Rare Childhood Epilepsies: Dravet Syndrome and Lennox-Gastaut Syndrome - Episode 3

Treatment Options and Patient Selection for Dravet and Lennox-Gastaut Syndromes

Two experts detail the current treatment options for Dravet and Lennox-Gastaut syndromes and how they select which patients should use a specific drug.

Elizabeth Thiele, MD, PhD: The past several years has been a pretty exciting time in the world of Dravet and LGS [Lennox-Gastaut syndrome] for clinicians taking care of this patient population and for the patient population, the families, and individuals because we’ve seen progress in our ability to treat these patients. Prior to these recent medications that we’re gaining still experience with, what did the treatment landscape look like? I know you were very involved in the North American recommendations for treatment in Dravet. How did you base it on what medications we had?

Elaine C. Wirrell, MD: In Dravet, before we had the recent approval of the 3 newer medications, most people were starting with clobazam or valproic acid. If you look at the evidence for those treatments, it’s based on small retrospective series, and responder rates are well below 50%. Although it didn’t make things worse, it didn’t make things a whole lot better. Many of those children continue to have very problematic seizures. For many of these children, we often would also consider use of ketogenic diet very early. With the new medications approved, the ketogenic diet can be a highly effective therapy for Dravet syndrome.

Elizabeth Thiele, MD, PhD: I agree. You and I are ketogenic diet zealots and have been for many years. It’s saying a patient with LGS is a bit different because we had FDA-approved medications for LGS, but there haven’t been any trials comparing them. A lot of us went on what our personal experience had been with the medications in these patient populations. There was a seizure or the comorbidities. What are the other unmet needs that this patient population has?

Elaine C. Wirrell, MD: The other unmet need that’s significant is the elevated risk of sudden unexpected death in epilepsy. If we look at the Dravet population—and that’s where we’ve got better studies— about 1 of 100 children die every year. Most often it’s death related to SUDEP [sudden unexpected death in epilepsy]. Sometimes it’s prolonged status or accidental injury as a result of a seizure. Most of it is SUDEP. If you look again, with the LGS population they have a significant SUDEP risk, and those are very concerning for the families. The impact that these disorders have on the families, parents, caregivers, and siblings of these children is a huge unmet need.

Elizabeth Thiele, MD, PhD: I agree. If have nothing more to add to that. We spend a lot of time in our program trying to take care of not only the patient with regard to managing their seizures but a child and the family unit, with siblings, etc. That’s definitely something that’s true. Improving the seizures can lead to improvement in many aspects of the family and the patient’s life.

This transcript has been edited for clarity.