The efficacy of vigabatrin therapy for treatment of West syndrome

Abstract

　　Objective: To examine the efficacy of vigabatrin (VGB) for treatment of West syndrome (WS) in our hospital. Methods: We retrospectively reviewed the clinical records of the patients who received VGB for treatment of WS. Before the approval in July 2016, we prescribed VGB, which was imported, after informed consents by parents. After the approval, we prescribed VGB for the patients who were registered Sabril Registration System for Prescription and performed ophthalmologic examination. The efficacy was confirmed if a patient became seizure-free or achieved>50% reduction of the number (50% responder rate) . Results: A total of 27 children (male, 17; female, 10; after approval, 8) were included in this study. The pathogenesis of WS was classified as tuberous sclerosis (14 children) , focal cortical dysplasia and/or other cortical dysgeneses (six children) , and abnormal birth history and hypoxic ischemic encephalopathy (four children) , and other cases (three children) . The average age at the start of VGB administration was 36.0 months. The efficacy rate was 86.7% in the TSC group, 50.0% in focal cortical dysplasia and/or other cortical dysgeneses group, and 0% in the other group. We had no patients with severe visual defects nor symptomatic vigabatrin-associated brain abnormalities on MRI. The adverse effects were agitation in eight patients and insomnia in three patients. Conclusions: VGB had excellent efficacy for TSC and focal cortical dysplasia and/or other cortical dysgeneses and no efficacy for the other patients.