Perampanel for the treatment of epilepsy with genetic aetiology: Real-world evidence from the PERMIT Extension study

Delanty, Norman; Mohanraj, Rajiv; Shankar, Rohit; Wehner, Tim; Stephen, Linda J.; D’Souza, Wendyl; Cappucci, Sheri; McMurray, Rob; Sainz-Fuertes, Ricardo; Villanueva, Vicente

doi:10.1016/j.eplepsyres.2024.107339

Cited by 2 publications

(2 citation statements)

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“…As shown previously [ 20 , 26 – 29 ], PER was particularly effective in individuals with a presumed genetic aetiology, with almost 50% of this subgroup achieving seizure freedom (total seizures) after 12 months of PER treatment. Among the aetiology subtypes, this subgroup experienced the highest seizure freedom and responder rates.…”

Section: Discussionsupporting

confidence: 73%

“…These findings are consistent with those of a multicentre project based on the framework of the Network for Therapy in Rare Epilepsies (NETRE), conducted in 137 individuals with 79 different aetiologies, which demonstrated that PER was efficacious and well tolerated in individuals with a range of rare genetic epilepsies (including mutations in SCN1A , GNAO1 , PIGA , PCDH19 , SYNGAP1 , CDKL5 , NEU1 , and POLG ), indicating that PER may have a targeted effect related to glutamate transmission [ 30 ]. Unlike the other aetiology subgroups, the majority of individuals in the genetic aetiology subgroup had only generalised seizures, and, as previously reported [ 26 ], the majority of these individuals had IGEs, which are thought to have a more favourable prognosis than other types of epilepsy [ 31 ]. Further research is therefore required to determine the effects of PER in individuals with other types of genetic epilepsies, which may be associated with more severe seizure outcomes.…”

Section: Discussionmentioning

confidence: 71%

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Perampanel for Treatment of People with a Range of Epilepsy Aetiologies in Clinical Practice: Evidence from the PERMIT Extension Study

Strzelczyk,

Maschio,

Pensel

et al. 2024

Neurol Ther

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Introduction It is important to assess the effectiveness of an antiseizure medication in treating different epilepsy aetiologies to optimise individualised therapeutic approaches. Data from the PERaMpanel pooled analysIs of effecTiveness and tolerability (PERMIT) Extension study were used to assess the effectiveness and safety/tolerability of perampanel (PER) when used to treat individuals with a range of epilepsy aetiologies in clinical practice. Methods A post hoc analysis was conducted of PERMIT Extension data from individuals with a known aetiology. Retention was assessed after 3, 6 and 12 months. Effectiveness was assessed after 3, 6 and 12 months and at the last visit (last observation carried forward). Effectiveness assessments included responder rate (≥ 50% seizure frequency reduction) and seizure freedom rate (no seizures since at least the prior visit). Safety/tolerability was assessed by evaluating adverse events (AEs) and AEs leading to discontinuation. Results PERMIT Extension included 1945 individuals with structural aetiology, 1012 with genetic aetiology, 93 with an infectious aetiology, and 26 with an immune aetiology. Retention rates at 12 months were 61.1% (structural), 65.9% (genetic), 56.8% (infectious) and 56.5% (immune). At the last visit, responder rates (total seizures) were 43.3% (structural), 68.3% (genetic), 37.0% (infectious) and 20.0% (immune), and corresponding seizure freedom rates were 15.8%, 46.5%, 11.1% and 5.0%, respectively. AE incidence rates were 58.0% (structural), 46.5% (genetic), 51.1% (infectious) and 65.0% (immune), and corresponding rates of discontinuation due to AEs over 12 months were 18.9%, 16.4%, 18.5% and 21.7%, respectively. The types of AEs reported were generally consistent across aetiology subgroups, with no idiosyncratic AEs emerging. Conclusion Although PER was effective and generally well tolerated when used to treat individuals with a range of epilepsy aetiologies in clinical practice, variability in its effectiveness and tolerability across the subgroups indicates that PER may be particularly useful for individuals with specific epilepsy aetiologies. Supplementary Information The online version contains supplementary material available at 10.1007/s40120-024-00618-5.

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Section: Discussionsupporting

confidence: 73%