From the Guidelines
Dravet syndrome treatment should prioritize stiripentol as a key medication, given its efficacy in reducing seizures and improving quality of life, as supported by recent studies 1. When considering treatment options for Dravet syndrome, it's essential to focus on seizure control and improving quality of life.
- First-line medications include stiripentol (Diacomit, 50mg/kg/day), which has shown promise in reducing urinary oxalate levels and improving symptoms in patients with Dravet syndrome 1.
- Other treatment options may include valproate (10-60mg/kg/day) and newer FDA-approved treatments like fenfluramine (Fintepla, 0.1-0.7mg/kg twice daily) and cannabidiol (Epidiolex, 5-20mg/kg/day).
- Medications to avoid include sodium channel blockers like carbamazepine, phenytoin, and lamotrigine, which can worsen seizures in Dravet syndrome.
- Non-pharmacological approaches, such as the ketogenic diet (4:1 ratio of fats to carbohydrates and protein), vagus nerve stimulation, and comprehensive developmental support, are also crucial in managing this complex genetic epilepsy syndrome.
- A multidisciplinary approach involving neurologists, dietitians, physical therapists, and other specialists is essential for managing Dravet syndrome, with regular monitoring for side effects and individualized treatment plans that typically continue lifelong 1.
From the FDA Drug Label
DIACOMIT is indicated for the treatment of seizures associated with Dravet syndrome in patients taking clobazam who are 6 months of age and older and weighing 7 kg or more . The effectiveness of DIACOMIT for the treatment of seizures associated with Dravet syndrome was established in 2 multicenter placebo-controlled double-blind randomized studies (Study 1 and Study 2), conducted according to similar protocols
- Treatment Options:
- Key Points:
From the Research
Treatment Options for Dravet Syndrome
The treatment of Dravet syndrome is challenging due to its highly pharmaco-resistant nature. Several studies have investigated various treatment options, including medications and dietary changes.
- First-line treatments include valproate and clobazam, although they are often insufficient to control seizures 3, 4, 5, 6.
- Adjunctive therapies that have shown efficacy in treating Dravet syndrome include stiripentol, topiramate, levetiracetam, bromide, and the ketogenic diet 3, 4, 5, 6, 7.
- Stiripentol is the only new drug to demonstrate efficacy in Dravet syndrome when combined with valproate and clobazam, and it has been approved as an orphan drug in Europe and the USA 3, 4, 6.
- Cannabidiol and fenfluramine are also promising therapies for Dravet syndrome, although they require further evaluation in randomized, controlled trials 4, 5, 7.
- Sodium channel-blocking anticonvulsants, such as carbamazepine and lamotrigine, should be avoided as they can exacerbate seizures 3, 5, 6, 7.
Management of Dravet Syndrome
In addition to prophylactic treatment, patients with Dravet syndrome require home-rescue medication and a status epilepticus protocol 7.
- A multidisciplinary team approach is necessary to provide holistic care, including specialists in physical, occupational, and speech therapy, neuropsychology, social work, and physical medicine 7.
- Non-pharmacologic strategies, such as avoidance of triggers that commonly induce seizures, can also help reduce seizure risk 7.
Medication Interactions and Side Effects
Potential drug-drug interactions and their consequences are a key consideration in everyday practice, including interactions between stiripentol, clobazam, and valproate, as well as between cannabidiol and clobazam 4.
- Regular monitoring of laboratory and clinical parameters, such as blood counts, liver function tests, and serum concentrations of anti-seizure medications, is necessary to minimize side effects and optimize treatment 4.