Evidence-based Treatment Options for Autism

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Children's Doctor

If there is one clear take-away from 2 decades worth of autism research, it is this: Early diagnosis of autism spectrum disorder (ASD) coupled with prompt, evidence-based and effective intervention will result in the best possible prognosis for the child.

All children with ASD — or even a suspected diagnosis — can benefit from early intervention (EI). Research shows that in an appropriate educational setting, EI for at least 2 years prior to the start of school can result in significant improvements, and some may gain enough skills to successfully enter a regular education elementary school setting. Effective programs focus on developing communication, social and cognitive skills. Physicians should suggest patients begin EI services as soon as the diagnosis is made. If patients have a long wait between a positive screen and a diagnostic evaluation, it’s best to initiate EI even before a formal diagnosis is made.

The most effective treatments available today are applied behavioral analysis, occupational therapy, speech therapy, physical therapy, and pharmacological therapy. Treatment works to minimize the impact of the core features and associated deficits of ASD and to maximize functional independence and quality of life. In 2012, the Missouri Autism Guidelines Initiative summarized the findings from 6 reviews on behavioral and pharmacological interventions in autism. The consensus paper includes current evidence of what interventions have been studied and shown effective.

Applied behavior analysis (ABA)

ABA uses behavioral psychology principles to systematically change behavior by encouraging positive behaviors and discouraging negative ones. In addition, ABA teaches new skills and applies those skills to new situations.

Pivotal response training (PRT) 

RPT is a form of incidental or naturalistic ABA aimed at increasing a child’s motivation to learn, monitor their own behavior, and initiate communication with others by focusing on behaviors that are seen as key to learning other skills, such as language, play and social skills. PRT works to generalize skills across many settings with different people.

Discrete trial teaching 

Is a common form of ABA in which what is being taught is broken down into smaller steps, then using prompts and rewards for each step. Prompts and rewards are phased out over time.

Early Start Denver Model (ESDM)

ESDM is a comprehensive ABA program for infants, toddlers and preschoolers ages 12 to 48 months with ASD. ESDM includes naturalistic ABA, interpersonal exchange, shared enjoyment in joint activities, and promotion of language and communication. The emphasis is on having fun within a developmental framework. Parent involvement is key to the success of the intervention. ESDM has been shown to be effective in a randomized clinical trial. Children who received 20 hours of ESDM per week (5 of the hours provided by parents) over 2 years showed more improvement in cognitive testing (IQ), adaptive skills, and autism symptoms than those who received typical community treatment.

Lovaas Model

The Lovaas Model consists of 20 to 40 hours of highly structured, discrete trial teachings, integrating ABA techniques into an early intervention (EI) program for children between the ages of 2 and 8 years, and no later than age 12. The technique utilizes child-specific reinforcers to motivate and reward success. Additionally, the use of language and imitation are crucial in this teaching model.

Speech therapy

Since people with ASD have deficits in social communication, speech therapy is an important component of treatment. A licensed speech-language pathologist (SLP) helps to improve a child’s communication skills, allowing them to better express their needs or wants. Speech therapy is often most effective when SLPs work with teachers, support personnel, families, and the child’s peers to promote functional communication in natural settings.

If a child is nonverbal and unable to develop verbal communication skill, the use of gestures, sign language, and picture communication programs are often useful tools to improve their abilities to communicate.

Occupational therapy (OT)

OT is often used as a treatment for the sensory integration issues associated with ASD. It is also used to help teach life skills that involve fine-motor movements, such as dressing, using utensils, cutting with scissors and writing. OT works to improve quality of life and the ability to participate fully in daily activities. Each OT program is based on individual evaluations and goals. OT for young children with ASD often focuses on improving sensory integration and sensorimotor issues. In older children, OT often focuses on improving social behavior and increasing independence.

Physical therapy (PT)

PT is used to improve gross motor skills and handle sensory integration issues, particularly those involving the individual’s ability to feel and be aware of their body in space. Similar to OT, physical therapy is used to improve the child’s ability to participate in everyday activities. PT works to teach and improve skills such as walking, sitting, coordination, and balance. PT is most effective when integrated in an EI program.


