Epidiolex fails to improve quality of life in children with Dravet syndrome | Effects of CBD may be hampered by the severity of childhood illnesses

Six months of treatment with an oral CBD solution. Epidiolex Among affected children and adolescents driven syndrome or Lennox-Gastaut syndrome (LGS) was not associated with improvements in caregiver-reported quality of life or adaptive behaviors.

That’s according to a small Korean study – although the researchers note that the ability to identify such improvements may have been hampered by the clinical severity of the included patients. These children had treatment-resistant seizures, significant developmental delays, and intellectual disability.

The relationship between CBD and [quality of life] needs to investigate larger numbers of patients,” the researchers wrote.

“Cannabidiol was found to be an effective anticonvulsant drug for patients with Lennox-Gastaut syndrome and Dravet syndrome, but it did not improve [quality of life] in pediatric patients with treatment-resistant epilepsy in our study.”

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the study, “Effects of cannabidiol on adaptive behavior and quality of life in pediatric patients with treatment-resistant epilepsy“in Journal of Clinical Neuroscience.

Cannabidiol, also known as CBD, is the main non-psychoactive component of the cannabis plant, and has been receiving a lot of attention lately for its healing properties.

Study of the effect of Epidiolex on quality of life

Jazz PharmaceuticalsEpidiolex is approved for treatment seizures In children with Dravet and LGS — two rare forms of acute childhood epilepsy — in many countries, including the United States, Europe, and Korea.

While its anti-seizure properties are well established, there is less data on the effects of CBD on quality of life in children with epilepsy, which can be significantly affected by seizures and the treatments used to control them.

A previous study showed that Improving CBD quality of life In pediatric patients, but children were only followed for three months, long-term effects have not been demonstrated.

Now, researchers in Korea have evaluated the effects on quality of life for six months of treatment with Epidiolex. The study included 41 children and adolescents, ages 2 to 18, with Dravet and treatment-resistant LGS. Among them, 11 had Dravit and 30 had LGS.

All children were treated with Epidiolex from May 2019 to December 2020 at Severance Children’s Hospital in Seoul, South Korea.

The 25 boys and 16 girls averaged 4.1 years of age, had failed to respond to more than two previous treatments, and had at least one seizure each month.

At the first study visit, caregivers were asked to keep seizure frequency diaries, and tests were administered to assess children’s development and cognitive function. All of these pediatric patients had severe developmental delay, and all but five had significant intellectual disability. The remaining five children had moderate to severe intellectual disability.

After a month, the participants started treatment with Epidiolex, which was titrated to a dose of 10 mg/kg per day. Examinations were performed three and six months after treatment.

A total of six children with Dravet (54.5%) and 20 with LGS (66.7%) completed six months of treatment. Data from these participants were included in quality of life and adaptive behavior analyzes comparing initiation of treatment (baseline) with six months of treatment.

Quality of life was measured by the provider-reported Quality of Life in Pediatric Epilepsy Questionnaire. Adaptive behaviors, or behaviors necessary to adapt and adapt to an individual’s environment, were assessed by the Child Behavior Checklist Test and the second edition of the Vinland Scales of Adaptive Behavior.

The results showed that after six months of treatment, there were no significant changes in overall quality of life, or in any area of ​​the questionnaire. These included bodily functions, well-being, cognition, social function, and behavior.

However, the researchers noted that any positive effects of CBD could have been overlooked.

“We may have failed to detect the differences because all of our patients have profound intellectual disabilities,” the researchers wrote.

They added that “slight changes in mood may have gone unnoticed by caregivers due to patients’ intellectual disabilities that prevent them from accurately self-reporting.”

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Combined therapy for Dravet syndrome |  Dravet Syndrome News |  illustration of a child's drawing

Severe disability among the participating children

Moreover, the questions asked of the caregivers may have had their answers influenced by many factors, the team noted.

Responses to questions like ‘was he/she obedient?’ and “Did he demand too much attention?” can be very subjective, and can vary depending on patients’ expectations.”

The researchers note that parents’ expectations about Epidiolex’s effectiveness may also have influenced their reporting.

“Now that CBD is more accessible and more data becomes available, expectations may be … lower and more realistic,” the team wrote.

In addition, adaptive behaviors did not change significantly with treatment, but patients did show a significant decrease in the area of ​​motor skills, indicating that children—particularly those with LGS—experienced a decline in their motor skills after six months of treatment. .

“These findings may be related to psychomotor decline, which often occurs in Lennox-Gastaut syndrome,” the team wrote, adding that “because our patients were severely disabled, changes in other domains may be less specific.”

The treatment resulted in fewer seizures. After three months of using Epidiolex, 15 of the 41 children (36.6%) had at least a 50% reduction in seizure frequency, and eight (19.5%) were seizure-free. After six months, 11 children continued to show at least a 50% reduction in seizures, and two remained seizure-free.

A total of 15 participants discontinued treatment six months earlier due to lack of efficacy (73.3%), adverse events (20%), or high cost (6.7%). Adverse events were reported in 16 children (39%), with the most common being behavioral changes (12.2%).

Overall, while the findings suggest that Epidiolex does not significantly affect quality of life or adaptive behaviors, the severity of the children’s illness likely makes these changes difficult to identify, according to the researchers.

The team noted that larger studies are needed to confirm the effects of cannabidiol on quality of life in children with this disease.