PMID- 38492461
OWN - NLM
STAT- Publisher
LR  - 20240316
IS  - 1872-6844 (Electronic)
IS  - 0920-1211 (Linking)
VI  - 202
DP  - 2024 Mar 2
TI  - Perampanel for the treatment of epilepsy with genetic aetiology: Real-world 
      evidence from the PERMIT Extension study.
PG  - 107339
LID - S0920-1211(24)00054-8 [pii]
LID - 10.1016/j.eplepsyres.2024.107339 [doi]
AB  - Genetic factors contribute to the aetiology of epilepsy in >50% of cases, and 
      information on the use of antiseizure medications in people with specific 
      aetiologies will help guide treatment decisions. The PERMIT Extension study 
      pooled data from two real-world studies (PERMIT and PROVE) to investigate the 
      effectiveness and safety/tolerability of perampanel (PER) when used to treat 
      people with focal and generalised epilepsy in everyday clinical practice. This 
      post-hoc analysis of PERMIT Extension explored the use of PER when used to treat 
      individuals presumed to have epilepsy with a genetic aetiology. Assessments 
      included retention rate (evaluated at 3, 6 and 12 months), effectiveness 
      (responder and seizure freedom rates; evaluated at 3, 6, 12 months and the last 
      visit [last observation carried forward) and tolerability (adverse events [AEs]). 
      Of the 6822 people with epilepsy included in PERMIT Extension, 1012 were presumed 
      to have a genetic aetiology. The most common genetic aetiologies were idiopathic 
      generalised epilepsy (IGE; 58.2%), tuberous sclerosis (1.1%), Dravet syndrome 
      (0.8%) and genetic epilepsy with febrile seizures plus (GEFS+; 0.5%). Retention 
      rates at 3, 6 and 12 months in the total genetic aetiology population were 89.3%, 
      79.7% and 65.9%, respectively. In the total genetic aetiology population, 
      responder rates at 12 months and the last visit were 74.8% and 68.3%, 
      respectively, and corresponding seizure freedom rates were 48.9% and 46.5%, 
      respectively. For the specific aetiology subgroups, responder rates at 12 months 
      and the last visit were, respectively: 90.4% and 84.4% (IGE), 100% and 57.1% 
      (tuberous sclerosis), 100% and 71.4% (Dravet syndrome), and 33.3% and 20.0% 
      (GEFS+). Corresponding seizure freedom rates were, respectively: 73.1% and 64.6% 
      (IGE), 33.3% and 22.2% (tuberous sclerosis), 20.0% and 28.6% (Dravet syndrome), 
      and 0% and 0% (GEFS+). The incidence of AEs was 46.5% for the total genetic 
      aetiology population, 48.8% for IGE, 27.3% for tuberous sclerosis, 62.5% for 
      Dravet syndrome, and 20% for GEFS+. Tolerability findings were consistent with 
      PER's known safety profile. PER was effective and generally well tolerated when 
      used in individuals with a presumed genetic epilepsy aetiology in clinical 
      practice. PER was effective across a wide range of genetic aetiologies.
CI  - Copyright (c) 2024 The Authors. Published by Elsevier B.V. All rights reserved.
FAU - Delanty, Norman
AU  - Delanty N
AD  - Department of Neurology, Beaumont Hospital, Dublin, Ireland. Electronic address: 
      normandelanty@beaumont.ie.
FAU - Mohanraj, Rajiv
AU  - Mohanraj R
AD  - Greater Manchester Neurosciences Centre, Salford Royal Hospital, UK.
FAU - Shankar, Rohit
AU  - Shankar R
AD  - Peninsula School of Medicine, Plymouth, UK.
FAU - Wehner, Tim
AU  - Wehner T
AD  - National Hospital for Neurology and Neurosurgery, UCLH Foundation Trust, and 
      Department of Clinical and Experimental Epilepsy, UCL, London, UK.
FAU - Stephen, Linda J
AU  - Stephen LJ
AD  - Epilepsy Unit, West Glasgow Ambulatory Care Hospital, Glasgow, Scotland, UK.
FAU - D'Souza, Wendyl
AU  - D'Souza W
AD  - Department of Medicine, St Vincent's Hospital Melbourne, The University of 
      Melbourne, Victoria, Australia.
FAU - Cappucci, Sheri
AU  - Cappucci S
AD  - Eisai Inc, Nutley, NJ, USA.
FAU - McMurray, Rob
AU  - McMurray R
AD  - Eisai Europe Ltd, UK.
FAU - Sainz-Fuertes, Ricardo
AU  - Sainz-Fuertes R
AD  - Eisai Europe Ltd, UK.
FAU - Villanueva, Vicente
AU  - Villanueva V
AD  - Refractory Epilepsy Unit, Hospital Universitario y Politecnico La Fe, Valencia, 
      Spain.
LA  - eng
PT  - Journal Article
DEP - 20240302
PL  - Netherlands
TA  - Epilepsy Res
JT  - Epilepsy research
JID - 8703089
SB  - IM
OTO - NOTNLM
OT  - Epilepsy
OT  - Epilepsy syndrome
OT  - Genetic etiology
OT  - Idiopathic generalized epilepsy
OT  - Perampanel
OT  - Real-world
EDAT- 2024/03/17 00:42
MHDA- 2024/03/17 00:42
CRDT- 2024/03/16 19:09
PHST- 2023/12/08 00:00 [received]
PHST- 2024/02/14 00:00 [revised]
PHST- 2024/02/29 00:00 [accepted]
PHST- 2024/03/17 00:42 [medline]
PHST- 2024/03/17 00:42 [pubmed]
PHST- 2024/03/16 19:09 [entrez]
AID - S0920-1211(24)00054-8 [pii]
AID - 10.1016/j.eplepsyres.2024.107339 [doi]
PST - aheadofprint
SO  - Epilepsy Res. 2024 Mar 2;202:107339. doi: 10.1016/j.eplepsyres.2024.107339.