Intranasal Oxytocin in Children and Adolescents with Autism Spectrum Disorder

RCT (n=290) of intranasal oxytocin vs. placebo showed no significant between-group differences in least-squares mean change from baseline on measures of social or cognitive functioning over a period of 24 weeks (−3.7 oxytocin vs. −3.5 placebo; p=0.61).

SPS commentary:

Editorial discusses what can be concluded from this well-powered, carefully conducted trial. It notes firstly, that oxytocin as administered in previous clinical studies and trials and in a method similar to most off-label use in clinical practice did not improve social function in a generalisable population of persons with autism spectrum disorder, and therefore these results do not support the current off-label use of oxytocin in the treatment of autism spectrum disorder. It add nevertheless, that it may be premature to reject the oxytocin signalling pathway (or efforts to increase social motivation in general) as a potential treatment target in autism spectrum disorder, pointing out potential mitigating factors in interpreting the trial results include age range of patients, particularly around dynamic periods of development, given that the physiologic effects of oxytocin differ over developmental stages and that there may be critical windows for treatment effects; the likely short half-life of oxytocin activity in the brain, which potentially limits the effectiveness of even sustained twice-daily administration; and the insensitivity of current measures to capture social motivation, even though they represent the current standard. It calls for a need to learn more about the neurobiologic basis of complex social behaviour and to create measures that can be used effectively to evaluate its components in clinical trials.

Source:

New England Journal of Medicine

Resource links:

Editorial