Intranasal oxytocin treatment for social deficits and biomarkers of response in children with autism
Open accessAbstract
Significance: Autism spectrum disorder (ASD) is characterized by social deficits. Emerging evidence suggests that the neuropeptide oxytocin, which regulates mammalian social functioning, may be a promising treatment for ASD. However, prior oxytocin treatment trials in ASD patients have produced equivocal results, perhaps because of variability in patients’ underlying neuropeptide biology. Here we provide evidence that oxytocin treatment improves social abilities in children with ASD and that individuals with the lowest pretreatment blood oxytocin concentrations benefit the most from oxytocin administration. These findings reveal a personalized component to oxytocin treatment which may have important implications for accurately testing oxytocin’s therapeutic potential, both for ASD and for a broad range of developmental and psychiatric disorders in which patients exhibit social impairments.
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Small randomized controlled trials of intranasal oxytocin administered for 4 to 24 weeks in persons with autism spectrum disorder have had equivocal results regarding improvements in social functioning, social cognition, or social attention.
“Small, randomized, controlled trials of intranasal oxytocin administered for 4 to 24 weeks in persons with autism spectrum disorder have had equivocal results with regard to oxytocin-associated improvements in social functioning, social cognition, or social attention.18–21”
From Intranasal Oxytocin in Children and Adolescents with Autism Spectrum Disorder (2021) · cites this paper as [18]
Split into 2 atoms — 1 confirmed, 1 partially supported, 0 disputed, 0 misattributed, 0 unverifiable. Expand the claim to see per-atom verdicts.
Experimental — accuracy not yet measuredOne previous trial showed a significant benefit of intranasal oxytocin on the SRS-2 total score only when the baseline plasma oxytocin level was incorporated into the analysis.
“One previous trial showed a significant benefit of intranasal oxytocin therapy on the SRS-2 total score only when the baseline plasma oxytocin level was incorporated into the analysis.18”
From Intranasal Oxytocin in Children and Adolescents with Autism Spectrum Disorder (2021) · cites this paper as [18]
In “Evaluation of Biomarker Inclusion in the Statistical Model” (page not available), the SRS Total Raw Score treatment effect was nonsignificant without pretreatment biomarkers; “Effects of OXT Treatment on Social Abilities” reports a significant effect with them included. But the comparison added both pretreatment blood oxytocin and a receptor gene-expression measure, so it does not establish that incorporating oxytocin alone produced the significant result. (LLM, conf=0.86)
Experimental — accuracy not yet measuredIntranasal OXT holds some promise for treating social deficits, although it may only be beneficial in subsets of patients with low OXT levels.
“intranasal OXT holds some promise [172, 173], although it may only be beneficial in subsets of patients with low OXT levels [174].”
From Neural circuits regulating prosocial behaviors (2023) · cites this paper as [174]
Results of OXT studies in children have overall been more equivocal with mixed results.
“Results of OXT studies in children have overall been more equivocal with mixed results.”
From An Update on Psychopharmacological Treatment of Autism Spectrum Disorder (2022) · cites this paper as [82]
Four studies showed positive short-term results of OXT administration on social responsiveness in children with ASD following 4 or 5 weeks of OXT administration.
“Although 4 studies showed positive short-term results of OXT administration on social responsiveness (following 4 or 5 weeks of OXT administration) [80–83]”
From An Update on Psychopharmacological Treatment of Autism Spectrum Disorder (2022) · cites this paper as [82]
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- Authors
- Parker, K. J., Oztan, O., Libove, R. A., Sumiyoshi, R. D., Jackson, L. P., Karhson, D. S., Summers, J. E., Hinman, K. E., Motonaga, K. S., Phillips, J. M., Carson, D. S., Garner, J. P., & Hardan, A. Y.
- Affiliations
- Stanford University (US)
- Stanford Medicine (US)
- Corresponding author
- Parker, Karen J.
- ORCID
- Venue
- Proceedings of the National Academy of Sciences
- Publisher
- National Academy of Sciences
- Published
- 2017-07-10
- Volume
- 114
- Issue
- 30
- Pages
- 8119-8124
- ISSN
- 0027-8424 (ISSN-L)
- 1091-6490
- PMC
- PMC5544319
- OpenAlex
- W2735278545
- Cited by
- 349
- References
- 51
- Citations by year
- 2026: 28 · 2025: 32 · 2024: 32 · 2023: 34 · 2022: 54 · 2021: 53 · 2020: 43 · 2019: 38 · 2018: 27 · 2017: 7 · 2015: 1
- FWCI
- 45.05
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Proceedings of the National Academy of Sciences (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Mosbacher Family Fund for Autism Research (N/A)
- The Child Health Research Institute (N/A)
- Yani Calmidis Memorial Fund for Autism Research (N/A)
- Autism Speaks (7895)
- Stanford University School of Medicine Postdoctoral Fellowship (N/A)
- HHS | NIH | National Institute of Mental Health (T32MH019908)
- Topics
- Social Sciences › Psychology › Social Psychology › Neuroendocrine regulation and behavior
- Life Sciences › Neuroscience › Cognitive Neuroscience › Autism Spectrum Disorder Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
- Keywords
- autism spectrum disorder, oxytocin, intranasal administration, social dysfunction, social functioning, treatment response biomarkers, neuropeptides, children, Social Responsiveness Scale, placebo response, treatment tolerability
- MeSH terms
- Autism Spectrum Disorder, Administration, Inhalation, Child, Female, Humans, Male, Oxytocics, Oxytocin, Social Skills
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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