Efficacy and tolerance of cannabidiol in the treatment of epilepsy in patients with Rett syndrome

Epilepsia Open. 2024 Feb;9(1):397-403. doi: 10.1002/epi4.12796. Epub 2023 Nov 15.

Abstract

We aim to assess the efficacy and tolerance of cannabidiol as adjunctive therapy for Rett syndrome (RTT) patients with epilepsy. We conducted a longitudinal observational study through a monocentric cohort of 46 patients with RTT. Patients were recruited from March 2020 to October 2022 and were treated with Epidyolex® (cannabidiol, CBD, 100 mg/mL oral solution). In our cohort, 26 patients had associated epilepsy (26/46 [56%]), and 10/26 (38%) were treated with CBD, in combination with clobazam in 50% of cases. The median dose at their last follow-up was 15 mg/kg/day. The median treatment duration was 13 months (range: 1-32 months). CBD reduced the incidence of seizures in seven out of 10 patients (70%) with one seizure-free patient, two patients with a reduction of seizures of more than 75%, and four patients with a decrease of more than 50%. No aggravation of symptoms or adverse effects were observed. Only one patient experienced a transitory drooling and somnolence episode at the CBD initiation. Half of the patients showed a reduction in agitation and/or anxiety attacks, and an improvement in spasticity was reported in 4/10 (40%) of patients. CBD appears to have potential therapeutic value for the treatment of drug-resistant epilepsy in Rett syndrome. CBD is well tolerated and, when used in combination with clobazam, may increase the effectiveness of clobazam alone.

Keywords: Rett syndrome; cannabidiol; pharmacoresistant epilepsy.

Publication types

  • Observational Study

MeSH terms

  • Anticonvulsants
  • Cannabidiol* / therapeutic use
  • Clobazam / therapeutic use
  • Epilepsy* / drug therapy
  • Humans
  • Rett Syndrome* / chemically induced
  • Rett Syndrome* / complications
  • Rett Syndrome* / drug therapy
  • Seizures / drug therapy
  • Seizures / etiology

Substances

  • Cannabidiol
  • Clobazam
  • Anticonvulsants