Abstract
Objective: The objective of this study was to evaluate quality of life (QoL) and caregiver qualitative changes in children with developmental and epileptic encephalopathies (DEEs) who were treated with a cannabidiol (CBD) transdermal gel.
Background: The most severe group of epilepsies, DEEs, are characterized by frequent epileptiform activity that contributes to severe cognitive impairment and often, behavioral disturbances. Patients are typically drug-resistant and frequently have severe to profound disability with comorbidities including autism spectrum disorder, cerebral palsy, gait abnormalities, respiratory disorders and infections, gastrointestinal problems, and an increased mortality risk.
Design/Methods: This was an open-label, multiple-dose study in children and adolescents with DEEs aged 3 to <18 years. Doses of 125, 250, 375, or 500 mg CBD transdermal gel were applied every 12±2 hours during an initial 26-week treatment period followed by an additional 24-week treatment period. The Epilepsy and Learning Disabilities Quality of Life (ELDQOL) scale-modified was used to assess QoL. Qualitative caregiver feedback for the first 26 weeks was collected and coded by 2 independent reviewers, and categorized responses were quantified.
Results: Of the 48 participants enrolled, 54.2% (26/48) were male. Mean (SD) results on the ELDQOL (N=40) showed significant improvements at Week 26 compared with baseline in seizure severity (−0.19 [0.428], p=0.008); behavior (−0.21 [0.384], p=0.001); and mood (−0.15 [0.270], p=0.001). Caregiver qualitative assessments (N=43) were consistent with improvements noted in the ELDQOL scale and included improvements in alertness, awareness, or energy (58%); seizures (51%); cognition/concentration (47%); socially-avoidant behaviors (37%); and school attendance (28%). Twenty-nine (60.4%) participants had ≥1 related adverse event over 26 weeks; 93% were mild or moderate.
Conclusions: Treatment with CBD transdermal gel may be associated with significantly improved cognitive and socio-behavioral symptoms and increased QoL in children and adolescents with DEEs and their families.
Disclosure: Dr. Sadleir has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Zynerba Pharmaceuticals. Dr. Sadleir has received research support from Zynerba Pharmaceuticals.Dr. Hulihan has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Zynerba Pharmaceuticals and Finn Partners. Dr. Messenheimer has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Zynerba, SK Life Sciences, Aquestive. Dr. Ali has nothing to disclose. Dr. Gutterman has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Zynerba Pharmaceuticals. Dr. Sebree has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Zynerba Pharmaceuticals. Dr. Scheffer has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with scientific advisory boards for BioMarin, Nutricia, GSK, Xenon Pharmaceuticals, UCB, received speaker honoraria, support for travel from BioMarin, UCB, GSK, Eisai. Dr. Scheffer has received royalty, license fees, or contractual rights payments from Royalty payment received for PCT/AU2012/001321. Dr. Scheffer has received research support from Trial investigator for GW Pharma, Zogenix, UCB, Ultragenyx, Ovid Therapeutics, Zynerba.
Letters: Rapid online correspondence
REQUIREMENTS
You must ensure that your Disclosures have been updated within the previous six months. Please go to our Submission Site to add or update your Disclosure information.
Your co-authors must send a completed Publishing Agreement Form to Neurology Staff (not necessary for the lead/corresponding author as the form below will suffice) before you upload your comment.
If you are responding to a comment that was written about an article you originally authored:
You (and co-authors) do not need to fill out forms or check disclosures as author forms are still valid
and apply to letter.
Submission specifications:
- Submissions must be < 200 words with < 5 references. Reference 1 must be the article on which you are commenting.
- Submissions should not have more than 5 authors. (Exception: original author replies can include all original authors of the article)
- Submit only on articles published within 6 months of issue date.
- Do not be redundant. Read any comments already posted on the article prior to submission.
- Submitted comments are subject to editing and editor review prior to posting.
