Dravet syndrome is a rare form of epilepsy that can appear in the first year of life. It causes frequent and often prolonged seizures.
If your child has Dravet syndrome, their doctor may prescribe multiple treatments to reduce the frequency of seizures as well as to stop seizures once they have started.
To learn more about the treatments for this condition, Healthline spoke with neurologist Dr. M. Scott Perry, the medical director of the Genetic Epilepsy Clinic at Cook Children’s Jane and John Justin Neurosciences Center in Fort Worth, Texas. He’s also a member of the Dravet Syndrome Foundation’s medical advisory board.
Read on to learn what Dr. Perry had to say.
If your child has Dravet syndrome, their doctor will prescribe maintenance medication to reduce the frequency of seizures. Your child should take maintenance medication on a regular basis as directed.
The Food and Drug Administration (FDA) has approved three maintenance medications to treat Dravet syndrome:
- cannabidiol (Epidiolex)
- fenfluramine (Fintelpa)
- stiripentol (Diacomit)
Your child’s doctor may also prescribe other maintenance medications that the FDA has not approved for Dravet syndrome specifically. This is known as off-label prescription.
Your child’s doctor may also recommend a ketogenic diet to help reduce the frequency of seizures. Another treatment option is vagal nerve stimulation, in which a small device is implanted in your child’s chest and used to send electrical signals to their brain.
In addition to maintenance medication, your child’s doctor will prescribe a rescue medication to help stop a prolonged seizure or a cluster of seizures after it starts.
“Children with Dravet syndrome are prone to not only frequent seizures but also prolonged seizures, so having a rescue medication is essential. You never know when a seizure is going to happen. You never know when it will be one that requires rescue,” said Perry.
Multiple rescue medications are available, including:
- clonazepam (Klonopin)
- diazepam (Diastat, Valtoco)
- lorazepam (Ativan)
- midazolam (Nayzilam, Versed)
When deciding which medications to prescribe, your child’s doctor will consider factors such as:
- their age
- the types of seizures they have
- how the medication is administered
- the risk of side effects and drug interactions
“The three maintenance medications that are specifically approved for Dravet syndrome are all approved for the treatment of children age 2 years and older,” said Perry.
If your child is under 2 years old, their doctor may still prescribe one of those medications off label. However, you might find it harder to get insurance reimbursement for off-label prescriptions.
Some rescue medications are only approved for older children and adults. Valtoco is approved for people ages 6 and up, and Nayzilam is approved for people ages 12 and up.
Types of seizures
“The next thing we have to consider is the types of seizures that the child is experiencing because certain medications may be better for certain seizures,” said Perry.
The FDA-approved medications Epidiolex, Fintepla, and Diacomit help reduce the frequency of convulsive seizures, which are common in people with Dravet syndrome.
Some people with Dravet syndrome also experience nonconvulsive seizures such as absence seizures. Those people may benefit from another type of maintenance medication.
“If the child’s primary seizure type of concern is absence seizures, then those [FDA-approved] drugs might not be the best choice,” said Perry. “Maybe valproic acid would be a better choice for that kid.”
Maintenance medications are administered orally, as liquids or pills.
Different types of rescue medications are administered in different ways:
- Diastat is a rectal gel, which is put inside a person’s rectum.
- Nayzilam and Valtoco are nasal medications, which are sprayed up a person’s nose.
- Klonopin, Ativan, and Versed are oral medications, which dissolve in a person’s mouth.
“[Diastat] has been available for a long time. It’s easy to use. It’s measured out for the child’s age and weight, so it’s ready to use if a seizure should happen,” said Perry.
“Some people certainly think of nasal administration more favorably,” he added.
Oral rescue medications tend to work less quickly than rectal or nasal medications, he continued.
“I use oral medications more for kids who are having brief but frequent seizures in a cluster rather than a continuous seizure, just because they take a little longer to get absorbed and be effective,” he said.
Side effects and drug interactions
Different types of antiseizure medications may cause different side effects, said Perry.
For some medications, the risk of side effects is higher for people with certain preexisting health conditions or other risk factors. Certain medications can also interact with other drugs, which can cause side effects or make the medications less effective.
“For instance, if they’re on clobazam and you’re thinking about adding Epidiolex, those two will interact to some degree. Those are the kinds of things you have to think about,” said Perry.
It may take some time to find the right treatment plan for your child. They may need more than one medication or other treatments to reduce the frequency of seizures and manage potential symptoms and complications
“Sometimes it’s just a matter of finding the right combination, and when you’ve got multiple treatment options, there might be multiple combinations to try before finding the one that works best,” said Perry.
Before your child’s doctor adds another medication to their treatment plan, they should speak with you about the potential benefits and risks.
“I’m always trying to figure out with parents, ‘What are we trying to achieve with the new medicine?'” said Perry. “If your child’s on a couple of medications, and we still haven’t achieved our goal for seizure control, it’s reasonable to try to add more treatments, but we don’t want to collect medicines if we can avoid it.”
If your child’s symptoms begin to improve after they start a new treatment, their doctor might try to gradually reduce the dose of other antiseizure medication that they take. In some cases, they might wean your child off one medication before prescribing another.
The cost of treatment for Dravet syndrome can be high, especially if your child is taking multiple medications.
If you have insurance but the company refuses to cover one of your child’s prescribed medications, let your child’s doctor know. They may be able to advocate for insurance approval.
If you don’t have adequate insurance coverage or any coverage at all, you might be able to find a manufacturer’s coupon or patient assistance program to offset the costs of care.
“If your insurance coverage is not adequate to cover it, many of the companies that make these drugs have programs that can reduce the cost through their own coupons,” said Perry.
“In some cases, they have patient assistance programs that will help cover the cost in lieu of insurance,” he added.
You might also find it helpful to use services such as GoodRx to look for medication discounts.
Researchers are testing several experimental treatments for Dravet syndrome, including disease-modifying therapies that might help address the underlying causes of the condition.
“There are some disease-modifying therapies on the horizon, and those are exciting because they get at the actual source of the problem,” said Perry.
“They’re not just about treating the seizures, they’re about correcting the problem with the SCN1A gene and thus could hopefully improve some of the other comorbid problems of the condition, like sleep problems and developmental issues,” he added.
Experimental treatments are only available to people who are taking part in a type of study known as a clinical trial.
“One great place to find information about clinical trials, as far as where they’re being done and the inclusion and exclusion criteria, is to go to a ClinicalTrials.gov,” said Perry.
To treat Dravet syndrome, your child’s doctor may prescribe multiple medications. They may also recommend changes to their diet or other therapies.
Speak with your child’s doctor about your treatment goals and options. They can help you understand the potential benefits and risks of different treatment approaches.
Your child may need to try more than one combination of treatments to learn what works best for them. Finding the right treatment plan can help reduce the frequency of their seizures and improve their quality of life.