Music therapy
The provision of specialist music therapy has some evidence of efficacy, supporting its use in the facilitation of improved socio-communication in children with ASD, although the largest randomized controlled trial on this failed to note an improvement in the core symptoms of ASD following 5 months of music therapy.[308]Geretsegger M, Fusar-Poli L, Elefant C, et al. Music therapy for autistic people. Cochrane Database Syst Rev. 2022 May 9;5(5):CD004381.
https://www.cochranelibrary.com/cdsr/doi/10.1002/14651858.CD004381.pub4/full
http://www.ncbi.nlm.nih.gov/pubmed/35532041?tool=bestpractice.com
[309]Bieleninik L, Geretsegger M, Mössler K, et al. Effects of improvisational music therapy vs enhanced standard care on symptom severity among children with autism spectrum disorder: The TIME-a randomized clinical trial. JAMA. 2017 Aug 8;318(6):525-35.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5817481
http://www.ncbi.nlm.nih.gov/pubmed/28787504?tool=bestpractice.com
The emphasis is on music therapy applied with specialist academic and clinical training.
Antipsychotics to address core symptoms of ASD
Risperidone and aripiprazole, which are currently used within clinical practice for irritability in some people with ASD, have in some studies demonstrated modest efficacy for the management of core symptoms such as repetitive behaviors and social communication difficulties.[218]McDougle CJ, Scahil L, Aman MG, et al. Risperidone for the core symptom domains of autism: results from the study by the autism network of the research units on pediatric psychopharmacology. Am J Psychiatry. 2005 Jun;162(6):1142-8.
http://ajp.psychiatryonline.org/doi/full/10.1176/appi.ajp.162.6.1142
http://www.ncbi.nlm.nih.gov/pubmed/15930063?tool=bestpractice.com
[220]Hirsch LE, Pringsheim T. Aripiprazole for autism spectrum disorders (ASD). Cochrane Database Syst Rev. 2016 Jun 26;(6):CD009043.
http://onlinelibrary.wiley.com/doi/10.1002/14651858.CD009043.pub3/full
http://www.ncbi.nlm.nih.gov/pubmed/27344135?tool=bestpractice.com
[310]Scahill L, Hallett V, Aman MG, et al. Brief Report: social disability in autism spectrum disorder: results from Research Units on Pediatric Psychopharmacology (RUPP) Autism Network trials. J Autism Dev Disord. 2013 Mar;43(3):739-46.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3886122
http://www.ncbi.nlm.nih.gov/pubmed/23104617?tool=bestpractice.com
[311]Marcus RN, Owen R, Kamen L, et al. A placebo-controlled, fixed-dose study of aripiprazole in children and adolescents with irritability associated with autistic disorder. J Am Acad Child Adolesc Psychiatry. 2009 Nov;48(11):1110-119.
http://www.ncbi.nlm.nih.gov/pubmed/19797985?tool=bestpractice.com
[312]Aman MG, Kasper W, Manos G, et al. Line-item analysis of the Aberrant Behavior Checklist: results from two studies of aripiprazole in the treatment of irritability associated with autistic disorder. J Child Adolesc Psychopharmacol. 2010 Oct;20(5):415-22.
http://www.ncbi.nlm.nih.gov/pubmed/20973712?tool=bestpractice.com
However, these studies focused on people with high levels of irritability and it is unclear whether the results are as effective in people with ASD without irritability. In view of this uncertainty, and the potential for adverse effects, routine use is not recommended.
Intranasal oxytocin
Intranasal oxytocin showed promise in preliminary trials for improving social cognition in both adults and children with ASD, although subsequent results have been mixed, and more evidence is required on its long-term safety.[313]Anagnostou E, Soorya L, Chaplin W, et al. Intranasal oxytocin versus placebo in the treatment of adults with autism spectrum disorders: a randomized controlled trial. Mol Autism. 2012 Dec 5;3(1):16.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3539865
http://www.ncbi.nlm.nih.gov/pubmed/23216716?tool=bestpractice.com
[314]Watanabe T, Kuroda M, Kuwabara H, et al. Clinical and neural effects of six-week administration of oxytocin on core symptoms of autism. Brain. 2015 Nov;138(pt 11):3400-12.
https://academic.oup.com/brain/article/138/11/3400/331027
http://www.ncbi.nlm.nih.gov/pubmed/26336909?tool=bestpractice.com
[315]Yatawara CJ, Einfeld SL, Hickie IB, et al. The effect of oxytocin nasal spray on social interaction deficits observed in young children with autism: a randomized clinical crossover trial. Mol Psychiatry. 2016 Sep;21(9):1225-31.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4995545
http://www.ncbi.nlm.nih.gov/pubmed/26503762?tool=bestpractice.com
[316]Ooi YP, Weng SJ, Kossowsky J, et al. Oxytocin and autism spectrum disorders: a systematic review and meta-analysis of randomized controlled trials. Pharmacopsychiatry. 2017 Jan;50(1):5-13.
http://www.ncbi.nlm.nih.gov/pubmed/27574858?tool=bestpractice.com
[317]Okamoto Y, Ishitobi M, Wada Y, et al. The potential of nasal oxytocin administration for remediation of autism spectrum disorders. CNS Neurol Disord Drug Targets. 2016;15(5):564-77.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5080861
http://www.ncbi.nlm.nih.gov/pubmed/27071789?tool=bestpractice.com
[318]Cai Q, Feng L, Yap KZ. Systematic review and meta-analysis of reported adverse events of long-term intranasal oxytocin treatment for autism spectrum disorder. Psychiatry Clin Neurosci. 2018 Mar;72(3):140-51.
https://onlinelibrary.wiley.com/doi/full/10.1111/pcn.12627
http://www.ncbi.nlm.nih.gov/pubmed/29232031?tool=bestpractice.com
[319]Sikich L, Kolevzon A, King BH, et al. Intranasal oxytocin in children and adolescents with autism spectrum disorder. N Engl J Med. 2021 Oct 14;385(16):1462-73.
https://www.nejm.org/doi/10.1056/NEJMoa2103583?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed
http://www.ncbi.nlm.nih.gov/pubmed/34644471?tool=bestpractice.com
Several larger trials of oxytocin are ongoing. Note that the concept of a medication targeted against the core symptoms of ASD may be controversial for some people on the autism spectrum and for their families.
Other interventions
Some other treatments have been suggested to be of benefit to children with ASD and their families. However, there is a lack of research evidence to support their use. Some interventions, therapies, or medications may be of benefit, but research-based evidence is lacking. For example, in the US, Floortime (a method developed to help young children with ASD develop further along their emotional milestones) is frequently used, but research evidence of benefits is limited to a noncontrolled study. Other approaches are nonevidence-based and unlikely to result in benefit. Some suggested treatments may cause harm. Clinicians and parents are advised to discuss treatment approaches with experts or to follow standard guidance from national protocols. Websites of mainstream ASD charities or parent groups generally contain a critique of approaches and can be helpful in allowing clinicians and parents to appraise evidence and expert opinion.[161]Parr J. Autism. BMJ Clin Evid. 2010 Jan 7;2010:0322.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2907623
Many websites offer hope of cure and programs that are described as effective in treating ASD; clinicians and parents are advised to interpret anecdotal or unpublished peer-reviewed research reports with caution.