Use of purified cannabidiol as add-on therapy in children with non-self-limited focal epilepsies of childhood: A multicenter study
Gabriela Reyes Valenzuela a , Alberto Espeche b , Sebastian Fortini c , Beatriz Gamboni d , Javier Adi d , Marco Semprino e , Lorena Fasulo e , Santiago Galicchio f , Pedro Cachia f , Santiago Chacón g , Agustin Calvo h , Lucas Beltran i , Claudia Bautista j , Viviana Venegas k , Carolina Alvarez k , Paula Ivarola a , Roberto Caraballo a
- a
- Hospital de Pediatría Prof. Dr. Juan P Garrahan, Buenos Aires, Argentina
- b
- Hospital Público Materno Infantil, Salta, Argentina
- c
- Hospital del Niño Jesús, Tucuman, Argentina
- d
- Hospital Pediátrico Humberto H Notti, Mendoza, Argentina
- e
- Clínica San Lucas, Neuquen, Argentina
- f
- Hospital de Niños Victor J Vilela, Rosario, Santa Fé, Argentina
- g
- Centro Neurología Infantil “CENI” Gualeguaychú, Argentina
- h
- Hospital Regional de Comodoro Rivadavia, Comodoro Rivadavia, Argentina
- i
- Hospital de Niños Juan Pablo ll, Corrientes, Argentina
- j
- Hospital San Roque, Parana, Entre Rios, Argentina
- k
- Clinica Alemana, Santiago de Chile, Chile
Affiliationer
Objective
Evidence for purified cannabidiol (CBD) in children with treatment-resistant non-self-limited focal epilepsies (non-SeLFEs) remains limited. We evaluated its long-term effectiveness and tolerability in a multicenter pediatric cohort.
Methods
We retrospectively analyzed 79 children with treatment-resistant non-SeLFEs treated with adjunctive purified CBD at 11 pediatric neurology centers in Argentina and Chile. All had previously received ≥ 4 ASMs. CBD dosing was individualized according to response and tolerability. Outcomes included changes in seizure frequency and interictal EEG abnormalities, and treatment tolerability.
Results
After a mean follow-up of 30 months (range, 6–44), 43/79 patients (54.4 %) achieved a ≥ 50 % reduction in seizure frequency, including four (5.1 %) who became seizure-free. The mean CBD dose was 34 mg/kg/day (range, 10–40). Response remained stable at 12, 18, 24, and 30 months. Responder rates did not differ significantly between patients with TSC and those with non-TSC focal epilepsies. No significant associations were found between response and seizure type, age at seizure onset, epilepsy duration, age at CBD initiation, or concomitant ASMs. Interictal epileptiform abnormalities improved among responders. Adverse events occurred in 23 patients (29.1 %), were predominantly mild and transient, and were generally manageable with dose adjustments; drowsiness was the most common. No serious treatment-related adverse events occurred.
Conclusion
Adjunctive purified CBD was associated with sustained seizure reduction and good long-term tolerability in children with treatment-resistant non-SeLFEs. Responses were observed across different focal epilepsy etiologies, supporting further evaluation of CBD in this population. Individualized dosing according to response and tolerability may be appropriate in this heterogeneous population.
