title: Efficacy of levetiracetam in the treatment of drug-resistant Rett syndrome.
abstract: 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+/-18.02mg/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.
authors.affiliation: Division of Neurology, Bambino Gesù Children's Hospital, IRCCS, P.zza S. Onofrio, Rome, Italy. nicola.specchio@opbg.net
authors.forename: Nicola
authors.initials: N
authors.lastname: Specchio
authors.forename: Martina
authors.initials: M
authors.lastname: Balestri
authors.forename: Pasquale
authors.initials: P
authors.lastname: Striano
authors.forename: Maria Roberta
authors.initials: MR
authors.lastname: Cilio
authors.forename: Rosaria
authors.initials: R
authors.lastname: Nardello
authors.forename: Santina
authors.initials: S
authors.lastname: Patanè
authors.forename: Maria Luisa
authors.initials: ML
authors.lastname: Margiotta
authors.forename: Giuseppe
authors.initials: G
authors.lastname: D'Orsi
authors.forename: Salvatore
authors.initials: S
authors.lastname: Striano
authors.forename: Silvia
authors.initials: S
authors.lastname: Russo
authors.forename: Luigi Maria
authors.initials: LM
authors.lastname: Specchio
authors.forename: Raffaella
authors.initials: R
authors.lastname: Cusmai
authors.forename: Lucia
authors.initials: L
authors.lastname: Fusco
authors.forename: Federico
authors.initials: F
authors.lastname: Vigevano
mesh_terms: D000293:Adolescent; D000704:Analysis of Variance; D000927:Anticonvulsants / Q000627:therapeutic use; D002648:Child; D004334:Drug Administration Schedule; D004569:Electroencephalography; D005260:Female; D006801:Humans; D000077287:Levetiracetam; D051783:Methyl-CpG-Binding Protein 2 / Q000235:genetics; D018579:Patient Selection; D010889:Piracetam / Q000031:analogs & derivatives* / Q000627:therapeutic use; D011446:Prospective Studies; D011788:Quality of Life; D015518:Rett Syndrome / Q000188:drug therapy* / Q000235:genetics; D012640:Seizures / Q000188:drug therapy* / Q000235:genetics; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000927:Anticonvulsants; C497392:MECP2 protein, human; D051783:Methyl-CpG-Binding Protein 2; D000077287:Levetiracetam; D010889:Piracetam