Abstracts

Rationale:
Epidiolex® is a 99% pure oral cannabidiol (CBD) extract and the first FDA-approved medication utilizing CBD. It is indicated for adjunctive therapy in patients ≥ 2 years with Lennox Gastaut (LGS) and Dravet syndromes (DS). The objective of this study was to evaluate the efficacy and tolerability of Epidiolex® in patients with varied refractory epilepsies at our institution.
Method:
We retrospectively reviewed the records of patients with refractory epilepsy treated with Epidiolex® at our institution from 2018 to 2020. Forty-six patients were divided into 4 subgroups: 1) LGS (n=21, 45.7%), 2) DS (n=3, 6.5%), 3) other generalized (n=16, 34.8%), 4) focal (n=6, 13%). Data collected included age, gender, developmental delay (DD), epilepsy type, EEG findings, prior antiepileptic drugs (AED), use of ketogenic diet (KD) or vagal nerve stimulation (VNS), Epidiolex® dosage, follow-up duration, treatment response, and adverse effects (AEs). Efficacy was defined as > 50% seizure reduction or seizure freedom. Descriptive analysis of data was performed in all patients and in each subgroup. Seizure reduction was compared between all subgroups. Kaplan-Meier survival estimates were used to assess retention rates of Epidiolex® (time to discontinuation due to side effects or lack of efficacy).
Results:
Twenty-four patients were male (52.2%) and 22 were female (47.8%). Mean age was 13 years (range 1.5-27). DD was present in all 46 patients (100%). Nine patients (19.6%) had previously used VNS and 8 patients (17.4%) had previously used KD. Thirty-five patients (76.1%) had failed ≥ 2 AEDs. Epidiolex® mean maintenance dose was 16.16 mg/kg/day (range 4.04-27.09). Mean length of treatment was 9.5 months (range 0.9-18.3). Twenty-four patients (52.2%) had > 50% seizure reduction and 22 (47.8%) were non-responders. In subgroup analysis, 11 patients (52.4%) with LGS, 2 patients (66.7%) with DS, 7 patients (43.7%) with other generalized epilepsy, and 4 patients (66.7%) with focal epilepsy were responders. No patients became seizure free. Fifteen patients discontinued treatment, 12 (80%) due to lack of efficacy and 3 (20%) due to AEs. Gastrointestinal (GI) complaints were seen in 8 patients (17.4%) and somnolence in 6 (13%). Kaplan-Meier analysis showed a mean time to Epidiolex® discontinuation of 13 months, (95% CI 11.6-15.5). There was no statistical difference among different diagnoses. The main factor leading to early discontinuation of Epidiolex® was GI side effects.
Conclusion:
In this small series of patients with refractory epilepsy, Epidiolex® was efficacious in up to 2/3 of patients including those with LGS, DS, and focal epilepsy. Reported AEs were GI symptoms or somnolence, and no serious AEs were reported. In our experience, Epidiolex ® is an effective and well tolerated adjunctive AED in the treatment of patients with varied refractory epilepsies. A larger randomized, prospective study is necessary to confirm our findings.
Funding:
:None