These early seizures, which are typically triggered by a fever or exposure to temperature changes, are usually convulsive or involve jerking movements on one side of the body. Between ages 1 and 5, most children with Dravet syndrome will begin to have myoclonic seizures, which involve brief jerks of a muscle or group of muscles. But the disorder can also cause numerous other types of seizures too. Dravet syndrome is caused by a genetic mutation, but it’s usually not inherited from parents. There is no cure for the disorder, but treatment focuses on reducing the severity and frequency of seizures and the associated complications, particularly status epilepticus (when seizures don’t stop or occur close together), which can be life-threatening.
Fevers, which should be treated with medications like ibuprofen (Advil) or acetaminophen (Tylenol)Overheating (typically from hot baths or exercise)Sudden temperature changes (hot or cold)Flashing lightsVisual stimuli like stripes and other contrasting patternsStress or excessive excitementVaccines (your child’s healthcare provider may advise you to give your child a fever reducer before a vaccination and for 24 hours afterward to stave off a fever)
First-Line Drugs for Dravet Syndrome
At the onset of Dravet syndrome, doctors may prescribe anticonvulsant medications like valproic acid and clobazam to control seizures, but they usually aren’t effective enough on their own. (3) There’s little research on how effective valproic acid is for managing the seizures associated with Dravet syndrome. But in some studies, about one-fourth to one-half of study participants responded well to the medication, experiencing more than a 50 percent reduction in the frequency of seizures. (4) However, valproic acid may cause several severe side effects, including liver damage, increased blood ammonia, pancreatitis (inflammation of the pancreas), and low blood platelet count. Less-severe side effects include altered appetite, hair loss, tremor, and sedation. Clobazam may also cause sedation, as well as ataxia (loss of control of bodily movements), decreased behavioral inhibitions, and increased salivation. One study suggested that about 28 percent of patients responded well to clobazam. In most cases, if one of these first-line drugs fails to control seizures, the other is used as well. But most children will require a second-line therapy for Dravet syndrome.
Cannabidiol (CBD) for Dravet Syndrome
In June 2018, the U.S. Food and Drug Administration approved Epidiolex (cannabidiol), which was both the first drug to be approved by the FDA for the treatment of Dravet syndrome and the first marijuana-derived drug to be approved. (5) Cannabidiol, or CBD, is derived from marijuana but does not cause the “high” associated with marijuana. The drug was approved for the treatment of seizures in patients with Dravet syndrome who are at least 2 years old. Epidiolex is a more than 98 percent pure oil-based CBD extract from the cannabis plant. (6) Researchers have conducted several clinical trials on the efficacy of Epidiolex for people who have Dravet syndrome or Lennox-Gastaut syndrome who were also taking other seizure medications. (5) When taken in addition to other medications, Epidiolex was shown to effectively reduce the frequency of seizures when compared with a placebo. Common side effects included sleepiness, sedation, and lethargy; elevated liver enzymes; reduced appetite; diarrhea; rash; fatigue, malaise, and weakness; insomnia, sleep disorder, and poor-quality sleep; and infections.
Stiripentol for Dravet Syndrome
In August 2018, the FDA approved a second drug for the treatment of Dravet syndrome seizures. The drug, Diacomit (stiripentol), is an anticonvulsant that is indicated only for people who have Dravet syndrome who are at least 2 years old and are also taking clobazam. (7) In clinical trials, people with Dravet syndrome who took Diacomit in addition to other seizure medications experienced fewer seizures than those taking a placebo with other seizure medications. (8) The most common side effects of Diacomit are sleepiness, reduced appetite, agitation, impaired balance or coordination, weight loss, low muscle tone, nausea, shaking, difficulty saying words; and insomnia. Other serious side effects may include excessive sleepiness, loss of appetite, weight loss, decrease in blood cell counts, and thoughts about suicide or death.
