title: Effectiveness and tolerability of perampanel in children and adolescents with refractory epilepsies-An Italian observational multicenter study.
abstract: To evaluate the efficacy and tolerability of Perampanel (PER) in children and adolescents with refractory epilepsies in daily clinical practice conditions.
  
  
  
  This Italian multicenter retrospective observational study was performed in 16 paediatric epilepsy centres. Inclusion criteria were: (i) ≤18 years of age, (ii) history of refractory epilepsy, (iii) a follow-up ≥5 months of PER add-on therapy. Exclusion criteria were: (i) a diagnosis of primary idiopathic generalized epilepsy, (ii) variation of concomitant AEDs during the previous 4 weeks. Response was defined as a ≥50% reduction in monthly seizure frequency compared with the baseline.
  
  
  
  62 patients suffering from various refractory epilepsies were included in this study: 53% were males, the mean age was 14.2 years (range 6-18 years), 8 patients aged <12 years. Mean age at epilepsy onset was 3.4 years and the mean duration of epilepsy was 10.8 years (range 1-16), which ranged from 2 seizures per-month up to several seizures per-day (mean number=96.5). Symptomatic focal epilepsy was reported in 62.9% of cases. Mean number of AEDs used in the past was 7.1; mean number of concomitant AEDs was 2.48, with carbamazepine used in 43.5% of patients. Mean PER daily dose was 7.1mg (2-12mg). After an average of 6.6 months of follow-up (5-13 months), the retention rate was 77.4% (48/62). The response rate was 50%; 16% of patients achieved ≥75% seizure frequency reduction and 5% became completely seizure free. Seizure aggravation was observed in 9.7% of patients. Adverse events were reported in 19 patients (30.6%) and led to PER discontinuation in 4 patients (6.5%). The most common adverse events were behaviour disturbance (irritability and aggressiveness), dizziness, sedation and fatigue.
  
  
  
  PER was found to be a safe and effective treatment when used as adjunctive therapy in paediatric patients with uncontrolled epilepsy.
authors: Department of Neurosciences, Neurology Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy; Department of Paediatrics, Child Neurology and Psychiatry, Sapienza University of Rome, Rome, Italy. Electronic address: Paola.deliso@opbg.net.
authors: P
authors: P
authors: De Liso
authors: Department of Neurosciences, Neurology Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
authors: F
authors: F
authors: Vigevano
authors: Department of Neurosciences, Neurology Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
authors: N
authors: N
authors: Specchio
authors: Department of Neurosciences, Neurology Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
authors: L
authors: L
authors: De Palma
authors: Epilepsy and Clinical Neurophysiology Unit, Scientific Institute, IRCCS Eugenio Medea, Conegliano, Treviso, Italy.
authors: P
authors: P
authors: Bonanni
authors: Epilepsy and Clinical Neurophysiology Unit, Scientific Institute, IRCCS Eugenio Medea, Conegliano, Treviso, Italy.
authors: E
authors: E
authors: Osanni
authors: Child and Adolescent Neuropsychiatry, Medical School, University of Salerno, Salerno, Italy.
authors: G
authors: G
authors: Coppola
authors: NESMOS Department, Faculty of Medicine and Psychology, Sant'Andrea Hospital, Sapienza University, Rome, Italy.
authors: P
authors: P
authors: Parisi
authors: Paediatric Neurology-Immunology and Endocrinology Unit, University of Siena, Siena, Italy.
authors: S
authors: S
authors: Grosso
authors: Department of Paediatrics, University of L'Aquila, L'Aquila, Italy.
authors: A
authors: A
authors: Verrotti
authors: Department of Paediatrics, Child Neurology and Psychiatry, Sapienza University of Rome, Rome, Italy.
authors: A
authors: A
authors: Spalice
authors: Department of Paediatrics, Child Neurology and Psychiatry, Sapienza University of Rome, Rome, Italy.
authors: F
authors: F
authors: Nicita
authors: Child Neuropsychiatry Unit, Polytechnic University of the Marche, Ancona, Italy.
authors: N
authors: N
authors: Zamponi
authors: Child Neuropsychiatry Unit, Polytechnic University of the Marche, Ancona, Italy.
authors: S
authors: S
authors: Siliquini
authors: Paediatric Neuropsychiatric Division, Spedali Civili, Brescia, Italy.
authors: L
authors: L
authors: Giordano
authors: Paediatric Neuropsychiatric Division, Spedali Civili, Brescia, Italy.
authors: P
authors: P
authors: Martelli
authors: Paediatric Neurology Unit, Children's Hospital A. Meyer, University of Firenze, Florence, Italy.
authors: R
authors: R
authors: Guerrini
authors: Paediatric Neurology Unit, Children's Hospital A. Meyer, University of Firenze, Florence, Italy.
authors: A
authors: A
authors: Rosati
authors: Paediatric Neurology Unit, Children's Hospital A. Meyer, University of Firenze, Florence, Italy.
authors: L
authors: L
authors: Ilvento
authors: Neurology Unit, Department of Neuroscience, Sant'Anna Hospital, Como, Italy.
authors: V
authors: V
authors: Belcastro
authors: Paediatric Neurology and Muscular Diseases Unit, Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, G. Gaslini Institute, Genoa, Italy.
authors: P
authors: P
authors: Striano
authors: Paediatric Neurology and Muscular Diseases Unit, Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, G. Gaslini Institute, Genoa, Italy.
authors: M S
authors: MS
authors: Vari
authors: Department of Child Neuropsychiatry, Epilepsy Centre, C. Poma Hospital, Mantova, Italy.
authors: G
authors: G
authors: Capovilla
authors: Department of Child Neuropsychiatry, Epilepsy Centre, C. Poma Hospital, Mantova, Italy.
authors: F
authors: F
authors: Beccaria
authors: Child Neuropsychiatry Unit, S. Andrea Hospital, Rome, Italy.
authors: O
authors: O
authors: Bruni
authors: Child Neuropsychiatry Unit, S. Andrea Hospital, Rome, Italy.
authors: A
authors: A
authors: Luchetti
authors: Child Neurology Unit, IRCCS Institute of Neurological Sciences, Bologna, Italy.
authors: G
authors: G
authors: Gobbi
authors: Child Neurology Unit, IRCCS Institute of Neurological Sciences, Bologna, Italy.
authors: A
authors: A
authors: Russo
authors: Epilepsy Unit, Child Neuropsychiatry Department, University Hospital, Cagliari, Italy.
authors: D
authors: D
authors: Pruna
authors: Multifactorial and Complex Diseases Research Area, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
authors: A E
authors: AE
authors: Tozzi
authors: Department of Neurosciences, Neurology Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
authors: R
authors: R
authors: Cusmai
mesh_terms: D000293:Adolescent; D000927:Anticonvulsants / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D000069279:Drug Resistant Epilepsy / Q000188:drug therapy*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007558:Italy; D008297:Male; D009570:Nitriles; D011728:Pyridones / Q000009:adverse effects / Q000627:therapeutic use*; D012189:Retrospective Studies; D012640:Seizures / Q000188:drug therapy; D016896:Treatment Outcome
chemical_list: D000927:Anticonvulsants; D009570:Nitriles; D011728:Pyridones; C551441:perampanel
keywords: Adolescents; Antiepileptic drug; Children; Perampanel; Refractory epilepsy; Treatment outcome