Clinical trial design in status epilepticus: problems and solutions

Abstract

This paper is written from a pediatric perspective. The starting point in trial design is the identification of important questions, particularly those about therapeutic purposes. The main justification for clinical trials is that status epilepticus is harmful and therefore needs to be treated. In terms of permanent CNS damage and epilepsy following status epilepticus, the rates in published studies vary between 4% and 40% depending on their quality (Raspall-Chaure et al., 2006) and none have looked for subtle cognitive impairments which might be suggested by the finding of acute hippocampal edema following febrile convulsive status epilepticus (CSE) (Scott et al., 2002, 2003). Thus, we are left with a general view that it would be better if we could safely reduce the time that children are convulsing. However, the expense and effort of a randomized controlled trial (RCT) needs to be justified as alternative approaches for example, well-designed epidemiological studies may also provide useful therapeutic information for achieving this goal. The types of questions requiring clinical trial fall into two groups: drug related and clinical approach related.