Seizures associated with conditions such as Dravet syndrome and Lennox-Gastaut syndrome (LGS) can be difficult to treat; these seizures may be treatment-averse and may not respond well to current anti-epileptic medication. However, shares Dravet Syndrome News, a Spanish Expanded Access Program (EAP) found that real-world data supported Epidiolex (also sometimes spelled as Epidyolex) as a therapeutic option for both adults and children living with these two conditions.
What is Epidiolex?
Epidiolex is a prescription, plant-derived, orally administered cannabidiol (CBD) medication. Outside of the above two conditions, Epidiolex is also approved for the treatment of people with tuberous sclerosis complex (TSC). Learn more about Epidiolex.
EAP Research
Within this study, published in Epilepsy & Behavior, Spanish researchers retrospectively analyzed data from 102 individuals with either Dravet syndrome or LGS who were treated with Epidiolex. A majority of the patients in the study had LGS. Patients had previously been treated with anti-seizure therapies but did not respond well. At the start of the study, patients took a mean of 3.4 anti-epileptic medicines. Patients were also experiencing a median 33 seizures and 15 disabling seizures each month. Findings from the study, which show how patients responded to Epidiolex treatment, show that:
- Epidiolex reduced seizure frequency in patients with Dravet syndrome by 50%. Across all seizure types, Epidiolex reduced seizure frequency by 47.6%. Altogether, seizures were reduced by half in 44.9% of patients by six months, though this response rate fell over time.
- Seizure frequency did increase within a small (under 18%) portion of patients. Slightly over ¼ of all patients shared that their seizure frequency did not change on Epidiolex treatment.
- Epidiolex was more effective when used in conjunction with clobazam.
- Seizure severity was reduced in about ⅔ of all patients. Patients also reported that this came with a higher quality-of-life (QOL).
- Patients with Dravet syndrome saw more significant treatment responses (within the first three months) than people with Lennox-Gastaut syndrome.
- One third of patients stopped treatment during the study, with a majority doing so due to a blend of effectiveness and adverse reactions.
- Adverse reactions associated with Epidiolex included appetite loss, diarrhea, and excessive sleepiness.
Ultimately, this EAP found similar results to clinical trials evaluating Epidiolex for LGS and Dravet syndrome. It suggests that Epidiolex could be an effective therapeutic option and overcome some of the barriers to care currently faced by patients.
Dravet Syndrome: An Overview
First described in 1978, and further explored on a genetic basis in 2001, Dravet syndrome is a rare epileptic encephalopathy that is characterized by frequent, prolonged, temperature-sensitive, and treatment-resistant seizures. Typically, children reach developmental milestones on time before seizures begin. Seizures may begin at any point within the first year of life, most often between months 4-12. Risk factors for diagnosis include seizures lasting for more than 10 minutes, occurring on one side of the body, or triggered by a warm water bath. Symptoms and characteristics of Dravet syndrome can include:
- Prolonged febrile and non-febrile seizures
- Myoclonic and partial seizures
- Status epilepticus
- Crouched gait
- Tonic-clonic seizures
- Ataxia (impaired balance and coordination)
- Psychomotor delays
- Sleep disturbances
- Developmental delays
