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Caregivers’ Perspectives on Epidyolex for Lennox-Gastaut and Dravet Syndromes

Published: 27/07/2023

In recent years, medical cannabis – and, in particular, a highly purified cannabidiol (CBD) formulation (Epidyolex in Europe; Epidiolex in the US) – has been increasingly explored for its potential in the treatment of retractable epilepsy syndromes, namely Lennox-Gastaut and Dravet syndromes. In the UK, this potential played a key role in the legalisation of medical cannabis in November 2018 and subsequent NICE recommendations for its use in this setting.

Past studies have demonstrated the ability of Epidyolex to reduce seizure frequency and severity in addition to secondary outcomes such as improvements in quality of life, adaptive behaviour, and cognitive measures. However, in the largest pivotal trials of Epidyolex in Dravet and Lennox-Gastaut syndromes, the number of patients who completed quality of life and adaptive behaviours scores were low, limiting the interoperability of results. To address this, a recent study interviewed caregivers to collect qualitative data on the further impact of ≥6 months of treatment with Epidyolex on outcomes beyond seizure control.

What are Lennox-Gastaut and Dravet syndromes?

Lennox-Gastaut syndrome and Dravet syndrome are both forms of rare, treatment-resistant epileptic encephalopathies. Both syndromes have early childhood onset and exhibit multiple seizure types in addition to other factors that may contribute to a lower quality of life such as affected mobility, cognitive impairment, and eating difficulties.

What are the Effects of Epidyolex in Lennox-Gastaut and Dravet Syndromes?

Epidyolex has demonstrated efficacy for seizure control with an acceptable safety profile in patients with Dravet and Lennox-Gastaut syndromes in four randomised controlled trials. Further evidence from open-label studies has also revealed that Epidyolex has positive impacts on health-related quality of life, including improved sleep, mood, communication, language use, and behaviour/alertness after treatment.

Design and Methods of the Study

Researchers from Jazz Pharmaceuticals and Acaster Lloyd Consulting conducted a qualitative interview study with caregivers of individuals with Dravet syndrome or Lennox-Gastaut syndrome treated with Epidyolex in the US, the UK, and Germany.

Interviews were conducted by three experienced interviewers using online conferencing and covered caregivers’ views about the symptoms of Dravet syndrome and Lennox-Gastaut syndrome currently experienced, as well as the broader health-related quality of life impacts on patients, caregivers, and other family members. Caregivers were also asked whether symptoms of broader impacts had changed over time and whether this occurred after the initiation of Epidyolex.

Results of the Study

A sample of 21 caregivers (Dravet syndrome = 14; Lennox-Gastaut syndrome = 7) was achieved – a smaller sample than the initial aim of 20 caregivers for each syndrome. Three caregivers were recruited through blind screening and 18 through unblinded screening. The majority of participants were from the US.

Current Symptoms, Functions, and Impacts of Lennox-Gastaut and Dravet Syndromes

All respondents reported the occurrence of epileptic seizures which were commonly reported to be unpredictable. Seizures also varied in type, length, and frequency varied on a monthly, weekly, or daily basis.

Caregivers stated that their child had limited cognitive function and delayed development. Some reported impaired short-term memory and struggles with planning, problem solving, and understanding abstract concepts such as time, safety, and danger. All caregivers noted that their child has difficulty communicating verbally.

A significant proportion of caregivers also noted that their child had problems with mobility and experienced behavioural difficulties (Dravet syndrome = 14; Lennox-Gastaut syndrome = 4), pain and discomfort (Dravet syndrome = 5; Lennox-Gastaut syndrome = 4), sleep disturbances such as Insomnia (Dravet syndrome = 4; Lennox-Gastaut syndrome = 2) and night-time seizures (Dravet syndrome = 3; Lennox-Gastaut syndrome = 1), and other symptoms including appetite and feeding issues, gastrointestinal symptoms and susceptibility to infections.

Both Lennox-Gastaut syndrome and Dravet syndrome were reported to affect the child’s participation in leisure activities and school and impact social activities/relationships and emotional well-being.

Impact on Caregivers

Caregivers commonly reported their day was focused on providing constant care for their child (Dravet syndrome = 6; Lennox-Gastaut syndrome = 5). In addition to direct care, many caregivers conducted administrative tasks, for example, managing and administering medication (Dravet syndrome = 8; Lennox-Gastaut syndrome = 4). Daily, leisure and social activities (e.g., time to exercise, travel or go out, socialise, and engage with hobbies) were also reported to be limited. This was largely attributed to the continuous supervision of their child, lack of trust in other caregivers due to complex needs, and the requirement to constantly change plans.

Almost all caregivers reported adjusting working hours (Dravet syndrome = 5; Lennox-Gastaut syndrome = 2) or quitting their jobs (Dravet syndrome = 7; Lennox-Gastaut syndrome = 3) in order to care for their child. In addition, caregivers commonly described how their caring responsibilities had impacted friendships and family life.

Changes to Symptoms, Functions and Impacts Since Taking Epidyolex

Many caregivers reported positive changes to their child’s cognitive function following treatment with Epidyolex, including improvements in their awareness of surroundings, attention span, and ability to learn new skills and retain information. These improvements were also reported for 1 individual with Dravet syndrome and another with Lennox-Gastaut syndrome who did not experience a reduction in severity and frequency of seizures. Improvements in cognitive function were also reported to have a positive impact on communication.

Some caregivers also reported improvements in their child’s daily activities, their ability to participate in school, emotional well-being, and interest in social activities. Furthermore, reduced seizure frequency and severity were reported to allow caregivers more time to focus on other aspects of care as well as family life. Caregivers also reported improved emotional well-being, particularly in relation to stress/anxiety about their child’s seizures.

However, negative changes were also reported by 10 caregivers following Epidyolex, including loose stools, diarrhoea, somnolence, worsening behavioural difficulties, and reduced appetite. These are broadly similar to the common adverse events reported in the largest pivotal trials of Epidyolex in Dravet syndrome and Lennox-Gastaut syndrome.


This qualitative study provides an in-depth exploration of the effects of Epidyolex beyond seizure control among children and young adults with Dravet syndrome and Lennox Gastaut syndrome, their caregivers and their wider family. These results warrant further investigation on the exact effects of Epidyolex on other symptoms seen in individuals with Lenox-Gastuat and Dravet Syndromes beyond their licensed use to reduce seizures.

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