Treatment-Refractory Dravet Syndrome: Considerations for Novel Medications

J Pediatr Health Care. 2022 Sep-Oct;36(5):479-488. doi: 10.1016/j.pedhc.2022.05.003.

Abstract

Before 2018, there were no U.S. Food and Drug Administration-approved medications for managing seizures in Dravet syndrome (DS). Common agents used in the antiepileptic drug regimens of patients with DS included clobazam, valproic acid, topiramate, and levetiracetam, among others; however, these agents alone rarely provide adequate seizure control. Management of seizures in DS changed in recent years with the approval of cannabidiol and stiripentol in 2018 and fenfluramine in 2020. This continuing education article summarizes available efficacy and safety data involving cannabidiol, stiripentol, and fenfluramine and provides a practical review of dosing strategies, pharmacokinetics, and monitoring interventions relevant to their use.

Keywords: Dravet syndrome; drug therapy; epilepsy; pediatrics.

MeSH terms

  • Anticonvulsants / therapeutic use
  • Cannabidiol* / therapeutic use
  • Epilepsies, Myoclonic* / drug therapy
  • Epileptic Syndromes
  • Fenfluramine / therapeutic use
  • Humans
  • Seizures
  • Spasms, Infantile

Substances

  • Anticonvulsants
  • Cannabidiol
  • Fenfluramine

Supplementary concepts

  • CDKL5 deficiency disorder