Dietz PM, Rose CE, McArthur D, Maenner M. National and state estimates of adults with autism spectrum disorder. J Autism Dev Disord. 2020;50:4258–66.

PubMed  Article  PubMed Central  Google Scholar 

Masi A, DeMayo MM, Glozier N, Guastella AJ. An overview of autism spectrum disorder, heterogeneity and treatment options. Neurosci Bull. 2017;33:183–93.

PubMed  Article  PubMed Central  Google Scholar 

Mason D, McConachie H, Garland D, Petrou A, Rodgers J, Parr JR. Predictors of quality of life for autistic adults. Autism Res. 2018;11:1138–47.

PubMed  Article  PubMed Central  Google Scholar 

Centers for Disease Control and Prevention. Treatment and Intervention Services for Autism Spectrum Disorder. https://www.cdc.gov/ncbddd/autism/treatment.html. Accessed 9 Dec 2021.

DeFilippis M, Wagner KD. Treatment of autism spectrum disorder in children and adolescents. Psychopharmacol Bull. 2016;46:18–41.

PubMed  PubMed Central  Google Scholar 

Dawson G, Burner K. Behavioral interventions in children and adolescents with autism spectrum disorder: a review of recent findings. Curr Opin Pediatr. 2011;23:616–20.

PubMed  Article  Google Scholar 

Vismara LA, Colombi C, Rogers SJ. Can one hour per week of therapy lead to lasting changes in young children with autism? Autism. 2009;13:93–115.

PubMed  Article  PubMed Central  Google Scholar 

Bolognani F, Del Valle Rubido, Squassante L, Wandel C, Derks M, Murtagh L, et al. A phase 2 clinical trial of a vasopressin V1a receptor antagonist shows improved adaptive behaviors in men with autism spectrum disorder. Sci Transl Med. 2019;11:eaat7838.

CAS  PubMed  Article  Google Scholar 

Parker KJ, Oztan O, Libove RA, Mohsin N, Karhson DS, Sumiyoshi RD, et al. A randomized placebo-controlled pilot trial shows that intranasal vasopressin improves social deficits in children with autism. Sci Transl Med. 2019;11:eaau7356.

CAS  PubMed  Article  PubMed Central  Google Scholar 

Kompier NF, Keysers C, Gazzola V, Lucassen PJ, Krugers HJ. Early life adversity and adult social behavior: focus on arginine vasopressin and oxytocin as potential mediators. Front Behav Neurosci. 2019;13:143.

CAS  PubMed  Article  PubMed Central  Google Scholar 

Umbricht D, Del Valle RM, Hollander E, McCracken JT, Shic F, Scahill L, et al. A single dose, randomized, controlled proof-of-mechanism study of a novel vasopressin 1a receptor antagonist (RG7713) in high-functioning adults with autism spectrum disorder. Neuropsychopharmacol. 2017;42:1914–23.

CAS  Article  Google Scholar 

Ratni H, Rogers-Evans M, Bissantz C, Grundschober C, Moreau J-L, Schuler F, et al. Discovery of highly selective brain-penetrant vasopressin 1a antagonists for the potential treatment of autism via a chemogenic and scaffold hopping approach. J Med Chem. 2015;58:2275–89.

CAS  PubMed  Article  Google Scholar 

Jacob S, Veenstra-Vanderweele J, Murphy D, McCracken J, Smith J, Sanders K, et al. Efficacy and safety of balovaptan for socialisation and communication difficulties in autistic adults in North America and Europe: a phase 3, randomised, placebo-controlled trial. Lancet Psychiatry. 2022;9:199–210.

PubMed  Article  Google Scholar 

Hollander E, Jacob S, Jou RJ, McNamara N, Sikich L, Tobe R, et al. A phase 2 randomized controlled trial of balovaptan in pediatric participants with autism spectrum disorder. JAACAP. 2020;59:S262–3.

Google Scholar 

Haflioadottir SH, Juhl CB, Nielsen SM, Henriksen M, Harries IA, Bliddal H, et al. Placebo response and effect in randomized clinical trials: meta-research with focus on contextual effects. Trials. 2021;22:493.

Article  Google Scholar 

Evers AWM, Colloca L, Blease C, Annoni M, Atlas LY, Bendetti F, et al. Implications of placebo and nocebo effects for clinical practice: expert consensus. Psychother Psychosom. 2018;87:204–10.

PubMed  Article  Google Scholar 

Kobak KA, Leuchter A, DeBrota D, Engelhardt N, Williams JBW, Cook IA, et al. Site versus centralized raters in a clinical depression trial—impact on patient selection and placebo response. J Clin Psychopharmacol. 2010;30:193–7.

PubMed  Article  Google Scholar 

Williams JB, Popp D, Kobak KA, Detke MJ. The power of expectation bias. Eur Psychiatry. 2012;27:1.

Google Scholar 

Siafis S, Ciray O, Schneider-Thoma J, Bighelli I, Krause M, Rodolico A, et al. Placebo response in pharmacological and dietary supplement trials of autism spectrum disorder (ASD): systematic review and meta-regression analysis. Mol Autism. 2020;11:66.

PubMed  Article  PubMed Central  Google Scholar 

Jones BDM, Razza LB, Weissman CR, Karbi J, Vine J. Magnitude of the placebo response across treatment modalities used for treatment-resistent depression in adults—a systematic review and meta-analysis. JAMA Psychiat. 2021;4:e2125531.

Google Scholar 

Rutherford BR, Roose SP. A model of placebo response in antidepressant clinical trials. Am J Psychiatry. 2013;170:723–33.

PubMed  Article  PubMed Central  Google Scholar 

Dobson ET, Strawn JR. Placebo response in pediatric anxiety disorders: implications for clinical trial design and interpretation. J Child Adolesc Psychopharmacol. 2016;26:686–93.

