Vigabatrin for tuberous sclerosis complex

Vigabatrin (VGB) was found to be an effective anti-epileptic drug to reduce infantile spasms in about 50% of patients and it has been found most effec tive in infantile spasms due to tuberous sclerosis (TSC) in which up to 95% of infants had complete cessation of their spasms. VGB was synthesized to enhance inhibitory gamma-aminobutyric acidergic (GABAergic) transmission by elevating GABA levels via irreversible inhibition of GABA transaminase. Th e mechanism underlying the particular efficacy of VGB in TSC is still unkno wn. However, its efficacy suggests that epileptogenesis in TSC may be relat ed to an impairment of GABAerggic transmission. VGB should be considered as the first line monotheraphy for the treatment of infantile spasms in infan ts with confirmed diagnosis of TSC. The efficacy of VGB treatment can be as sessed in less than 10 days, but usually a few days treatment with a dose o f about 100 mg/kg/day stops infantile spasms. The cessation of the spasms i s associated with a marked improvement of behaviour and mental development. Unfortunately, it has become clear that the use of VGB is associated with a late appearance of visual-field defects in up to 50% of patients. Current ly the minimum duration and doses of VGB treatment that can produce side ef fects are unknown. The feasibility of using short treatment periods (2-3 mo nths) should be investigated. (C) 2001 Elsevier Science B.V. All rights res erved.