Summary Recent clinical trials indicate that cannabidiol (CBD) may reduce seizure frequency in pediatric patients with certain forms of treatment-resistant epilepsy. Many of these patients experience significant impairments in quality of life (QOL) in physical, mental, and social dimensions of health. In this study, we measured the caregiver-reported Quality of Life of Childhood Epilepsy (QOLCE) in a subset of patients enrolled in a prospective, open-label clinical study of CBD. Results from caregivers of 48 patients indicated an 8.2 ± 9.9 point improvement in overall patient QOLCE (p<0.001) following 12 weeks of CBD. Subscores with improvement included energy/fatigue, memory, control/helplessness, other cognitive functions, social interactions, behavior and global QOL. These differences were not correlated to changes in seizure frequency or adverse events. The results suggest that CBD may have beneficial effects on patient QOL, distinct from its seizure-reducing effects, however further studies in placebo-controlled, double-blind trials are necessary to confirm this finding.

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CDKL5 deficiency disorder (CDD) is an X-linked disorder resulting from mutations in the CDKL5 gene, which encodes a kinase involved in synaptic plasticity, glutaminergic signaling, and dendrite formation. 1,2 Girls are ~4-fold more often affected, but boys are more severely affected. The incidence is ~1 per 50 000 births. 1 CDD typically presents in the first 3 months of life with treatment-resistant epilepsy (TRE) and hypotonia followed by global developmental delays and cortical visual impairment. 1,2 Infantile spasms and other generalized or mixed generalized/focal epilepsies may be the initial seizure type, with evolution to multiple seizure types that often straddle or fail to conform to standard classifications. Seizures often respond initially but recur, and most children have daily seizures despite multiple antiseizure medication (ASM) regimens. 1,2 Fenfluramine (FFA) enhances serotonin release, positively modulates sigma-1 receptors, 3 and has potent, durable efficacy in treating convulsive seizures in Dravet syndrome and

West Syndrome is characterized by infantile spasms, a hypsarrhythmic electroencephalogram (EEG) pattern, and a poor neurodevelopmental prognosis. First-line treatments include adrenocorticotrophic hormone (ACTH) and vigabatrin, but adverse effects often limit their use. CPP-115 is a high-affinity vigabatrin analogue developed to increase therapeutic potency and to limit retinal toxicity. Here, we present a child treated with CPP-115 through an investigational new drug protocol who experienced a marked reduction of seizures with no evidence of retinal dysfunction. Given the potential consequences of ongoing infantile spasms and the limitations of available treatments, further assessment of CPP-115 is warranted.