Objective: Dravet syndrome is an epilepsy syndrome of severe epileptic encephalopathy associated with pharmacoresistant seizures and high mortality rate. Tuberous sclerosis complex also has high rate of pharmacoresistant epilepsy as a comorbidity. The aim of this study is to investigate the efficacy and safety of cannabidiol as an adjunctive therapy in patients with pharmacoresistant epilepsy of these two diseases in Taiwan. Methods: A clinical retrospective study was conducted from January 2022 to August 2023 in the pediatric/adult neurology clinics at 4 tertiary medical centers. We reviewed the data obtained from 29 patients whose seizures were pharmacoresistant to more than two antiseizure medications, received cannabidiol therapy with compassionate use. The efficacy and adverse events were recorded. Results: There were 29 patients enrolled in this study. One did not receive cannabidiol due to personal issue. 28 patients (17 Dravet syndrome; 11 tuberous sclerosis complex) received cannabidiol treatment. 2 patients discontinued cannabidiol due to adverse events. 26 patients (14 females; 12 males) continue cannabidiol treatment currently with average age of 16.9±9.3 years (4-42). The dosage of cannabidiol was 12.3±6.8 mg/kg/day (0.5-25). Seizures frequency and intensity were decreased in 82 % (23/28). Adverse events were 10 diarrhea, 3 aggressive behavior, 2 poor appetite, 1 skin eash,3 sleepiness. 1 status epilepticus, 1 constipation. Overall benefit rate was 82% with the feedback by parents. Conclusions: Cannabidiol was efficacious and generally well-tolerated as an add-on treatment for pharmacoresistant epilepsy in patients with Dravet syndrome and tuberous sclerosis complex in Taiwan.

Kuang-Lin Lin
Chang Gung Children's Hospital
Taiwan

Pi-Chuan Fan
National Taiwan University Hospital
Taiwan

Wang-Tso Lee Lee
National Taiwan University Hospital
Taiwan

Pi-Lien Hung
Kaohsiung Chang Gung Memorial Hospital
Taiwan

I-Jun Chou
Chang Gung Children's Hospital
Taiwan

Hsiang-Yu Yu
Taipei Veteran General Hospital
Taiwan

Meng-Han Tsai
Kaohsiung Chang Gung Memorial Hospital
Taiwan