C J Yatawara, S L Einfeld, I B Hickie, T A Davenport, A J Guastella. The effect of oxytocin nasal spray on social interaction deficits observed in young children with autism: a randomized clinical crossover trial. Molecular Psychiatry. Published online October 27, 2015.
Interventions for autism are limited. The synthetic hormone oxytocin may provide a potential treatment to improve core social and behavioral difficulties in autism, but its efficacy has yet to be evaluated in young children who potentially may benefit to a greater extent. We investigated the efficacy, tolerability and safety of oxytocin treatment in young children with autism using a double-blind, randomized, placebo-controlled, crossover, clinical trial. Thirty-one children with autism received 12 International Units (IU) of oxytocin and placebo nasal spray morning and night (24 IU per day) for 5 weeks, with a 4-week washout period between each treatment. Compared with placebo, oxytocin led to significant improvements on the primary outcome of caregiver-rated social responsiveness. Overall, nasal spray was well tolerated, and the most common reported adverse events were thirst, urination and constipation. This study is the first clinical trial to support the potential of oxytocin as an early intervention for young children with autism to help improve social interaction deficits.
Inhaling the synthetic hormone oxytocin (multiple brands) led to significant improvements in social interactions in young children with autism spectrum disorder (ASD) in a randomized, double- blind crossover study conducted in Australia.
"We used some of the most widely used assessments of social responsiveness for children with autism," Adam Guastella, PhD, of the Autism Clinic for Translational Research, Brain and Mind Centre, at the University of Sydney, said in a statement.
"We found that following oxytocin treatment, parents reported their child to be more socially responsive at home, and our own blind, independent clinician ratings also supported improved social responsiveness in the therapy rooms of the Brain and Mind Centre," Dr Guastella added...
Compared with placebo, oxytocin led to significant improvements in the main primary outcome of caregiver-rated social responsiveness (P < .01). However, oxytocin had no main effect on the second primary outcome of caregiver reports of the severity of repetitive behavior.
"Significant main effects" were found for the secondary measures of caregiver-rated emotional and behavioral difficulties (P < .001). Experimenter-rated impressions of clinical global improvement were significantly greater for oxytocin compared with placebo (72% vs 41%, P < .05)...
"These findings require confirmation in larger studies," the researchers note. Studies are also needed to determine how oxytocin may improve social behavior and to document how related treatments might be used to boost established social learning interventions. Noting that they cannot rule out a placebo effect, the researchers believe future studies need to consider methods to control for placebo effects to improve detection of therapeutic responses.
Angela Sirigu, PhD, of the Institute of Cognitive Science, Centre de Neuroscience Cognitive, Lyon, France, told Medscape Medical News these results are "encouraging and important because the trial was in children, and it shows that oxytocin has a beneficial cumulative effect. They are not novel since we have already shown (in our 2010 paper in PNAS) that oxytocin alleviates the social impairments of autistic (adult) patients. Therefore, I am happy to see these findings confirming ours.
"The only criticism I have is they used subjective scales only to document the improvement. They don't have rigorous lab testing, such as tasks known to be sensitive to oxytocin effects. Otherwise, I think it is an important addition to the oxytocin literature and autism," Dr Sirigu said.
Evdokia Anagnostou, MD, clinician scientist, University of Toronto, Canada, who was not involved in the study, agrees.
"This is a very promising randomized controlled trial in young children with ASD. Given the paucity of any medications treating the social core deficits of ASD, these are encouraging data, but larger studies will be necessary," Dr Anagnostou told Medscape Medical News.