Efficacy and tolerability of perampanel as add-on therapy in Dravet syndrome: A prospective real-world study.

OBJECTIVE

To investigate the efficacy and tolerability of perampanel (PER) on Dravet syndrome in China by a prospective real-world study.

METHODS

We prospectively enrolled children with Dravet syndrome from the neurology clinic of Shenzhen Children's Hospital from September 2020 to October 2021. The average monthly seizure frequency within 2 months before enrollment was recorded as the baseline. PER was administered after enrollment. The response rate (the rate of seizure reduction ≥50% compared to the baseline) was regarded as the major index. The adverse events and retention rates were regarded as the minor index. All indexes were calculated after 3 and 6 months' follow-up, respectively. The outcome-predictive value of various clinical factors was analyzed.

RESULTS

A total of 21 children were enrolled, including 15 boys and 6 girls, with a median age of 4 years and 8 months (1 year and 3 months to 15 years and 6 months). Averagely, 2.57 ASMs had been administrated and failed to control seizures before enrollment. The response rates were 52.4% (11/21) and 47.6% (10/21) after 3 and 6 months of PER administration, respectively. Adverse events were observed in 8 patients (38.1%), including irritability (3/8, 37.5%), fatigue (3/8, 37.5%), unstable walking (2/8, 25%), somnolence (2/8, 25%), and sluggish response (2/8, 25%); most of them were mild and temporary. The retention rate was 90.5% (19/21) at 6 months' follow-up. Two patients discontinued PER due to failure of seizure control, and no patients discontinued due to adverse events.

SIGNIFICANCE

This study provides a comprehensive analysis of PER's effectiveness and tolerability in treating DS, highlighting its importance as an additional treatment option. It broadens the range of anti-seizure medications (ASMs) that may be effective for patients with DS.

PLAIN LANGUAGE SUMMARY

In the neurology clinic of Shenzhen Children's Hospital, 21 children with Dravet syndrome were administered perampanel. Perampanel, as a novel anti-seizure medication approved for focal and generalized epilepsies as add-on therapy, showed sufficient efficacy and a satisfactory safety and tolerability profile in this study.