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Randomized trial of vigabatrin in patients with infantile spasms
R D Elterman1, W D Shields, K A Mansfield
1Dallas Pediatric Neurology Associates, The Center for Epilepsy Treatment, Medical City Dallas Hospital, Texas 75230, USA. RoyDElterman@aol.com
Insights
High-dose vigabatrin (VGB) is effective for treating infantile spasms (IS) in infants. This study confirmed VGB
Area of Science:
- Pediatric Neurology
- Epileptology
- Clinical Pharmacology
Background:
- Infantile spasms (IS) are a severe pediatric epilepsy with limited treatment options.
- Vigabatrin (VGB) is a common treatment for IS outside the United States.
- Efficacy and safety of VGB in recent-onset IS require further evaluation.
Purpose of the Study:
- To evaluate the efficacy and safety of vigabatrin in infants with recent-onset infantile spasms.
- To compare low-dose versus high-dose vigabatrin treatment outcomes.
- To assess the time to response and long-term safety profile of vigabatrin.
Main Methods:
- A 2-week randomized, single-masked, multicenter study with a 3-year follow-up.
- Included infants (<2 years) with recent-onset IS (<3 months duration) and no prior treatment.
- Patients received either low-dose (18-36 mg/kg/day) or high-dose (100-148 mg/kg/day) vigabatrin.
Main Results:
- Higher response rates were observed in the high-dose vigabatrin group (24/67) compared to the low-dose group (8/75) (p < 0.001).
- Response rates increased significantly within 2 weeks and continued to improve over 3 months.
- Vigabatrin was well-tolerated, with only nine patients discontinuing due to adverse events.
Conclusions:
- Vigabatrin demonstrates significant efficacy and a favorable safety profile in treating infantile spasms.
- High-dose vigabatrin therapy leads to a faster and more pronounced treatment response.
- The findings support vigabatrin's use, especially in infantile spasms secondary to tuberous sclerosis.
Background:
Infantile spasms are a rare but devastating pediatric epilepsy that, outside the United States, is often treated with vigabatrin. The authors evaluated the efficacy and safety of vigabatrin in children with recent-onset infantile spasms.
Methods:
This 2-week, randomized, single-masked, multicenter study with a 3- year, open-label, dose-ranging follow-up study included patients who were younger than 2 years of age, had a diagnosed duration of infantile spasms of no more than 3 months, and had not previously been treated with adrenocorticotropic hormone, prednisone, or valproic acid. Patients were randomly assigned to receive low-dose (18-36 mg/kg/day) or high-dose (100-148 mg/kg/day) vigabatrin. Treatment responders were those who were free of infantile spasm for 7 consecutive days beginning within the first 14 days of vigabatrin therapy. Time to response to therapy was evaluated during the first 3 months, and safety was evaluated for the entire study period.
Results:
Overall, 32 of 142 patients who were able to be evaluated for efficacy were treatment responders (8/75 receiving low-dose vigabatrin vs 24/67 receiving high doses, p < 0.001). Response increased dramatically after approximately 2 weeks of vigabatrin therapy and continued to increase over the 3-month follow-up period. Time to response was shorter in those receiving high-dose versus low-dose vigabatrin (p = 0.04) and in those with tuberous sclerosis versus other etiologies (p < 0.001). Vigabatrin was well tolerated and safe; only nine patients discontinued therapy because of adverse events.
Conclusions:
These results confirm previous reports of the efficacy and safety of vigabatrin in patients with infantile spasms, particularly among those with spasms secondary to tuberous sclerosis.