive neurological disorder characterized by a wide spectrum of phenotypes. Epilepsy is reported to occur in 50-90% of patients with RTT; some develop medically refractory epilepsy. The aim of this study is to investigate the efficacy of levetiracetam (LEV) in drug-resistant patients with RTT. This prospective, pragmatic, open-label study consisted of an 8-week baseline period and a 6-month evaluation period. Efficacy variable was the mean frequency of monthly seizures before, and after 3 and 6 months of treatment with LEV. Eight female patients, aged 7.5-19 years (M12.8 ± 5) entered the study. Mean age at epilepsy onset was 25.8 ± 14.1 months. All patients showed MeCP2 mutation. Patients had been treated with a mean of 3.4 AEDs (2-7) before LEV. The mean LEV dose was 44.84 ± 18.02 mg/kg/day. The mean monthly seizure frequency for all types of seizures during the baseline period was 21.3 ± 8.1 (range 10-35); after 3 months it was 3.3 ± 4.1 (range 0-9) and after 6 months of LEV treatment it was 1.5 ± 2 (range 0-4), p <0.0001. The mean follow-up period was 20.2 ± 13 months. Mild sleepiness occurred in two patients, one reported intermittent agitation. Levetiracetam appeared effective in our series of drug-resistant RTT patients. All reported a reduction in seizure frequency and consequently a better quality of life.

Original languageEnglish
Pages (from-to)112-117
Number of pages6
JournalEpilepsy Research
Issue number2-3
Publication statusPublished - Feb 2010


  • Drug-resistance
  • EEG
  • Focal seizures
  • Levetiracetam
  • Myoclonic seizures
  • Rett syndrome

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology


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