@article{fff7db1265704121ba5262488839c310,
title = "Medical treatment in infants and young children with epilepsy: Off-label use of antiseizure medications. Survey Report of ILAE Task Force Medical Therapies in Children",
abstract = "Objective: Antiseizure medications (ASMs) remain the mainstay of epilepsy treatment. These ASMs have mainly been tested in trials in adults with epilepsy, which subsequently led to market authorization (MA). For treatment of – especially young – children with epilepsy, several ASMs do not have a MA and guidelines are lacking, subsequently leading to “off-label” use of ASMs. Even though “off-label” ASM prescriptions for children could lead to more adverse events, it can be clinically appropriate and rational if the benefits outweigh the risks. This could be the case if “on-label” ASM, in mono- or polytherapy, fails to achieve adequate seizure control. Methods: The Medical Therapies Task Force of the International League Against Epilepsy (ILAE) Commission for Pediatrics performed a survey to study the current treatment practices in six classic, early life epilepsy scenarios. Our aim was not only to study first- and second-line treatment preferences but also to illustrate the use of “off-label” drugs in childhood epilepsies. Results: Our results reveal that several ASMs (e.g. topiramate, oxcarbazepine, benzodiazepines) are prescribed “off-label” in distinct scenarios of young children with epilepsy. In addition, recent scientific guidelines were not always adopted by several survey respondents, suggesting a potential knowledge gap. Significance: We report the relatively common use of “off-label” prescriptions that underlines the need for targeted and appropriately designed clinical trials, including younger patients, which will also result in the ability to generate evidence-based guidelines.",
keywords = "International League Against Epilepsy, children, epilepsy treatment, off-label, questionnaire",
author = "Jo Sourbron and St{\'e}phane Auvin and Alexis Arzimanoglou and Cross, {J. Helen} and Hans Hartmann and Ronit Pressler and Kate Riney and Kenji Sugai and Wilmshurst, {Jo M.} and Elissa Yozawitz and Lieven Lagae",
note = "Funding Information: JS has no conflict of interest to disclose. SA received honoraria or consultancy fees from Arvelle, Biomarin, Eisai, GW Pharma, Neuraxpharm, Nutricia, UCB Pharma, Xenon, Zogenix. SA is an associate editor for Epilepsia. AA, in agreement with his Institution, has served as principal investigator or member of DMCs. in clinical trials for Eisai, UCB, GW Pharma; received consulting fees from Jazz, Zogenix, Eisai, Takeda, Biocodex, Encoded Therapeutics and Neuraxpharm; unrestricted research grants from, UCB, Caixa Foundation and Jazz and academic research grants from EJP‐RD and the EU. AA is Editor‐in‐Chief Emeritus for Epileptic Disorders (JLE editions) and Associate Editor for the European Journal of Pediatric Neurology (Elsevier). HC has acted as an investigator for studies with GW Pharma, Zogenix, Vitaflo, Stoke. Therapeutics and Marinus. She has been a speaker and on advisory boards for GW Pharma, Zogenix, UCB and Nutricia; all remuneration has been paid to her department. She holds as endowed chair at UCL Great Ormond Street Institute of Child Health; she holds grants from NIHR, EPSRC, GOSH Charity, ERUK, the Waterloo Foundation and the National Institute of Health Research (NIHR) Biomedical Research Centre at Great Ormond Street Hospital. HH has no interests to declare. RP is an investigator for studies with UCB and does consultancy work for Kephala, Ireland. She served as a speaker and/or on Advisory Boards for Natus, GW, Eisai, and UCB. Her research is supported by the National Institute of Health Research (NIHR) Biomedical Research Centre at Great Ormond Street Hospital, Cambridge Biomedical Research Centre, the NIHR and the Evelyn Trust. KR has received honoraria for educational symposia, advisory boards and/or consultancy work from Eisai, LivaNova, Medlink Neurology, Novartis and UCB Australia Ltd. Her institution has supported clinical trials for Biogen Idec Research Ltd., DSLP, Eisai Inc., Epigenyx Therapeutics Inc., GW Research Ltd, Janssen‐Cilag, Marinus Pharmaceuticals Inc., Medicure International Inc., LivaNova, Neurocrine Biosciences Inc., Noema Pharma, Novartis, SK Lifesciences Inc., UCB Australia Ltd., UCB Biopharma SRL and Zogenix Inc. KS received honoraria from Eisai. JW is a member of the South African Sanofi advisory board and is an associate editor for Epilepsia. EY has no conflict of interest to disclose. LL received grants, and is a consultant and/or speaker for Zogenix; LivaNova, UCB, Shire, Eisai, Novartis, Takeda/Ovid, NEL, Epihunter. This research did not receive any specific grant from funding agencies in the public, commercial, or not‐for‐profit sectors. We confirm that we have read the Journal's position on issues involved in ethical publication and affirm that this report is consistent with those guidelines. Publisher Copyright: {\textcopyright} 2022 The Authors. Epilepsia Open published by Wiley Periodicals LLC on behalf of International League Against Epilepsy.",
year = "2023",
month = mar,
doi = "10.1002/epi4.12666",
language = "English (US)",
volume = "8",
pages = "77--89",
journal = "Epilepsia Open",
issn = "2470-9239",
publisher = "Wiley-Blackwell Publishing Ltd",
number = "1",
}