Evaluating Bumetanide in Autism

Background: Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental disorder characterized by social communication deficits and the presence of restricted interests and repetitive behaviors. The criteria that can be used are variable including biological ones which are more objective (biological variables, electrophysiological, brain imaging, eye tracking etc) or subjective depending on an evaluation by the treating doctor or the parents (ABC, CARS, BOSCC, SRS, CYBOCS, RRB, Conners, etc.) with some scales centered on developmental level (vineland), alterations of life quality (CGI).

Objectives: determine the efficacy of bumetanide in children with autism

Methods: In our various trials, we used primarily CARS) and CGI and more recently SRS and in parallel more pilot studies eye-tracking and fMRI.

Results: CARS (used as a primary criterion) allowed us to show an improvement on the main symptoms of autism including socialization. CGI revealed a general clinical improvement on the dimension of this improvement albeit not specific. Although limited in scope and number of patients, functional magnetic resonance imaging provided useful information on the activation by emotional faces of early visual areas, and areas involved in emotional, social and attentional processing. SRS was highly efficient to detect global changes.

Conclusions: Our results suggest that a heterogeneous disorder such as autism requires many evaluation criteria and objective measures by eye tracking and imaging studies to validate the working hypothesis. Future trials will include -but with a long duration track- alterations of developmental processes. Our experience with our selected primary and secondary outcome measures will be discussed.