May 26, 2023

1 in 4 Patients with Epilepsy May Stop Epidiolex Due to Side Effects 

Article published by Dravet Syndrome News

Up to 1 in 4 people with hard-to-treat epilepsy, including those with Dravet syndrome, were found to stop taking Epidiolex (cannabidiol) shortly after starting treatment, primarily due to side effects or a lack of efficacy, according to a real-world study.

“Epidiolex is generally well-tolerated and the majority continued long-term treatment,” researchers wrote, but noted that the data suggest that most patients who discontinued the therapy did so “within the first several months of treatment.”

“Further studies designed to evaluate early identification and potential mitigation of adverse [side] effects … are warranted,” the team wrote, adding that more research is needed to confirm these findings.

The study, “Real-world, long-term evaluation of the tolerability and therapy retention of Epidiolex (cannabidiol) in patients with refractory epilepsy,” was published in the journal Epilepsy & Behavior.

In people with epilepsy, problems in the functioning of the brain cause bursts of uncontrolled electrical activity called seizures. These events can lead to a wide range of mild to severe symptoms that in Dravet patients may present as developmental and cognitive delays, movement problems, and difficulties speaking and sleeping.

Previous trial data suggested Epidiolex can cause several potential therapy-limiting adverse effects or side effects, such as diarrhea, liver enzyme increases (a sign of liver damage), decreased appetite, somnolence or sleepiness, and sleep problems. The results also indicated that treatment interactions with certain medications may be therapy-limiting for some patients.

Now, a team of researchers at the University of Wisconsin-Madison, in the U.S., evaluated the long-term effectiveness of Epidiolex. The focus was on a combination of efficacy and tolerability in people with refractory epilepsy in a real-world setting.

The study included 108 patients seen at the researchers’ center and who took Epidiolex for at least two weeks between December 2018 and December 2020.

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