Infantile spasms is an age-specific
epileptic syndrome in infants and young children. Although the exact mechanism is unknown,
adrenocorticotrophic hormone (
ACTH) has been the mainstay for the therapeutic management of
infantile spasms and other developmental
epilepsies. Clinical benefits of
ACTH in
infantile spasms could partially relate to its stimulatory effects on the release of adrenocorticosteroids and
neurosteroids.
Glucocorticoids,
pyridoxine and
ketogenic diet therapy have all been used for the treatment of refractory
infantile spasms. Recent studies indicate that several newer
anticonvulsant agents, which are positive allosteric modulators of
GABA(A) receptors, are as effective as
ACTH in acutely controlling
infantile spasms. The efficacy of agents that enhance
GABA-mediated inhibition (such as
vigabatrin and
benzodiazepines) for rapid and complete abolition of
infantile spasms has been demonstrated in several clinical studies.
Ganaxolone, a novel
neuroactive steroid has, however, demonstrated outstanding efficacy and better tolerability in children with intractable
infantile spasms.
Zonisamide,
topiramate,
deoxycorticosterone and
neurosteroids are emerging as effective treatment approaches. These new
antiepileptic drugs represent a potential nonhormonal approach for
infantile spasms, but additional studies are needed to verify their efficacy and tolerability. Future studies will hopefully identify rational antiseizure drugs that not only control
infantile spasms but also abrogate its risk on the development of the brain.