Randomised, placebo-controlled study of vigabatrin as first-line treatmentof infantile spasms

Purpose: Vigabatrin (VGB) has been shown to be an effective drug in the tre atment of infantile spasms (West syndrome) in predominantly retrospective a nd open but also in prospective studies. This prospective, randomised, and placebo-controlled trial of VGB in infantile spasms was considered to be ju stified and feasible to confirm or refute these previous findings. Methods: Forty children with newly diagnosed infantile spasms received eith er VGB or placebo for 5 days in a double blind, placebo-controlled, paralle l-group study, after which all the infants continuing in the study were tre ated openly with VGB for a minimum of 24 weeks. Results: Compared with baseline, at the end of the double-blind phase, the patients treated with VGB had a 78% (95% confidence interval, 55-89%) reduc tion in spasms compared with 26% (-56-65%) in the group treated with placeb o (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, LS children (38% of the original 40 patients or 42% of the 36 patients who entered the open phase) were spasm free with VGB mon otherapy. 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 syn drome and supports the results of previously published open and prospective trials. It further confirms that VGB could be considered as the drug of fi rst choice in treating infantile spasms.