Pharmaceutical treatments can ameliorate some of the behavioral symptoms of ASD, including irritability, aggression and self-injurious behavior. By medically reducing interfering or disruptive behaviors, other treatments, including ABA, may be more effective. Medications should be prescribed and monitored by a qualified physician.

  • Risperidone is the first Food and Drug Administration-approved medication for the treatment of symptoms associated with of ASD in children and adolescents, including aggressive behavior, deliberate self-injury, and temper tantrums.
  • Aripriprazole is also FDA-approved for the treatment of irritability in children and adolescents with ASD. A 2009 study published in Pediatrics found that in a group of 98 children, by week 8, 52% of those taking aripiprazole (in the form of Abilify®) experienced a 25% or greater reduction in autism-related irritability symptoms compared with 14% of those who took the placebo.

Reference and suggested readings

The Missouri Autism Guidelines Initiative. Autism Spectrum Disorders: Guide to Evidence-based Interventions: a 2012 Consensus Publication. The Missouri Autism Guidelines Initiative. www.autismguidelines.dmh.mo.gov. Accessed June 5, 2917.

Zwaigenbaum L, Bauman ML, Choueiri R, et al. Early intervention for children with autism spectrum disorder under 3 years of age: recommendations for practice and research. Pediatrics. 2015;136 Suppl 1:S60-S81.

Makrygianni MK, Reed P. A meta-analytic review of the effectiveness of behavioural early intervention programs for children with autistic spectrum disorders. Research in Autism Spectrum Disorders. 2010;4(4);577-593.

The Lovaas Model. The Lovaas Institute website. http://www.lovaas.com. Accessed June 5, 2017.

Dawson G, Rogers S, Munson J, et al. Randomized, controlled trial of an intervention for toddlers with autism: the Early Start Denver Model. Pediatrics. 2010:125(1):e17-e23. [Early Start Denver Model]

Arnold LE, Vitiello B, McDougle C, et al. Parent-defined target symptoms respond to risperidone in RUPP autism study: customer approach to clinical trials. J Am Acad Child Adolesc Psychiatry. 2003;42(12):1443-1450.

McCracken JT, McGough J, Shah B, et al. Risperidone in children with autism and serious behavioral problems. N Engl J Med. 2002;347(5):314-321

Myers SM, Johnson CP. Management of children with autism spectrum disorders. Pediatrics. 2007;120(5):1162-1182.

Scahill L, McDougle CJ, Aman MG, et al. Effects of risperidone and parent training on adaptive functioning in children with pervasive developmental disorders and serious behavioral problems. J Am Acad Child Adolesc Psychiatry. 2012;51(2):136-146.

Shea S, Turgay A, Carroll A, et al. Risperidone in the treatment of disruptive behavioral symptoms in children with autistic and other pervasive developmental disorders. Pediatrics. 2004;114(5):e634-e641.

Troost PW, Lahuis BE, Steenhuis MP, et al. Long-term effects of risperidone in children with autism spectrum disorders: a placebo discontinuation study. J Am Acad Child Adolesc Psychiatry. 2005:44(11);1137-1144.

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;20(5):415-422.

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;48(11):1110-9.

Marcus RN, Owen R, Manos G, et al. Safety and tolerability of aripiprazole for irritability in pediatric patients with autistic disorder: a 52-week, open-label, multicenter study. J Clin Psychiatry. 2011;72(9):1270-6.

Owen, R, Sikich L, Marcus RN, et al. Aripiprazole in the Treatment of Irritability in Children and Adolescents With Autistic Disorder. Pediatrics. 2009;124(6):1533-40.

Stigler, KA, Diener JT, Kohn AE, et al. Aripiprazole in pervasive developmental disorder not otherwise specified and Asperger’s disorder: a 14-week, prospective, open-label study. J Child Adolesc Psychopharmacol. 2009;19(3):265-274.


Autism Referral Information and Resources

Find information about making a referral and resources for patient families.

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Center for Autism Research (CAR)

In addition to coordinating research on autism spectrum disorder, CAR offers many resources and educational materials.

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