Randomised, Placebo‐Controlled Study of Vigabatrin as First‐Line Treatment of Infantile Spasms

Abstract

Summary:Purpose: Vigabatrin (VGB) has been shown to be an effective drug in the treatment of infantile spasms (West syndrome) in predominantly retrospective and open but also in prospective studies. This prospective, randomised, and placebo-controlled trial of VGB in infantile spasms was considered to be justified and feasible to confirm or refute these previous findings.Methods: Forty children with newly diagnosed infantile spasms received either VGB or placebo for 5 days in a double blind, placebo-controlled, parallel-group study, after which all the infants continuing in the study were treated openly with VGB for a minimum of 24 weeks.Results: Compared with baseline, at the end of the doubleblind phase, the patients treated with VGB had a 78% (95% confidence interval, 55-89%) reduction in spasms compared with 26% (-56-65%) in the group treated with placebo (p = 0.020). Seven VGB-treated patients and two placebo-treated patients were spasm free on the final day of the double-blind period (p = 0.063). At the end of the study, 15 children (38% of the original 40 patients or 42% of the 36 patients who entered the open phase) were spasm free with VGB monotherapy. No patient withdrew from the study because of an adverse event.Conclusions: This unique randomized, placebo-controlled study is the first to demonstrate the efficacy of a specific drug in the treatment of West syndrome and supports the results of previously published open and prospective trials. It further confirms that VGB could be considered as the drug of first choice in treating infantile spasms.

Recent Advances in the Pharmacotherapy of Infantile Spasms

“…In the US, vigabatrin was approved by the FDA in 2009, and vigabatrin and ACTH are now considered to be the first-line therapies [23]. Clinical studies (Class I) have shown that vigabatrin is superior to placebo in decreasing the frequency of infantile spasms [31].…”

“…After pooling the data of eight separate prospective and retrospective studies, vigabatrin had a good response on spasms in 44 % of 478 patients [11,22,31,39,40,[45][46][47]. In the pooled data of nine separate prospective and retrospective studies, ACTH had a good response on spasms in 59 % of 476 patients [22, 24, 26-29, 34, 48, 49].…”

Topiramate Rapidly Raises Brain GABA in Epilepsy Patients

“…Children with infantile spasms have very low GABA and homocarnosine levels (49). TPM and VGB are the two most effective drugs that improve the outcome of children with infantile spasms (9,50,51). Both drugs are the most potent of newer AEDs that increase human GABA, homocarnosine, and pyrrolidinone (52).…”

Vigabatrina aumenta atividade da superóxido dismutase no corpo estriado de ratos após crises convulsivas induzidas pela pilocarpina

“…A vigabatrina é uma droga antiepiléptica atualmente licenciada em mais de 85 países para o tratamento da epilepsia focal em adultos e para espasmos infantis [8][9][10][11] . É útil também para o tratamento de crises convulsivas associadas com a esclerose tuberosa 12 .…”