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

Jo Sourbron, Stéphane Auvin, Alexis Arzimanoglou, J. Helen Cross, Hans Hartmann, Ronit Pressler, Kate Riney, Kenji Sugai, Jo M. Wilmshurst, Elissa Yozawitz, Lieven Lagae

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish (US)
JournalEpilepsia Open
DOIs
StateAccepted/In press - 2022
Externally publishedYes

Keywords

  • International League Against Epilepsy
  • children
  • epilepsy treatment
  • off-label
  • questionnaire

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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