PubMed  Article  PubMed Central  Google Scholar 

Rutherford BR, Pott E, Tandler JM, Wall MM, Roose SP, Lieberman JA. Placebo response in antipsychotic clinical trials—a meta-analysis. JAMA Psychiat. 2014;71:1409–21.

Article  Google Scholar 

Kubo K, Fleichhacker WW, Suzuki T, Yasui-Furukori N, Mimura M, Uchida H. Placebo effects in adult and adolescent patients with schizophrenia: combined analysis of nine RCTs. Acta Psychiatr Scand. 2019;139:108–16.

CAS  PubMed  Article  Google Scholar 

Luu S, Province H, Berry-Kravis E, Hagerman R, Hessl D, Vaidya D, et al. Response to placebo in Fragile X syndrome clinical trials: an initial analysis. Brain Sci. 2020;10:629.

CAS  Article  PubMed Central  Google Scholar 

Dunlop BW, Thase ME, Wun CC, Fayyad R, Guico-Pabia CJ, Musgnung J, et al. A meta-analysis of factors impacting detection of antidepressant efficacy in clinical trials: the importance of academic sites. Neuropsychopharmacology. 2012;37:2830–6.

CAS  PubMed  Article  PubMed Central  Google Scholar 

Fraugas D, Diaz-Caneja CM, Pina-Camacho L, Umbricht D, Arango C. Predictors of placebo response in pharmacological clinical trials of negative symptoms in schizophrenia: a meta-regression analysis. Schizophr Bull. 2018;45:57–68.

Article  Google Scholar 

Meister R, Abbas M, Antel J, Peters T, Pan Y, Bingel U, et al. Placebo response rates and potential modifiers in double-blind randomized controlled trials of second and newer generation antidepressants for major depressive disorder in children and adolescents: a systematic review and meta-regression analysis. Eur Child Adolesc Psychiatry. 2020;29:253–73.

PubMed  Article  Google Scholar 

Brogger-Mikkelsen M, Ali Z, Zibert JR, Andersen AD, Thomsen SF. Online patient recruitment in clinical trials: systematic review and meta-analysis. J Med Internet Res. 2020;22:e22179.

PubMed  Article  PubMed Central  Google Scholar 

King BH, Dukes K, Donnelly CL, Sikich L, McCracken JT, Scahill L, et al. Baseline factors predicting placebo response to treatment in children and adolescents with autism spectrum disorders: a multisite randomized clinical trial. JAMA Pediatr. 2013;167:1045–52.

PubMed  Article  PubMed Central  Google Scholar 

Yoo H. Genetics of autism spectrum disorder: current status and possible clinical applications. Exp Neurobiol. 2015;24:257–72.

PubMed  Article  PubMed Central  Google Scholar 

Cataldo I, Azhari A, Esposito G. A review of oxytocin and arginine-vasopressin receptors and their modulation of autism spectrum disorder. Front Mol Neurosci. 2018;11:27.

PubMed  Article  CAS  PubMed Central  Google Scholar 

Charman T, Loth E, Tillmann J, Crawley D, Wooldridge C, Goyard D, et al. The EU-AIMS Longitudinal European Autism Project (LEAP): clinical characterisation. Mol Autism. 2017;8:27.

PubMed  Article  PubMed Central  Google Scholar 

Stavropoulos KKM, Baker E. Priming the pathway: combining oxytocin and behavioral intervention to improve outcomes in autism spectrum disorder: In: Wu W, Kostoglou-Athanassiou I, editors. Oxytocin and health [internet]. London: IntechOpen; 2021. https://www.intechopen.com/chapters/76208. https://doi.org/10.5772/intechopen.96859

Masi A, Lampit A, Glozier N, Hickie IB, Guastella AJ. Predictors of placebo response in pharmacological and dietary supplement treatment trials in pediatric autism spectrum disorder: a meta-analysis. Transl Psychiatry. 2015;5:e640.

CAS  PubMed  Article  PubMed Central  Google Scholar 

Anagnostou EJ, Jones N, Huerta M, Halladay AK, Wang P, Scahill L, et al. Measuring social communication behaviors as a treatment endpoint in individuals with autism spectrum disorder. Autism. 2015;19:622–36.

PubMed  Article  Google Scholar 

Brugha TS, Doos L, Tempier A, Einfeld S, Howlin P. Outcome measures in intervention trials for adults with autism spectrum disorders; a systematic review of assessments of core autism features and associated emotional and behavioural problems. Int J Methods Psychiatr Res. 2015;24:99–115.

PubMed  Article  PubMed Central  Google Scholar 

Bolte EE, Diehl JJ. Measurement tools and target symptoms/skills used to assess treatment response for individuals with autism spectrum disorder. J Autism Dev Disord. 2013;43:2491–501.

PubMed  Article  Google Scholar 

Akhondzadeh S, Fallah J, Mohammadi MR, Imani R, Mohammadi M, Salehi B, et al. Double-blind placebo-controlled trial of pentoxifylline added to risperidone: effects on aberrant behavior in children with autism. Prog Neuropsychopharmacol Biol Psychiatry. 2010;34:32–6.

CAS  PubMed  Article  Google Scholar 

Scahill L, Aman MG, Lecavalier L, Halladay AK, Bishop SL, Bodfish JW, et al. Measuring repetitive behaviors as a treatment endpoint in youth with autism spectrum disorder. Autism. 2015;19:38–52.

PubMed  Article  Google Scholar 

Moricke E, Buitelaar JK, Rommelse NNJ. Do we need multiple informants when assessing autistic traits? The degree of report bias on offspring, self, and spouse ratings. J Autism Dev Disord. 2016;46:164–75.

PubMed  Article