title: Improving Outcomes in Infantile Spasms: Role of Pharmacotherapy.
abstract: Infantile spasms, and specifically within the context of West syndrome , is one of the most common epileptic encephalopathies to occur in early infancy. Early recognition and treatment can improve neurodevelopmental outcome in some cases, although the underlying aetiology is probably the most important prognostic factor in both spasm suppression and developmental outcome. Corticosteroids, either adrenocorticotrophic hormone (ACTH) or prednisolone, and vigabatrin are currently the preferred first-line treatment options. Vigabatrin is the treatment of choice when the underlying cause is tuberous sclerosis complex (TSC). Emerging evidence suggests that a combination of steroid and vigabatrin may be more effective in the suppression of spasms and resolution of hypsarrhythmia, the electro-encephalographic signal of spasms. Several other anti-epileptic drugs (AEDs) (levetiracetam, nitrazepam, sodium valproate, topiramate, zonisamide) are usually used as add-on or adjunctive treatment in refractory cases. Pyridoxine (or pyridoxal phosphate) and the ketogenic diet are established treatment options in refractory cases. There is some evidence that neuro-active steroids, including ganaxolone, may be effective; however, clinical trials undertaken intermittently for over a decade have yet to prove their efficacy, not only for the suppression of infantile spasms but also for the resolution of hypsarrhythmia, which may be as important as seizure control in developmental outcome in these children. Insights into developing novel treatment options have emerged from rodent models of infantile spasms, and research is continuing into the efficacy of rapamycin in improving outcomes in infantile spasms. This review provides a brief overview of the existing scientific literature around treatment options and outlines emerging newer treatment options in infantile spasms.
authors.affiliation: The Roald Dahl Department of Neurophysiology, Alder Hey in the Park Children's NHS Foundation Trust, Eaton Road, Liverpool, L12 2AP, UK. anand.iyer@alderhey.nhs.uk.
authors.forename: Anand
authors.initials: A
authors.lastname: Iyer
authors.affiliation: The Roald Dahl Department of Neurophysiology, Alder Hey in the Park Children's NHS Foundation Trust, Eaton Road, Liverpool, L12 2AP, UK.
authors.forename: Richard
authors.initials: R
authors.lastname: Appleton
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D000324:Adrenocorticotropic Hormone / Q000627:therapeutic use; D000927:Anticonvulsants / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D011736:Pyridoxine / Q000627:therapeutic use; D012640:Seizures / Q000188:drug therapy; D013036:Spasms, Infantile / Q000188:drug therapy*; D014402:Tuberous Sclerosis / Q000188:drug therapy; D020888:Vigabatrin / Q000627:therapeutic use
chemical_list: D000305:Adrenal Cortex Hormones; D000927:Anticonvulsants; D000324:Adrenocorticotropic Hormone; D020888:Vigabatrin; D011736:Pyridoxine