Diacomit may be effective for hard-to-treat seizures outside Dravet

Responders seen in nearly half of group of 30 Dravet patients, 24 others

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

Share this article:

Share article via email
Two medical professionals look at a tablet one is holding, while talking.

Diacomit (stiripentol), known to help control seizures in people with Dravet syndrome, also may aid those with other childhood disorders marked by hard-to-treat seizures, according to a study out of Spain.

“Although further prospective studies are needed, our findings suggest that add-on [Diacomit] may be a therapeutic option for many patients with DREE [refractory developmental and epileptic encephalopathies], not just those with Dravet syndrome,” the researchers wrote.

The study, “Efficacy and tolerability of add-on stiripentol in real-world clinical practice: An observational study in dravet syndrome and non-dravet developmental and epileptic encephalopathies,” was published in Epilepsia Open.

Recommended Reading
An illustration shows an up-close view of the human digestive system.

Gastrointestinal, eating problems found to be common in Dravet

Diacomit is approved specifically for Dravet syndrome in the US and Europe

Dravet syndrome is marked by seizures that usually develop early in childhood and often are hard to control. Dravet is one of a broader group of diseases called DREEs that are marked by hard-to-treat seizures early in life.

Diacomit has been approved as an adjunct therapy to treat seizures in Dravet patients in Europe since 2007 and in the U.S. since 2018.

Unlike other anti-seizure medicines used in Dravet that were originally developed for other indications, Diacomit was specifically developed to treat Dravet. Currently, it is only officially authorized for use in this disease.

Theoretically, the therapy also might help to control seizures in other DREEs, but there hasn’t been much formal study testing this idea.

To learn more, a team of scientists in Spain conducted a review of data on patients treated with Diacomit at Hospital Ruber International in Madrid between 2000 and early 2023.

The study covered 82 patients, 55 with Dravet syndrome and 27 with other DREEs, such as Lennox-Gastaut syndrome or myoclonic atonic epilepsy (also called Doose syndrome). Patients ranged in age from 1 to nearly 60 years old, although most of them were children.

Most of the patients were taking at least three anti-seizure medications. In addition to Diacomit, the most commonly used treatments were Depacon (sodium valproate), followed by Onfi (clobazam) and Keppra (levetiracetam).

Researchers noted that, compared to people with other DREEs, those with Dravet were generally younger when seizures first appeared. Consistently, treatment with Diacomit and other anti-seizure medications typically started earlier in life for children with Dravet syndrome.

Responders were those with a 50% or greater drop in seizure frequency

Among these 82 patients, about two-thirds (68.3%) were still taking Diacomit one year after starting the therapy. Most who stopped the treatment did so because it was not effective, but the rate of discontinuation was similar among patients with Dravet or non-Dravet DREEs.

Data on seizure frequency was available for 54 patients, 30 with Dravet and 24 with other DREEs. In the months prior to starting on Diacomit, their median seizure frequency was eight seizures per month.

After a year on Diacomit, about two-thirds (65%) of these patients were deemed responders, meaning their seizure frequency had decreased by at least 50%. Around 1 in 3 patients (33%) had a reduction in seizure frequency of at least 90%, with about 1 in 5 (18%) being seizure-free.

These rates were comparable among patients with Dravet or with other DREEs.

“Within our small sample, we were unable to find differences in outcomes and retention rates between the Dravet syndrome group and the non-Dravet DREE group,” the scientists wrote.

Nearly half of the patients (46.3%) reported side effects with Diacomit’s use, given at a daily median dose of 38.5 mg/kg; the most common included drowsiness, reduced appetite, and irritability. There weren’t notable differences in side effect profiles in patients with Dravet syndrome or other diseases; about a handful of patients discontinued Diacomit because of side effects.

The researchers noted that this study was done at a single center and included only a few dozen patients, which may not be a big enough sample size to detect statistically meaningful differences.

Still, they said these findings indicate that Diacomit may be a helpful treatment option for at least some non-Dravet DREEs. For example, they noted that two of three patients with myoclonic atonic epilepsy showed an “excellent response” to Diacomit.

While the scientists noted that more research is needed to verify these results, “given the relative rarity of epileptic encephalopaties and the scarcity of available data, our series provide a significant contribution to the literature,” they wrote.

The company that makes Diacomit, Biocodex, was not directly involved in this study.