Lack of effectiveness and seizure worsening with cenobamate in pediatric patients with Dravet syndrome DOI

Rita Cagigal,

Celia Romero‐del‐Rincon,

Ana Laura Fernández‐Perrone

et al.

Epilepsia, Journal Year: 2025, Volume and Issue: unknown

Published: April 28, 2025

Abstract Dravet syndrome (DS) is a severe developmental and epileptic encephalopathy primarily caused by loss‐of‐function (LoF) variants in the SCN1A gene. Despite advancements precision medicine, seizure control remains challenging. Cenobamate, novel antiseizure medication, has shown positive results small case series of adults with DS, but its efficacy children unclear. This retrospective single‐center study analyzed therapeutic response to cenobamate six pediatric patients (median age = 8.5 years) DS LoF variants. Patients were treated for median follow‐up 7 months. Baseline frequency was assessed over 3 months prior treatment initiation. Response defined as ≥50% reduction worsening ≥25% increase compared baseline. No patient met responder criteria. Three experienced worsening, including one who started presenting status epilepticus after long epilepticus‐free period, remaining three showed no improvement. Adverse effects included sleepiness, restlessness, loss appetite, discontinued all due lack effectiveness or worsening. Our findings suggest that lacks may exacerbate seizures some cases. These highlight need age‐specific approaches decisions genetic epilepsies.

Language: Английский

Lack of effectiveness and seizure worsening with cenobamate in pediatric patients with Dravet syndrome DOI

Rita Cagigal,

Celia Romero‐del‐Rincon,

Ana Laura Fernández‐Perrone

et al.

Epilepsia, Journal Year: 2025, Volume and Issue: unknown

Published: April 28, 2025

Abstract Dravet syndrome (DS) is a severe developmental and epileptic encephalopathy primarily caused by loss‐of‐function (LoF) variants in the SCN1A gene. Despite advancements precision medicine, seizure control remains challenging. Cenobamate, novel antiseizure medication, has shown positive results small case series of adults with DS, but its efficacy children unclear. This retrospective single‐center study analyzed therapeutic response to cenobamate six pediatric patients (median age = 8.5 years) DS LoF variants. Patients were treated for median follow‐up 7 months. Baseline frequency was assessed over 3 months prior treatment initiation. Response defined as ≥50% reduction worsening ≥25% increase compared baseline. No patient met responder criteria. Three experienced worsening, including one who started presenting status epilepticus after long epilepticus‐free period, remaining three showed no improvement. Adverse effects included sleepiness, restlessness, loss appetite, discontinued all due lack effectiveness or worsening. Our findings suggest that lacks may exacerbate seizures some cases. These highlight need age‐specific approaches decisions genetic epilepsies.

Language: Английский

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