Fenfluramine for Dravet Syndrome
In June 2020, the FDA approved another medication to treat Dravet syndrome, Fintepla (fenfluramine). (9) Fintepla was approved for the treatment of seizures associated with Dravet syndrome in people 2 years old and older. Two clinical studies found that people who took Fintepla had significantly greater reductions in the frequency of convulsive seizures than subjects who took a placebo. Because Fintepla is associated with valvular heart disease (VHD) and pulmonary arterial hypertension (PAH), people who take it are required to undergo cardiac monitoring using echocardiograms before treatment, every six months during treatment, and once three to six months after they are no longer taking the treatment. The most common side effects in clinical studies were reduced appetite; drowsiness, sedation, and lethargy; diarrhea; constipation; abnormal echocardiogram; fatigue; lack of coordination, balance disorder, gait disturbance (trouble with walking); increased blood pressure; drooling, saliva overproduction; fever; upper respiratory tract infection; vomiting; weight loss; risk of falls; and status epilepticus.
Topiramate for Dravet Syndrome
Physicians may also prescribe topiramate as another second-line treatment for Dravet syndrome. (4) Topiramate is an antiepileptic drug that acts on numerous seizure-related mechanisms in the brain. Four studies showed that 35 to 78 percent of people with Dravet syndrome who took topiramate experienced a more than 50 percent reduction in seizure frequency, with up to 17 percent of patients becoming seizure-free at least in the short term. Side effects include sleepiness, reduced appetite, slowing of verbal processing, metabolic acidosis (too much acid in bodily fluids), and kidney stones.
Ketogenic Diet for Treating Dravet Syndrome
The ketogenic diet, which is high in fats and low in carbohydrates, is another second-line therapy for Dravet syndrome, as well as other epilepsy disorders that are difficult to control. Studies have shown that about two-thirds of people with Dravet syndrome respond well to a ketogenic diet. Some of them experience less-frequent seizures while on the diet, or no seizures at all. The ketogenic diet requires a person to get approximately 90 percent of their daily calories from fat instead of carbohydrates. (10) Sources of fat for the diet include butter, heavy whipping cream, mayonnaise, and canola and olive oil. The ketogenic diet is very strict — it needs to be tailored to work with the total caloric needs of each individual, and the consumption of carbohydrates and proteins must be carefully controlled. For these reasons, experts recommend that people who have Dravet syndrome or parents of children with Dravet syndrome work closely with a dietitian to develop an effective plan. Potential side effects of the diet include various gastrointestinal issues, such as nausea, vomiting, and constipation, in addition to kidney stones and high cholesterol. (4) One advantage of the ketogenic diet is that it causes fewer side effects, such as lethargy and cognitive and behavioral issues, than drug treatments. (11)
Other Dravet Syndrome Therapies
If initial treatments are not effective for seizure control, doctors may prescribe one of a few other therapies. Levetiracetam, another antiepileptic drug, was found to effectively reduce seizure frequency in 64 percent of participants in a small study of 28 patients. (4) However, other retrospective studies show significantly lower efficacy (closer to 11 percent). The major side effect of the medication is behavioral disinhibition. Vagus nerve stimulation (VNS) is a type of treatment in which a device implanted under the skin in the chest delivers electrical impulses to the brain via the vagus nerve. (12) It may help treat seizures by increasing blood flow to certain areas of the brain, raising levels of neurotransmitters, and changing EEG (electroencephalogram) patterns during a seizure. Several relatively small retrospective case series have shown the treatment to effectively reduce seizure frequency in 25 to 35 percent of participants. (4)
Antiseizure Medications to Avoid
There are numerous antiepileptic agents on the market, but not all can be used to control or prevent seizures in people who have Dravet syndrome. For most people, Dravet syndrome is caused by a mutation in the SCN1A gene, which is involved in encoding the sodium channels along which neurons (nerve cells) transmit messages, thus allowing proper brain function. (13) Research shows that antiepileptic agents that act on sodium channels (sodium channel blockers) may worsen Dravet syndrome seizures. (4,13) Medications to avoid include:
Tegretol (carbamazepine)Trileptal (oxcarbazepine)Lamictal (lamotrigine)Sabril (vigabatrin)Banzel (rufinamide)Dilantin (phenytoin)Fosphenytoin (Cerebyx, Prodilantin)
