Intranasal Oxytocin for Patients With Autism Spectrum Disorder: A Comprehensive Meta-Analysis of Preclinical and Clinical Studies

The neuropeptide oxytocin (OXT) has been identified as a potential therapeutic intervention for ASD due to its involvement in social bonding and affiliative behaviors. The present study aimed to assess the effectiveness and safety of intranasal administration of OXT in preclinical and clinical investigations related to ASD. The random-effects meta-analysis method evaluated the combined effect sizes, heterogeneity, and publication bias. Additionally, subgroup analyses were conducted to investigate potential moderators. We confirmed 54 preclinical, animal model, and clinical trials on 2593 cases with ASD through systematic search in PubMed, Web of Science, CNKI, Scopus, and Cochrane library databases from 1980 to 2023. The least-squares mean change of ABC-mSW was significantly lower in the OXT group than in Placebo (SMD, -1.45; 95% CI, -2.24, -0.66; P = 0.0003; I2 = 93%). The analysis showed significantly low OXT plasma concentration after intranasal OXT compared with placebo (SMD, 0.94; 95% CI, 0.48, 1.40; P < 0.0001; I2 = 82%). Our analysis showed only CGI improvement was significantly seen in patients with ASD receiving intranasal OXT compared with placebo (SMD, 0.07; 95% CI, 0.00, 0.14; P = 0.04; I2 = 0%). The meta-analysis suggests that the administration of intranasal OXT may have a positive impact on the behavior of individuals with ASD, particularly in terms of enhancing social and communicative abilities. Nevertheless, turning preclinical research discoveries into clinical applications poses a significant hurdle. Additional research is necessary to ascertain the most effective dose, delivery techniques, and long-term consequences to optimize the treatment efficacy and safety for individuals with ASD. Future research endeavors should aim to investigate the heterogeneity of ASD and the variability in responsiveness to interventions involving intranasal OXT.