Abstracts

Rationale: Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) are both rare and severe developmental and epileptic encephalopathies characterized by frequent and intractable childhood-onset seizures. Highly purified pharmaceutical CBD is a FDA-approved treatment for seizures associated with LGS and DS. This qualitative study aimed to increase understanding of the impact of CBD on non-seizure-related outcomes (e.g., behavior, cognition, mood and health-related quality of life [HRQoL]) among individuals with DS or LGS and their caregivers.

Methods: Interviews were conducted with caregivers of individuals with DS or LGS in the UK, US, and Germany, who have been treated with CBD (Epidiolex®, 100 mg/mL oral solution) for a ≥ 6 month duration. Participants were recruited through patient associations or a market research agency, and were sent a socio-demographic and clinical background questionnaire to complete via email. Interviews were conducted via telephone by qualitative researchers and explored the symptoms and impacts of DS and LGS, and non-seizure-related effects of CBD. Interviews were recorded and data were analyzed using thematic analysis.

Results: Eighteen interviews were conducted with caregivers of individuals with DS (n=13) and LGS (n=5), aged 4-22 years. Current symptoms included frequent seizures, cognitive impairment, communication, mobility and behavioral difficulties, sleep disruption, and reduced appetite. All individuals required 24-hour supervision and the majority (n=14) needed assistance with self-care due to their seizures and cognitive impairment. Caregivers reported that child’s symptoms impacted their overall HRQoL, with most of them experiencing stress, anxiety, sleep problems, and lack of time for self.

Most caregivers (n=11) reported beneficial HRQoL impacts of CBD, including improvements in awareness, mood, language, social skills, mobility (e.g., balance and coordination), behavior (e.g., less tantrums), appetite, school participation and ability to retain information. Seizure frequency reduction was also commonly (n=13) reported, resulting in caregivers having greater confidence to go out and socialize as a family and having more time for themselves (e.g., for leisure and social activities) due to greater trust in others caring for the individual. A few caregivers (n=4) reported no effects, or only short-term beneficial effects (i.e., lasting 1-6 months). Some caregivers (n=6) reported adverse events of CBD, including loose stools, diarrhea, somnolence, worsening behavioral difficulties, reduced appetite, and burning sensation in throat.

Conclusions: Individuals with DS or LGS experience a range of symptoms and functional issues that impact their broader HRQoL. Results of this study suggest that, in addition to reduced seizure frequency, CBD may have a wide range of non-seizure-related beneficial effects, which warrant further investigation. Quantitative studies with larger sample sizes are required to determine generalizability of findings.

Funding: Please list any funding that was received in support of this abstract.: GW Pharmaceuticals Ltd.