Jazz Pharmaceuticals presents new findings from Caregiver Survey on outcomes of Epidiolex for adult and pediatric patients with severe, childhood-onset epilepsies.

These data were presented at the 2022 American Epilepsy Society (AES) annual meeting taking place December 2–6, 2022, in Nashville, TN.

Epidiolex, a pharmaceutical formulation of highly purified cannabidiol (CBD), is the first and only prescription, plant-derived, cannabis-based medicine approved by the FDA. It is indicated for the treatment of seizures associated with LGS, DS, and tuberous sclerosis complex (TSC) in patients aged one year and older.

"Severe, childhood-onset epilepsies like LGS and DS are lifelong conditions that have significant, long-term impacts on both caregivers and patients. These results are critical in broadening the knowledge base of the potential benefits of Epidiolex treatment across all age groups and identifying additional seizure and non-seizure outcomes to explore in future studies," said lead author and investigator Timothy B. Saurer, Medical Affairs Director of Jazz Pharmaceuticals. "We look forward to additional insights from the BECOME survey as we work to further characterize the specific effects of Epidiolex in both adult and pediatric patients living with rare, treatment-resistant epilepsy conditions."

Results: The results from both poster presentations suggest that a substantial proportion of the caregivers of people with LGS or DS treated with Epidiolex reported improvements in many patients' seizure and non-seizure outcomes, regardless of age group (patients aged less than 18 years and more than 18 years). Limitations to the survey included retrospective caregiver accounts, post-hoc analyses that were not pre-specified, and selection bias due to study design. In addition, the studies were not blinded and there were no comparator arms.

Seizure-Related Outcomes by Age (Poster 3.428) The seizure-related outcome analysis found a large majority of caregivers reported patient seizure improvements as measured by decreased frequency in at least one seizure type (84% aged less than 18 years and more than 18 years) and severity (77% aged less than 18 years; 75% in more than 18 years). Respondents reported patient improvements in specific seizure types: convulsive seizures (72% each), drop seizures (71% each), non-convulsive/non-drop seizures (66% aged less than 18 years; 69% in more than 18 years) and night-time seizures (61% and 63%). Many respondents also reported reductions in use of rescue medications (57%), emergency room visits (56% and 51%), hospitalizations (55% and 50%) and occurrence of seizure-related injuries (48% and 49%).

Non-Seizure-Related Outcomes by Age (Poster 3.429): A substantial proportion of caregivers of patients with LGS or DS, regardless of age, reported improvements in alertness, cognition and executive function, emotional and social function, language and communication, physical function, sleep, and daily activities since initiating Epidiolex treatment. A significant proportion of respondents in both age groups (patients aged less than 18 years and more than 18 years) reported any improvement (possible, minor, definite) in one or more questions regarding the following non-seizure outcome areas: Alertness, cognition, and executive function (87% and 81%) and emotional and social function (82% and 80%).

In patients aged less than 18 years, the most frequently reported improvement was the ability to learn new things (76%). For patients more than 18 years, the most frequently reported improvement was in alertness (70%). Rates of worsening varied by behavior and ranged from 0% to 20% of respondents. The most common side effects for Epidiolex include: sleepiness, decreased appetite, diarrhea, increase in liver enzymes, feeling very tired or weak, rash, sleep problems, fever, vomiting and infections.

About the BECOME (BEhavior, COgnition, and More with Epidiolex) Caregiver Survey: The U.S.-based survey, BECOME (global outcomes survey assessing changes in BEhavior, Cognition, and More with Epidiolex), characterized and quantified real-world seizure and non-seizure outcomes in patients with LGS (80 percent) or DS (20 percent) treated with Epidiolex for three or more months. The investigators conducted an online survey of 498 caregivers (97 percent parents). The caregivers were asked to compare the past month to the period prior to initiation of Epidiolex treatment by answering questions with answers that were multiple choice or ranked on worsening-to-improving scales. All of the questions were based on those from validated measures and other previously published caregiver reports. Epidiolex-associated adverse events were not assessed in this survey. Limitations to the survey included retrospective caregiver accounts and selection bias due to study design.