Oxytocin Nasal Spray Promising in Children With Autism

Megan Brooks

November 04, 2015

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.

The study was published online October 27 in Molecular Psychiatry.

Significant Effects

Participants included 31 children (27 boys) aged 3 to 8 years who met Diagnostic and Statistical Manual of Mental Disorders, 4th Edition, Text Revision criteria for autistic disorder, Asperger's disorder, or pervasive developmental disorder–not otherwise specified.

In a blinded crossover fashion, they 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 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).

Overall, the nasal spray was generally well tolerated, the investigators say. The most common adverse events were increased thirst as well as increased daytime and nighttime urination and constipation, which were reported by twice as many children receiving oxytocin (10 reports) than those receiving placebo (five reports).

"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.

Promising Confirmation

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.

The study had no commercial funding. The authors have disclosed no relevant financial relationships.

Mol Psychiatry. Published online October 27, 2015. Abstract


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