Effectiveness and tolerability of perampanel in children and adolescents with refractory epilepsies-An Italian
P De Liso1, F Vigevano2, N Specchio2
1Department 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.
Insights
Perampanel (PER) is effective and well-tolerated for children and adolescents with refractory epilepsy. This adjunctive therapy showed a 50% response rate, with manageable adverse events in clinical practice.
Area of Science:
- Pediatric Neurology
- Epileptology
- Clinical Pharmacology
Background:
- Refractory epilepsies in children and adolescents pose significant treatment challenges.
- Limited data exists on the real-world efficacy and tolerability of newer antiepileptic drugs in this population.
- Perampanel (PER) is an adjunctive therapy approved for epilepsy treatment.
Purpose of the Study:
- To evaluate the efficacy and tolerability of Perampanel (PER) as an add-on therapy.
- To assess PER's effectiveness in pediatric patients with refractory epilepsies under routine clinical conditions.
Main Methods:
- A multicenter, retrospective observational study involving 16 Italian pediatric epilepsy centers.
- Inclusion criteria: patients ≤18 years with refractory epilepsy, ≥5 months PER add-on therapy.
- Response defined as ≥50% reduction in monthly seizure frequency; adverse events and retention rates were recorded.
Main Results:
- 62 patients (mean age 14.2 years) with refractory epilepsies were analyzed.
- A 50% response rate was observed, with 16% achieving ≥75% seizure reduction and 5% becoming seizure-free.
- Retention rate was 77.4%; common adverse events included behavioral disturbances, dizziness, sedation, and fatigue, leading to discontinuation in 6.5%.
Conclusions:
- Perampanel (PER) demonstrated safety and efficacy as adjunctive therapy for pediatric patients with uncontrolled epilepsy.
- The findings support PER's utility in managing refractory epilepsies in children and adolescents in real-world settings.
Purpose:
To evaluate the efficacy and tolerability of Perampanel (PER) in children and adolescents with refractory epilepsies in daily clinical practice conditions.
Patients And Methods:
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.
Results:
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.
Conclusion:
PER was found to be a safe and effective treatment when used as adjunctive therapy in paediatric patients with uncontrolled epilepsy.
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