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Comparative Effectiveness of Therapies for Children With Autism Spectrum Disorders

Clinician Summary – Jun. 30, 2011

Comparative Effectiveness of Therapies for Children With Autism Spectrum Disorders


Table of Contents

Key Clinical Issue

What evidence is available regarding the effectiveness, benefits, and harms of therapies used to address the core and associated symptoms seen among children ages 2–12 years with autism spectrum disorders?

Background Information

Autism spectrum disorders (ASDs) are neurodevelopmental disorders that include: autistic disorder, Asperger syndrome, and pervasive developmental disorder—not otherwise specified. Approximately 1 in every 110 children in the United States has been diagnosed with an ASD. Treatments for ASDs focus on improving core deficits in social communication, addressing challenging behaviors, treating commonly associated difficulties (e.g., anxiety, attention difficulties, sensory difficulties), promoting functional independence, and improving quality of life. The individual goals for treatment vary by child, and a combination of therapies is used to achieve these goals. There is a general lack of consensus regarding treatment effectiveness. Treatment choice may be influenced by availability and costs.


Efforts toward early intervention for ASDs have been encouraging. Research evidence on the effectiveness of therapies for ASDs has shown promise in some areas, but since this is a young field, these results need to be replicated and expanded. There is some evidence to guide choices among medical interventions (for challenging and repetitive behaviors) and early intensive behavioral interventions. There is little or no comparative evidence on which to make decisions about: medical interventions for social or communication symptoms; most behavioral interventions; and educational, allied health, and complementary and alternative medicine (CAM) interventions. For most interventions, the evidence is insufficient to permit an estimate of their benefits or harms. This does not mean that these interventions are not associated with benefits or harms but that further study is required. Evidence suggests that there is an undefined subgroup of children for whom early and intensive behavioral interventions may elicit robust gains while others may not demonstrate marked improvement.

A note about this Clinician Guide

A systematic review of 159 unique clinical studies published in 2000 or later was conducted by independent researchers, funded by the Agency for Healthcare Research and Quality, to synthesize the evidence on what is known and not known about this clinical issue.

This topic was nominated through a public process. The research questions and the results of the report were subject to expert input, peer review, and public comment.

The results of this review are summarized here for use in your decisionmaking and in discussions with patients. The full report, with references for included and excluded studies, is available at

Clinical Bottom Line: Evidence of Effectiveness or Harm

Confidence Scale

High: evidence high
There are consistent results from good-quality studies. Further research is very unlikely to change the conclusions.

Moderate: evidence medium
Findings are supported, but further research could change the conclusions.

Low: evidence low
There are very few studies, or existing studies are flawed.

Insufficient: evidence insufficient
Signifies that evidence is either unavailable or does not permit estimation of an effect.

Literature Review Methods

The systematic review of research summarized here included 159 articles written in English. Studies involved children ages 2–12 years with an ASD or ages 0–2 years at risk for diagnosis of an ASD. All forms of treatment and study designs, with the exception of individual case reports, were reviewed. Studies of behavioral, educational, CAM, and allied health interventions with fewer than 10 subjects were excluded, as were studies of medical interventions with fewer than 30 subjects. A list of included and excluded articles can be found in the full report.

Note Regarding Possible Harms

Other than for risperidone and aripiprazole, there was not enough evidence to estimate the severity and frequency of potential adverse events associated with any of the interventions. According to the United States Food and Drug Administration, there are serious safety issues associated with the use of chelation products. Even when used under medical supervision, these products can cause serious harm, including dehydration, kidney failure, and death.

Gaps in Knowledge

There are no or few studies that describe the following:

Future Research Needs

Continuing improvements in methodological rigor in the field, including:

Large, publicly funded, multisite studies of existing interventions across all treatment types and studies with extended followup times.

Research on medical interventions for which no research has been conducted and on atypical antipsychotics that may be less associated with adverse events than are risperidone and aripiprazole.

What To Discuss With Your Patients

  • Types of therapies and specialists to consider.
  • Treatment goals and realistic expectations.
  • Timing of interventions and the potential value of early interventions versus taking a “wait-and-see” approach.
  • Side effects of medications and longevity of side effects.
  • Daily routine, impact on the family, and the psychological needs of the child and the family.
  • Support groups, local services, and sources of trusted information.
  • Experience of the treatment team in working with children with ASDs.

Resource for Patients

Therapies for Children With Autism, A Review of the Research for Caregivers is a free companion to this clinician guide. It can help parents and caregivers talk with their health care professionals about treatment options. It provides information about:

  • Types of programs and therapies available to children with ASDs.
  • Available evidence on each program or therapy.
  • What to ask when planning therapies and programs for ASDs.

Ordering Information

For electronic copies of Therapies for Children With Autism, A Review of the Research for Caregivers (AHRQ Pub. No. 11-EHC029-A), this clinician guide, and the full systematic review, visit To order free print copies, call the AHRQ Publications Clearinghouse at 800-358-9295.


The information in this summary is based on Comparative Effectiveness of Therapies for Children With Autism Spectrum Disorders, Comparative Effectiveness Review No. 26, prepared by the Vanderbilt Evidence-based Practice Center under Contract No.290-2007-10065-I for the Agency for Healthcare Research and Quality, April 2011. Available at:

This summary was prepared by the John M. Eisenberg Center for Clinical Decisions and Communications Science at Baylor College of Medicine, Houston, TX. It was written by Kim Farina, Ph.D., Thomas Workman, Ph.D., Carla Sharp, M.D., and Michael Fordis, M.D. 

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