Background Levetiracetam, a second-generation anti-epileptic drug (AED) with a good efficacy and safety profile, is licensed as monotherapy for adults and children older than 16 years with focal seizures with or without secondary generalization. However, it is increasingly being used off-label in younger children.
Objectives We critically reviewed the available evidence and discuss the present status of levetiracetam monotherapy in children 0-16 years old.
Data Sources We systematically searched the literature using PubMed, Web of Science and Embase up to August 2014 for articles on levetiracetam monotherapy in children. Keywords were levetiracetam, monotherapy and child*. The titles and abstracts of 532 articles were evaluated by AW, of which 480 were excluded. The full texts of the other 52 articles were assessed for relevance.
Results We covered one review, one opinion statement and 32 studies in this review, including four randomized controlled trials, ten open-label prospective studies, eight retrospective studies, and ten case reports. The formal evidence for levetiracetam monotherapy in children is minimal: it is potentially efficacious or effective as initial monotherapy in children with benign epilepsy with centrotemporal spikes. In all of the published studies, however, efficacy and tolerability of levetiracetam seemed to be good and comparable to other AEDs.
Conclusion The data of 32 studies on levetiracetam monotherapy in children were insufficient to confirm that levetiracetam is effective as initial monotherapy for different types of seizures and/or epilepsy syndromes. There is still an urgent need for well designed trials to justify the widespread use of levetiracetam monotherapy in children of all ages.