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Safety and Tolerability of Transdermal Cannabidiol Gel in Children With Developmental and Epileptic Encephalopathies:
Ingrid E Scheffer1,2, Joe Hulihan3, John Messenheimer4
1University of Melbourne, Austin Health and Royal Children's Hospital, Florey Institute, Melbourne, Victoria, Australia.
Insights
Cannabidiol (CBD) transdermal gel showed promise for treating drug-resistant epilepsies in children. The treatment was safe and well-tolerated, significantly reducing seizure frequency and improving quality of life.
Area of Science:
- Neurology
- Pharmacology
- Pediatrics
Background:
- Developmental and epileptic encephalopathies (DEEs) represent severe, drug-resistant epilepsies in children.
- There is a critical need for alternative therapies beyond oral medications to manage seizures and improve developmental outcomes in this population.
Purpose of the Study:
- To evaluate the safety and tolerability of cannabidiol (CBD) transdermal gel in pediatric patients with DEEs.
- To assess the impact of CBD transdermal gel on seizure frequency, sleep patterns, and overall quality of life.
Main Methods:
- A nonrandomized controlled trial involving 48 children and adolescents (aged 3-18) with DEEs over a 6.5-month treatment period.
- Participants received twice-daily applications of CBD transdermal gel at flexible doses (125-500 mg).
- Safety was assessed via adverse events and skin examinations; seizure frequency (focal impaired awareness seizures [FIAS] and tonic-clonic seizures [TCS]) was the primary efficacy outcome.
Main Results:
- The CBD transdermal gel was generally safe and well-tolerated, with most treatment-related adverse events being mild or moderate.
- Significant reductions in monthly seizure frequency for FIAS and TCS were observed, with a median reduction of 43.5% over the study period.
- Caregivers reported improvements in social engagement, irritability, alertness, energy, sleep, cognition, and concentration.
Conclusions:
- Cannabidiol (CBD) transdermal gel presents a safe and effective alternative for managing drug-resistant epilepsies in children.
- The treatment demonstrated efficacy in reducing seizure burden and positively impacted various aspects of patients' daily lives and well-being.
Importance:
Developmental and epileptic encephalopathies (DEEs) are the most severe group of drug-resistant epilepsies. Alternatives to oral therapies are urgently needed to reduce seizures and improve developmental outcomes and comorbidities in this medically complex population.
Objective:
To assess the safety and tolerability of cannabidiol (CBD) transdermal gel in children with DEEs and to evaluate seizure frequency, sleep, and quality of life.
Design, Setting, And Participants:
This nonrandomized controlled trial was conducted in 2 centers in Australia and New Zealand from April 2018 to July 2019. Children and adolescents aged 3 to 18 years with DEEs who were receiving a stable regimen of 1 to 4 antiseizure medications were eligible for this study. After 1-month baseline and titration periods, patients entered a 5.5-month flexible-dosing maintenance period for a total of 6.5 months of treatment. Data were analyzed throughout the 6.5-month treatment period.
Interventions:
Twice-daily applications of CBD transdermal gel at doses of 125 to 500 mg for 6.5 months.
Main Outcomes And Measures:
Safety and tolerability assessments included adverse events (AEs) and examination of skin. The outcome for seizures was the median percentage change from baseline in monthly (28-day) seizure frequency of focal impaired awareness seizures (FIAS) and tonic-clonic seizures (TCS) over 6.5 months.
Results:
Of 48 patients (mean [SD] age, 10.5 [3.8] years; 26 [54%] boys), 29 (60%) had at least 1 treatment-related AE over 6.5 months; 44 of 46 treatment-related AEs (96%) were mild or moderate. Treatment-related AEs that occurred in at least 5% of patients were application-site dryness, application-site pain, and somnolence (each reported by 4 patients [8%]). The only treatment-related gastrointestinal AE was diarrhea, reported in a single patient. CBD treatment was associated with reductions in FIAS and TCS frequency. Analysis of the 33 patients with FIAS and TCS showed a median (interquartile range) monthly reduction in seizures of 58% (-5.3% to 81.8%) at 5 months and 43.5% (-23.8% to 57.5%) over the entire 6.5-month study period. Parents and caregivers noted improvements in social or interpersonal engagement and irritability (33 of 43 [77%] participants); alertness, energy, and sleep (23 of 43 [53%]); and cognition or concentration (20 of 43 [47%]).
Conclusions And Relevance:
In this study, CBD transdermal gel was safe, well tolerated, and was associated with reductions in FIAS and TCS frequency and disease burden.
Trial Registration:
ClinicalTrials.gov Identifier: ACTRN12618000516280.
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