Six children with TS with focal seizures were identified, four of which are positive for TSC 2 mutation, one positive for TSC 1, and one had no mutation identified. For three patients, vigabatrin was the first line anti-epileptic drug (AED), while it was added on to other AED(s) for the rest. Age range for onset of epilepsy is 4 to 33 months, and initiation age of vigabatrin ranged from 4 months to 9 years 9 months (3/6 <1 year and 3/6 > 32 months age). Duration of Vigabatrin treatment ranges from 9 to 43 months. Three cases have been seizure free for over 12 months, two patients improved (>50% reduction), and one patient remains intractable. Side effects do not show visual compromise. However, encephalopathy was appreciated when combined with CBD in one patient. Two patients have autism spectrum disorder, two patients are developmentally normal without autism, and three are developmentally delayed. All but one patient required another AED: either levetiracetam, oxcarbazepine, or some combination of both.

Observed vigabatrin efficacy in TS children with epilepsy adds to the data and shows that it is safe and effective. Almost all required additional AED(s) to control seizures. In one case, one child developed encephalopathy in combination with CBD. Otherwise, vigabatrin has been safe over the long term.