Dravet Patients Responded Well to Treatment with Perampanel as Add-On Therapy

Iqra Mumal, MSc avatar

by Iqra Mumal, MSc |

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Perampanel, as an add-on therapy, efficiently reduced seizure rates in children with Dravet syndrome, a real-world study shows.

The study, “Efficacy and tolerability of perampanel in children and adolescents with pharmacoresistant epilepsy: The first real-world evaluation in Asian pediatric neurology clinics,” was published in the journal Epilepsy & Behaviour.

Children with epilepsy who do not respond to pharmacological therapy are at significantly higher risk of developing cognitive issues, reduced quality of life, and behavioral and mental health problems. That makes it crucial to control seizures early on in childhood.

Seizures are caused by disorganized and sudden electrical activity in the brain, generally by the over-activation of particular receptors that reside on the surface on nerve cells. In particular, over-activation of the α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptor is known to be highly toxic and linked to epileptic seizures.

Perampanel (brand name Fycompa) is a novel anti-epileptic therapeutic that antagonizes the activity of the AMPA receptor. Perampanel (PER) has been widely used to treat seizures in patients with epilepsy who are 12  years or older.

Some studies have suggested that the therapy is both effective and safe in pediatric patients. However, there is a lack of clinical data demonstrating tolerability and effectiveness of PER in real-world clinical settings, particularly in Asian children.

As race and ethnicity are known to affect how individuals react to drugs, it is important to determine their safety and effectiveness among patients of all ethnicities.

Taiwanese researchers investigated the effectiveness and safety of PER as an add-on therapy in 66 Asian epileptic patients who were 18 years or younger and who were resistant to more than two antiepileptic therapies. Among these, there were five patients with Dravet syndrome.

Patients were classified as responsive if they had a seizure reduction greater than 50% compared to baseline levels. Participants were followed for up to 12 months.

At 3, 6 and 12 months,  30.3%, 37.5%, and 34.7% of patients were considered responsive and 7.6%, 8.9%, and 14.3% became completely seizure-free, respectively.

Among the five Dravet patients, four exhibited a 50% reduction in seizures since the last visit, at which point two patients were seizure-free.

“Notably, our results indicated that PER was conspicuously efficacious in patients with Dravet syndrome, achieving clinically meaningful improvement in four of five patients and even seizure freedom in two patients,” the authors wrote.

Considering that standard antiepileptic drugs — such as valproate and clobazam — rarely provide satisfactory seizure control in patients with Dravet syndrome, the results hold promise for PER as an add-on therapy in this patient population.

Most of the adverse events reported were not considered serious. These occurred in 35.7% of patients and included irritability, skin rash, dizziness, and somnolence (sleepiness or drowsiness). Twelve percent of the participants discontinued use of PER due to adverse side effects.

“The current data support the value of adjunctive PER in child and adolescent patients with pharmacoresistant epilepsy in daily clinical practice. Perampanel was efficacious and generally well-tolerated as an add-on treatment for epilepsy,” the authors concluded.