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Abstract Details

Quality of Life and Qualitative Caregiver Assessments in Children and Adolescents with Developmental and Epileptic Encephalopathies Treated with Cannabidiol Transdermal Gel: An Open-label Clinical Trial
Epilepsy/Clinical Neurophysiology (EEG)
P11 - Poster Session 11 (8:00 AM-9:00 AM)
12-006

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.
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.
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.

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.
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.
Authors/Disclosures

PRESENTER 		No disclosure on file
Joe Hulihan, Jr., MD Dr. Hulihan has received personal compensation for serving as an employee of MARINUS PHARMACEUTICALS. Dr. Hulihan has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for ZYNERBA PHARMACEUTICALS.
John A. Messenheimer, Jr., MD Dr. Messenheimer has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Zynerba Pharmaceuticals. Dr. Messenheimer has received intellectual property interests from a discovery or technology relating to health care. Dr. Messenheimer has received intellectual property interests from a discovery or technology relating to health care. Dr. Messenheimer has received intellectual property interests from a discovery or technology relating to health care.
No disclosure on file
Donna Gutterman Donna Gutterman has received personal compensation for serving as an employee of Zynerba Pharmaceuticals.
No disclosure on file
Ingrid E. Scheffer, MBBS, PhD, FRACP, AO (Melbourne Brain Centre) Dr. Scheffer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biohaven Pharmaceuticals, Inc. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Consultant for Longboard Pharmaceuticals . Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Consultant for UCB. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Consultant for Biocodex. Dr. Scheffer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Bellberry Ltd. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Einstein Visiting Fellowship. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. Dr. Scheffer has received intellectual property interests from a discovery or technology relating to health care. Dr. Scheffer has received intellectual property interests from a discovery or technology relating to health care. Dr. Scheffer has received intellectual property interests from a discovery or technology relating to health care. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Akumentis. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Biocodex. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Chiesi . Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Travel/Conference/Speaker honoraria with Stoke Therapeutics. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Zuellig Pharma Inc.. Dr. Scheffer has a non-compensated relationship as a Trial Investigator with Anavex Life Sciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Cerebral Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Cerecin that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Cereval Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Biohaven Ltd. that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Scientific Advisory Board, Trial investigator with Encoded Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with EpiMinder Inc that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with ES-Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Bright Minds Biosciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Marinus Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Neurocrine BioSciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Neuren Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Praxis Precision Medicines that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator, Scientific Advisory Board with Takeda Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Xenon Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Zogenix that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with Neurology that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with Epileptic Disorders that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with Progress in Epileptic Disorders series that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with The Lancet Neurology that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Director with Australian Academy of Health and Medical Sciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Director with Royal Society (Australia). that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Shanghai Zhimeng Biopharma that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with SK Life Science that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Supernus Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with UCB Biopharma SRL that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Ultragenyx that is relevant to AAN interests or activities.