Response to cannabidiol in epilepsy of infancy with migrating focal seizures associated with KCNT1 mutations: An open-label, prospective, interventional study

Poisson, Kelsey E.; Wong, Matthew; Lee, Chon; Cilio, Maria Roberta

doi:10.1016/j.ejpn.2019.12.024

Cited by 17 publications

(28 citation statements)

References 28 publications

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“…Nevertheless, in a study that evaluated three patients with EIMFS secondary to KCNT1 mutations treated with CBD, no benefit was shown in two, while the third, who did not have an overall reduction in seizure frequency, showed a notable reduction in seizure intensity associated with possible developmental progression. [24] A 10-month-old boy with EIMFS reported by another group, however, showed developmental gains and a sustained decrease in seizures when CBD was added to his antiepileptic regimen [25].…”

Section: Discussionmentioning

confidence: 99%

Effectiveness of cannabidiol in a prospective cohort of children with drug-resistant epileptic encephalopathy in Argentina

Caraballo

Demirdjian

Reyes

et al. 2020

Seizure

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We report our preliminary findings regarding effectiveness, safety, and tolerability of cannabidiol (CBD) added to antiepileptic therapy in a cohort of children with drug-resistant epileptic encephalopathies (EEs) with a mean follow-up of 8.5 months (range, 3-12 months). Methods: A prospective cohort study was designed with the aim of assessing the effectiveness, safety, and tolerability of the addition of CBD to standard antiseizure medications (ASMs) in children with drug-resistant EE enrolled at a single center (

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Section: Discussionmentioning

confidence: 99%

Effectiveness of cannabidiol in a prospective cohort of children with drug-resistant epileptic encephalopathy in Argentina

Caraballo

Demirdjian

Reyes

et al. 2020

Seizure

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“…1). The included studies were randomized placebo-controlled trials [13,43,47], open-label interventional studies and their subgroup analyses [14, 15, 17, 18, 20-24, 26-30, 32, 34-38, 41, 42, 48-52], retrospective chart reviews [39,44,46], clinical series [19,40,53], and case reports [16,25,31,33,45,54]. The participants of the studies included patients of pediatric age [14-17, 19, 28, 30, 31, 40, 41, 45, 46, 50, 52, 53], adult age [25,32,33,36,47,48,51,54], and both pediatric and adult age [13, 18, 20-24, 26, 27, 29, 34, 35, 37-39, 42-44, 49].…”

Section: Resultsmentioning

confidence: 99%

“…Open-label drug trials provide class III evidence for the efficacy of CBD administration in patients with CDKL5 deficiency disorder and Aicardi, Doose, and Dup15q [20]; 5-20 mg/kg [39] [17]; N = 20 [27]; N = 5 [30] Seizure frequency reduction: 100% (week 8) [ [16]; N = 1 [30]; N = 2 [40] Reduction in seizure frequency >90 at M6, with seizure-free periods; improvement in alertness [16] CBD discontinued after 6 months for inefficacy [ [30]; N = 2 [49] Reduction >50% in seizures frequency: 1/1 (generalized tonic-clonic), 1/1 (tonic seizures) after 43 months of CBD treatment [30] AEs: decreased appetite, weight loss, diarrhea, elevated transaminases [30] Change in seizure frequency: −20.8 to 42.8 % after receiving a stable dosage of CBD for at least 2 weeks [49]. CBD daily dose: 20 mg/kg [49] syndromes, which are common causes of epileptic encephalopathies.…”

Section: Discussionmentioning

confidence: 99%

Highly Purified Cannabidiol for Epilepsy Treatment: A Systematic Review of Epileptic Conditions Beyond Dravet Syndrome and Lennox–Gastaut Syndrome

