Short-term efficacy and tolerability of rufinamide adjunctive therapy in children with refractory generalised

Se Hee Kim1, Jeong Ho Lee, Hye Won Ryu

  • 1Department of Pediatrics, Pediatric Clinical Neuroscience Center, Seoul National University Children's Hospital, South Korea.

Insights

Rufinamide adjunctive therapy showed efficacy in children with refractory generalised epilepsies, particularly Lennox-Gastaut syndrome. However, response rates varied among epilepsy types, with Dravet syndrome and myoclonic seizures showing less improvement.

Area of Science:

  • Pediatric Neurology
  • Clinical Pharmacology
  • Epileptology

Background:

  • Refractory generalised epilepsy in children presents significant treatment challenges.
  • Adjunctive therapies are crucial for managing difficult-to-treat epilepsy syndromes.
  • Lennox-Gastaut syndrome and Dravet syndrome are severe forms of childhood epilepsy.

Purpose of the Study:

  • To evaluate the efficacy and tolerability of rufinamide as an adjunctive therapy.
  • To assess treatment response in children with various refractory generalised epilepsy types.
  • To identify patient subgroups that may benefit most or least from rufinamide.

Main Methods:

  • A cohort study involving 53 children with refractory generalised epilepsy.
  • Rufinamide was administered as adjunctive therapy.
  • Seizure reduction of over 50% at three and six months defined treatment responders.

Main Results:

  • Overall response rates were 37.7% at three months and 34.0% at six months.
  • Patients with Lennox-Gastaut syndrome and epilepsy with spasms/tonic seizures demonstrated higher response rates.
  • Subgroups with Dravet syndrome and epilepsy with myoclonic seizures showed lower responsiveness to rufinamide.

Conclusions:

  • Rufinamide is an effective and well-tolerated adjunctive treatment for many children with refractory generalised epilepsies.
  • Treatment outcomes vary significantly based on the specific epilepsy syndrome.
  • Further research is needed to optimize rufinamide use in specific pediatric epilepsy populations.

Related Concept Videos

Antiepileptic Drugs: Potassium Channel Activators01:20

Antiepileptic Drugs: Potassium Channel Activators

Ezocgabine or retigabine, an antiepileptic drug of remarkable efficacy, has revolutionized the management of seizures. It is a potassium channel activator, explicitly targeting the family of Q subtype potassium channels. It enhances the transmembrane potassium currents, regulating neuronal excitability. This action stabilizes the resting membrane potential, a pivotal factor in mitigating the hyperexcitability that characterizes epilepsy.
Ezogabine has gained approval as an adjunctive treatment...
Antiepileptic Drugs: Modulators of Neurotransmitter Release Mediated by SV2A Protein01:20

Antiepileptic Drugs: Modulators of Neurotransmitter Release Mediated by SV2A Protein

Antiepileptic drugs, such as levetiracetam (Keppra) and brivaracetam (Briviact), have emerged as crucial tools in managing epilepsy. These medications exert their therapeutic effects by targeting the synaptic vesicle protein SV2A, a transmembrane glycoprotein primarily found in the brain.
SV2A is a transmembrane glycoprotein located predominantly in the brain, modulating the release of neurotransmitters for neuronal communication. Both levetiracetam and brivaracetam exhibit a high affinity for...
Antiepileptic Drugs: Glutamate Antagonists01:14

Antiepileptic Drugs: Glutamate Antagonists

Glutamate is a fundamental neurotransmitter in the central nervous system, playing a vital role in neuronal communication and various cognitive processes. Glutamate stands as the principal excitatory neurotransmitter in the brain. Its presence is crucial for the communication between neurons, underpinning essential processes such as synaptic transmission, neuronal excitability, and plasticity. These functions are vital for higher-order cognitive processes, including learning and memory. The...
Epilepsy and Seizures: Overview01:24

Epilepsy and Seizures: Overview

Epilepsy is a chronic neurological disease marked by recurrent, unpredictable seizures. These seizures are caused by abnormal electrical discharges in the brain, leading to behavior, sensation, or consciousness alterations. They can also cause transient impairment of awareness, interfering with daily activities.
Various factors can trigger epilepsy, including genetic factors, brain damage, metabolic causes, and unknown etiology. Diagnosis of epilepsy involves electroencephalography (EEG), which...
Pharmacokinetics in Pediatric Patients: Drug Excretion01:26

Pharmacokinetics in Pediatric Patients: Drug Excretion

In pediatric medicine, understanding the renal function and drug elimination nuances is crucial for administering safe and effective treatments. Newborns, in particular, display markedly slower renal functions than adults, profoundly affecting how drugs are cleared from their bodies. This slower drug clearance requires clinicians to extend the dosing intervals for many medications to prevent drug accumulation and toxicity while ensuring therapeutic efficacy.One key area where these adjustments...
Antiepileptic Drugs: GABAergic Pathway Potentiators01:18

Antiepileptic Drugs: GABAergic Pathway Potentiators

γ-aminobutyric acid or GABA, plays a pivotal role as an inhibitory neurotransmitter in the brain. GABA pathway potentiators, also known as GABAergic drugs, are a class of pharmaceutical agents designed to enhance the functioning of the GABAergic system. These medications primarily treat epilepsy, a neurological disorder characterized by recurrent seizures.
The key GABA pathway potentiators used in epilepsy management are as follows.
Benzodiazepines are a well-known class of drugs used for their...