Sulthiame in the initial therapy of West syndrome – update of a prospective placebocontrolled multicenter study

Objective: For most of the currently used antiepileptic drugs in West syndrome (WS) no placebocontrolled studies do exist. To determine the effectiveness of sulthiame (Stm) on statistical grounds this study was introduced.

Methods: On the basis of a 9-day high dose pyridoxine (PDX) therapy Stm (5mg/kg/day) was tested add-on randomized and double-blind against placebo (PC) from day 4. Without ceasing of spasms and hypsarrhythmia the dosage of the study medication was doubled for the last 3 days.

Results: 51 children (3.5–18 Mo) with newly diagnosed WS were enrolled. 1 child (1.96%) responded during the initial PDX phase. 28 patients received Stm, 22 PC. Four children did not complete the scheme (3 Stm, 1 PC). No child in the PC group responded. In the Stm group 8/25 (32%, 95%-confidence interval (CI): 15,0–53,5%, p=0,005) responded. On an intention-to-treat basis a responder rate of 28.5% (8/28, 95%-CI: 13,2–49,7%, p=0,006) was registered for Stm. When the child responding to PDX is added to the placebo group, the results remain significant (p=0,025).

Conclusion: Stm is effective in the treatment of WS. Its effectiveness is comparable to other antiepileptic drugs. The responder rate reported for vigabatrin in the only placebocontrolled study so far not exceeds that of Stm as demonstrated here. The responder rate attributable to PDX is too low to recommend its high dose use for longer periods as only one child responded during the initial 3-day PDX monotherapy period. Due to our data the use of Stm can be recommended in the initial therapy of WS especially when taking the lack of severe side effects and the short time to prove its effectiveness into account.

Keywords: West syndrome, sulthiame, pyridoxine, placebocontrolled study