Treatment of West Syndrome: Present and Future Perspectives

Abstract

The efficacy and side effects of various antiepileptic drugs (AEDs), especially pyridoxine, ACTH and valproate sodium (VPA), in the treatment of West syndrome were reviewed.
ACTH remains to be the most effective treatment for West syndrome. However, there are significant adversive effects with prolonged ACTH therapy. The efficacy of pyridoxine at the dose of 40-50 mg/ kg/day is less encouraging, but there are no serious adversive effects, as opposed to ACTH.
Some reports have indicated the possible efficacy of VPA in regular, large (40-100 mg/kg/day) and very high (100-300 mg/kg/day) doses. However, in patients under the age of 2 years, monotherapy using VPA in lower doses should be employed to minimize the risk of VPA-induced adversive effects, such as fatal hepatic toxicity.
Consequently, a safer and more effective treatment is required.
We have reported a pilot study on combination therapy with high-dose pyridoxine phosphate (40-50 mg/kg/day) and low-dose ACTH (0.01 mg/kg/day) in 25 children with West syndrome, mainly on the basis of neurochemical analysis of the ictal epileptic spasms.
The response rate was similar to those achieved with high- dose ACTH, but a quicker cessation of spasms was obtained with the combination therapy than ACTH monotherapy. Transient increases in liver enzymes occurred in 50%, but none of the patients developed more serious side effects.
Further study is required to determine the efficacy of this combination therapy.