番茄社区

Epidiolex, a pharmaceutical formulation of cannabidiol (CBD), continued to show benefits for people with Lennox-Gastaut syndrome and Dravet syndrome, real-world data showed.

Seizure frequency was reduced, and emotional functioning, cognition, executive function, and communication were improved among patients using CBD for an average of 2 years, reported Anne Berg, PhD, of Lurie Children's Hospital of Chicago and Northwestern University, in a presentation at the American Epilepsy Society annual meeting.

The findings were from the BECOME survey of 498 caregivers of people with Lennox-Gastaut or Dravet syndrome -- rare, severe epileptic encephalopathies with onset in infancy or early childhood.

"This survey demonstrated what is heard anecdotally in the clinic from individual parents and caregivers: that Epidiolex may, in addition to improving seizure control, have a noticeable positive impact on behavior and cognition for many patients," Berg told 番茄社区.

Prescription Epidiolex, a highly-purified form of CBD, is associated with Lennox-Gastaut or Dravet syndrome in patients ages 1 or older. The drug also is indicated for seizures in people with tuberous sclerosis complex.

Survey respondents came from a caregiver database of patients with Lennox-Gastaut syndrome (396 people) or Dravet syndrome (102 people) in the U.S. who were treated with Epidiolex 100 mg/mL oral solution for 3 months or more. Caregivers were asked to compare the past month with the period before starting CBD.

The 20-minute questionnaire was administered online and was based on questions from validated measures and previously published caregiver reports. Adverse events were not assessed in the survey.

Nearly all respondents (97%) were parents. Patients had an average age of 16 (17 for Lennox-Gastaut and 12 for Dravet) and 52% were male. Patients were treated with CBD for an average of 2 years, were taking a median dose of 14 mg/kg/day, and used a median of four concomitant antiseizure medications.

Most caregivers (84%) reported that seizure frequency had improved since starting CBD treatment. These improvements were seen in convulsive seizures (72%), drop seizures (72%), non-convulsive/non-drop seizures (68%), and nighttime seizures (62%). About one in five respondents (21%) reported worsening in at least one type of seizure.

Many respondents (68%) said seizure severity had improved, and 67% reported more seizure-free days per week. Overall, 16% of respondents stated patients were completely seizure-free during the past month.

Outcomes in non-seizure domains also were better since starting CBD. Alertness, cognition, and executive function improved in 85% of patients, emotional functioning in 82%, and language and communication in 79% of non-verbal patients and 74% of verbal patients. Worsening was reported in at least one question of each domain by 6% to 25% of respondents.

The most frequently reported improvements included being alert (71%), learning new things (71%), being aware of surroundings (71%), being able to engage with others (68%), being happy (67%), and paying attention (66%).

About half of caregivers reported that patient sleep (51%), activities of daily living (51%), and physical functioning (46%) were better with CBD.

Nearly all caregivers (93%) planned to continue CBD treatment. Most (91%) said they would continue because patients had less seizure burden, but 77% also cited improvements in non-seizure outcomes as a reason.

Limitations of the research included possible recall and selection bias due to study design. The survey was conducted with caregivers of people taking Epidiolex, and findings do not apply to other CBD products.