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Randomized trial comparing vigabatrin and hydrocortisone in infantile spasms due to tuberous sclerosis
C Chiron1, C Dumas, I Jambaqué
1Department of Child Neurology, Hôpital Saint Vincent de Paul, Paris, France.
Insights
Vigabatrin is more effective than hydrocortisone for treating infantile spasms in infants with tuberous sclerosis. Vigabatrin achieved a higher spasm-free rate and faster results with fewer side effects.
Area of Science:
- Pediatric Neurology
- Epileptology
- Clinical Pharmacology
Background:
- Infantile spasms (IS) are a severe epilepsy syndrome in infants.
- Tuberous sclerosis complex (TSC) is a genetic disorder associated with a high incidence of IS.
- Current treatments for IS include oral steroids and vigabatrin, but comparative efficacy data are limited.
Purpose of the Study:
- To compare the efficacy and safety of vigabatrin versus oral hydrocortisone as monotherapy for newly diagnosed infantile spasms in infants with tuberous sclerosis.
- To determine the optimal first-line treatment for IS in this specific patient population.
Main Methods:
- A prospective, randomized, multicenter study.
- 11 infants received vigabatrin (150 mg/kg/day), and 11 received hydrocortisone (15 mg/kg/day) for 1 month.
- Non-responders were crossed over to the alternative treatment for a 2-month period.
Main Results:
- Vigabatrin achieved a 100% spasm-free rate (11/11) compared to 45% for hydrocortisone (5/11) (P < 0.01).
- Mean time to spasm cessation was significantly shorter with vigabatrin (3.5 days) versus hydrocortisone (13 days) (P < 0.01).
- Vigabatrin showed a better safety profile, with fewer adverse events (5) compared to hydrocortisone (9) (P = 0.006).
Conclusions:
- Vigabatrin is a highly effective and well-tolerated first-line treatment for infantile spasms in infants with tuberous sclerosis.
- Vigabatrin demonstrates superior efficacy and a more favorable safety profile compared to hydrocortisone for this indication.
- These findings support considering vigabatrin as the preferred initial therapy for infantile spasms associated with tuberous sclerosis.
Abstract:
Vigabatrin has been shown to be efficient in infants with infantile spasms and tuberous sclerosis, in open studies. In order to compare vigabatrin to oral steroids, a prospective randomized multicenter study was implemented using both drugs as monotherapy in newly diagnosed patients with infantile spasms and tuberous sclerosis. Eleven infants received vigabatrin (150 mg/kg per day) and 11 hydrocortisone (15 mg/kg per day) for 1 month. Spasm free patients continued vigabatrin or progressively stopped hydrocortisone in 1 month, non-responders were crossed to the other drug for a new 2 month-period. All vigabatrin patients (11/11) were spasm-free versus 5/11 hydrocortisone infants (P < 0.01). Seven patients were crossed to vigabatrin (six for inefficacy, one for adverse events) and became also totally controlled. Mean time to disappearance of infantile spasms was 3.5 days on vigabatrin versus 13 days on hydrocortisone (P < 0.01). Five patients exhibited side effects on vigabatrin but nine on hydrocortisone (P = 0.006). Vigabatrin should therefore be considered as the first choice treatment for infantile spasms due to tuberous sclerosis.