et al. 2021

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Background Cannabidiol (CBD), which is one major constituent of the Cannabis sativa plant, has anti-seizure properties and does not produce euphoric or intrusive side effects. A plant-derived, highly purified CBD formulation with a known and constant composition has been approved by the US Food and Drug Administration for the treatment of seizures associated with Dravet syndrome, Lennox-Gastaut syndrome, and tuberous sclerosis complex. In the European Union, the drug has been authorized by the European Medicines Agency for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome, in conjunction with clobazam, and is under regulatory review for the treatment of seizures in patients with tuberous sclerosis complex. Objectives This systematic review aimed to summarize the currently available body of knowledge about the use of this US Food and Drug Administration/European Medicines Agency-approved oral formulation of pharmaceutical-grade CBD in patients with epileptic conditions, especially developmental and epileptic encephalopathies other than Dravet syndrome and Lennox-Gastaut syndrome. Methods The relevant studies were identified through MEDLINE and the US National Institutes of Health Clinical Trials Registry in October 2020. There were no date limitations or language restrictions. The following types of studies were included: clinical trials, cohorts, case-control, cross-sectional, clinical series, and case reports. Participants had to meet the following criteria: any sex, any ethnicity, any age, diagnosis of epilepsy, receiving plant-derived, highly purified (> 98% w/w) CBD in a sesame oil-based oral solution for the treatment of seizures. Data extracted from selected records included efficacy, tolerability, and safety outcomes. Results Five hundred and seventy records were identified by database and trial register searching. Fifty-seven studies were retrieved for detailed assessment, of which 42 were eventually included for the review. The participants of the studies included patients of both pediatric and adult age. Across the trials, purified CBD was administered at dosages up to 50 mg/ kg/day. In a randomized double-blind controlled trial in patients with tuberous sclerosis complex, CBD was associated with a significantly greater percent reduction in seizure frequency than placebo over the treatment period. Open-label studies suggested the effectiveness of CBD in the treatment of children and adults presenting with other epilepsy syndromes than those addressed by regulatory trials, including CDKL5 deficiency disorder and Aicardi, Dup15q, and Doose syndromes, SYNGAP1 encephalopathy, and epilepsy with myoclonic absences. The most common adverse events observed during treatment with CBD included somnolence, decreased appetite, diarrhea, and increased serum aminotransferases. Conclusions The currently available data suggest that response to treatment with a highly purified, plant-derived CBD oilbased solution can be seen in patients across a broad range of epilepsy disorders and et...

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“…Recently, Poisson et al (2020) evaluated CBD response in three patients with EIMFS secondary to KCNT1 mutations; two subjects showed no benefit and voluntarily discontinued CBD. One patient showed an overall decrease in seizure frequency, however, had significant decrease in seizure intensity with the possible progression of development.…”

Section: Migrating Focal Seizures Associated With Kcnt1 Mutationsmentioning

confidence: 99%

“…Recently, Poisson et al. (2020) evaluated CBD response in three patients with EIMFS secondary to KCNT1 mutations; two subjects showed no benefit and voluntarily discontinued CBD.…”

Section: Drug-resistant Pediatric Epilepsymentioning

confidence: 99%

Cannabidiol Treatment for Refractory Epilepsies in Pediatrics

et al. 2020

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Cannabis extracts in oil are becoming increasingly available, and, during the last years, there has been growing public and scientific interest about therapeutic properties of these compounds for the treatment of several neurologic diseases, not just epilepsy. The discovered role of the endocannabinoid system in epileptogenesis has provided the basis to investigate the pharmacological use of exogenously produced cannabinoids, to treat epilepsy. Although, physicians show reluctance to recommend Cannabis extracts given the lack of high-quality safety available data, from literature data cannabidiol (CBD) results to be a promising and safe anticonvulsant drug with low side-effect. In particular, according to early studies, CBD can reduce the frequency of seizures and lead to improvements in quality of life in children affected by refractory epilepsy. So, for these reasons, the detailed study of the interactions between CBD and anticonvulsant drugs (AEDs) administered simultaneously in polytherapy, is arousing increasing interest, to clarify and to assess the incidence of adverse effects and the relation between dose escalation and quality of life measures. To date, in pediatric age, CBD efficacy and safety is not supported by well-designed trials and strong scientific evidence are not available. These studies are either retrospective or small-scale observational and only during the last years Class I evidence data for a pure form of CBD have been available, as demonstrated in placebo-controlled RCTs for patients affected by Lennox-Gastaut syndrome and Dravet syndrome. It is necessary to investigate CBD safety, pharmacokinetics and interaction with other AEDs alongside performing double-blinded placebo-controlled trials to obtain conclusive data on its efficacy and safety in the most frequent epilepsies in children, not just in the epileptic encephalopathy. This review was aimed to revise the available data to describe the scientific evidence for CBD in Pediatric Epilepsies.

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Response to cannabidiol in epilepsy of infancy with migrating focal seizures associated with KCNT1 mutations: An open-label, prospective, interventional study

Cited by 17 publications

References 28 publications

Effectiveness of cannabidiol in a prospective cohort of children with drug-resistant epileptic encephalopathy in Argentina

Effectiveness of cannabidiol in a prospective cohort of children with drug-resistant epileptic encephalopathy in Argentina

Highly Purified Cannabidiol for Epilepsy Treatment: A Systematic Review of Epileptic Conditions Beyond Dravet Syndrome and Lennox–Gastaut Syndrome

Cannabidiol Treatment for Refractory Epilepsies in Pediatrics

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