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Therapeutic trial of vigabatrin in refractory infantile spasms
C Chiron1, O Dulac, D Beaumont
1Service de Neuropédiatrie, Hôpital St Vincent de Paul, Paris, France.
Insights
Vigabatrin effectively treated drug-resistant infantile spasms in children, showing significant spasm reduction and good tolerability. This offers hope for managing this challenging childhood epilepsy.
Area of Science:
- Pediatric Neurology
- Epileptology
- Pharmacology
Background:
- Infantile spasms are a severe epilepsy form with poor prognosis.
- Treatment resistance is common, necessitating novel therapeutic approaches.
Purpose of the Study:
- To evaluate vigabatrin as add-on therapy for intractable infantile spasms.
- To assess the efficacy and tolerability of vigabatrin in drug-resistant cases.
Main Methods:
- An open-label study involving 70 children with treatment-resistant infantile spasms.
- Vigabatrin was administered as add-on therapy to existing anticonvulsants.
Main Results:
- 43% of patients achieved complete spasm suppression; 68% had >50% reduction.
- Tuberous sclerosis patients showed the best initial response.
- Long-term response rates varied by etiology (symptomatic vs. cryptogenic).
- Vigabatrin was generally well-tolerated with transient side effects.
Conclusions:
- Vigabatrin is a promising treatment for drug-resistant infantile spasms.
- It offers a viable option for patients with poor prognoses.
- Further research into long-term outcomes and specific etiologies is warranted.
Abstract:
Infantile spasms usually start during the first year of life and constitute one of the most difficult types of epilepsy to treat. They carry a very poor prognosis for both epilepsy and mental development. Seventy children, including 47 infants, with intractable infantile spasms were entered into an open study with vigabatrin as add-on therapy to the usual anticonvulsant treatment. All were resistant to previous treatments, including corticosteroids (43 patients), carbamazepine, benzodiazepines, and sodium valproate. Two children withdrew from the study because of intolerance to vigabatrin (hypotonia or hypertonia) before evaluation of efficacy could be made. Of the remaining 68 children, 29 (43%) showed complete suppression of spasms. Forty-six children had a greater than 50% reduction in spasms. The best response was observed in those with tuberous sclerosis (12/14 compared with 12/18 with symptomatic infantile spasms of other origin and 22/36 with cryptogenic infantile spasms). Following the initial response to treatment of these patients (n = 68), a long-term response was confirmed in 75% of children with symptomatic infantile spasms and 36% of children with cryptogenic infantile spasms. In eight children, all other anticonvulsant medication could be definitively withdrawn. Tolerability appeared excellent, with 52 of 70 patients reporting no side effects. Somnolence, hypotonia, weight gain, excitation, and insomnia were the most common problems at the beginning of the study and were usually transient. Given the poor prognosis of this type of childhood epilepsy, vigabatrin appears to be a very interesting advance in the management of drug-resistant infantile spasms.