Purified cannabidiol as add-on therapy in children with treatment-resistant infantile epileptic spasms syndrome
Gabriela Reyes Valenzuela 1, Adolfo Gallo 1, Agustin Calvo 2, Santiago Chacón 3, Lorena Fasulo 4, Santiago Galicchio 4, Javier Adi 5, Pablo Sebastian Fortini 1, Roberto Caraballo 6
- 1Department of Neurology, Hospital de Pediatría Juan P Garrahan, Combate de los Pozos, Buenos Aires 1881 Argentina.
- 2Department of Pediatric Neurology, Hospital Regional de Comodoro Rivadavia, Argentina.
- 3Pediatric Neurology Center CENI, Gualeguaychú, Argentina.
- 4Department of Neurology, Clinica San Lucas, Neuquen, Argentina.
- 5Department of Pediatric Neurology, Hospital Notti, Mendoza, Argentina.
- 6Department of Neurology, Hospital de Pediatría Juan P Garrahan, Combate de los Pozos, Buenos Aires 1881 Argentina. Electronic address: rhcaraballo@arnet.com.ar.
Affiliationer
Objective: The aim of this study was to assess efficacy, safety, and tolerability of highly purified cannabidiol oil (CBD) as add-on therapy for the treatment of a series of patients with infantile epileptic spasms syndrome (IESS) who were resistant to antiseizure medications and ketogenic dietary therapy.
Material and methods: We conducted a retrospective analysis of the medical records of 28 infants with treatment-resistant IESS aged 6 to 21 months who received highly purified CBD between July 2021 and June 2023. Data were collected on neurological examinations, EEG, Video-EEG and polygraphic recordings, imaging studies, laboratory testing, and seizure frequency, type, and duration, and adverse effects. As the primary outcome, a reduction of frequency of epileptic spasms (ES) was assessed. ES freedom was considered after a minimal time of 1 month without ES.
Results: Sixteen male and 12 female patients, aged 6-21 months, who received CBD for treatment-resistant IESS were included. The etiology was structural in 10, Down syndrome in seven, genetic in nine, and unknown in two. Initial CBD dose was 2 mg/kg/day, which was uptitrated to a median dose of 25 mg/kg/day (range, 2-50). Prior to CBD initiation, patients had a median of 69 ES in clusters per day (range, 41-75) and of 10 focal seizures per week (range, 7-13). After a mean and median follow-up of 15 and 12.5 months (range, 6-26 months), seven patients were ES free and 12 had a >50 % ES reduction. Five of seven patients (71 %) with Down syndrome and 3/5 (60 %) with cerebral palsy responded well. Adverse effects were mild. EEG improvements correlated with ES reductions.
Conclusion: In this study evaluating the use of CBD in children with IESS, 19/28 (67.8 %) had a more than 50 % ES reduction with good tolerability.
Keywords: Cerebral palsy; Infantile epileptic spasms; Purified cannabidiol; Treatment-resistant.
