Autism Treatment Reviews for Physicians

Autism Treatment Reviews

Written by
Peggy Halliday, MEd, BCBA
Zachary Houston, MS, BCBA
Elisabeth Kinney, MS, BCBA
Scott M. Myers, MD

Although screening and early recognition of autism spectrum disorders (ASDs) are important, the role of the primary healthcare provider does not end with diagnosis. Management responsibilities after the diagnosis of ASDs include providing high quality medical care and guiding families to effective interventions and accurate information sources. Primary healthcare providers, such as pediatricians, family physicians, nurse practitioners, and physician assistants, may take part in any or all of these duties in addition to making referrals to subspecialists and coordinating services.

In a national survey conducted in 2007, pediatricians and family physicians reported low self-perceived competency in providing care for children with ASDs and a desire for education (Golnik, Ireland, & Borowsky, 2009). Fortunately, in recent years, literature reviews and guidelines have been published which summarize the evidence and help medical professionals to manage their patients with ASDs. In this article, we summarize the treatment recommendations of five reviews that have been published in the medical literature in the last four years, including the American Academy of Pediatrics (AAP) guidelines (Carr & LeBlanc, 2007; Myers & Johnson, 2007; Golnik, Ireland, & Borowsky, 2009; Myers, 2009; Carbone, Farley & Davis, 2010; Munshi, Gonzalez-Heydrick, Augenstein, & D’Angelo, 2011).

General Management Issues 

Broad treatment goals include improving quality of life by: (1) correcting or minimizing the core deficits (social impairment, communication impairment, and restricted, repetitive behaviors and interests) and associated deficits, (2) maximizing functional independence by facilitating learning and academic achievement, acquisition of self-care and daily living skills, and development of play and leisure skills, and (3) eliminating or minimizing problem behaviors that interfere with functioning (Myers & Johnson, 2007; Myers, 2009). Most interventions that are helpful for achieving these goals are carried out by parents and professionals such as teachers, therapists, and behavior specialists- not by physicians. However, efforts to optimize health are likely to have a positive impact on educational progress and quality of life.

The medical home model of care, which is advocated for children with ASDs and other special healthcare needs, includes provision of care that is accessible, continuous, comprehensive, family-centered, coordinated, compassionate, and culturally effective (Myers, 2009). Reviewers point out that in the case of patients with ASDs, office visits and physical examinations may be challenging and require extra time and effort.

In addition to issues specific to their neurodevelopmental disorder, individuals with ASDs have the same basic healthcare needs as other children and they benefit from routine health promotion and disease prevention efforts, including immunizations. In some cases, medical therapy may play an important role in treating problem behaviors such as aggression and self-injury, either by treating a coexisting psychiatric or neurologic condition or addressing an underlying medical problem (such as an ear infection or constipation) to alleviate pain or discomfort (Myers & Johnson, 2007; Myers, 2009; Carbone, Farley, & Davis, 2010; Munshi, Gonzalez-Heydrick, Augenstein, & D’Angelo, 2011). Currently, medical therapies are directed at specific symptoms or coexisting conditions rather than the ASD itself. For example, children with ASDs who have seizures or gastrointestinal problems (such as chronic diarrhea or constipation) should be evaluated and treated the same way as any other child with these symptoms would be evaluated and treated.

Educational/Habilitative/Behavioral Interventions 

Easy access to the Internet has resulted in widespread dissemination of both information and misinformation about treatments for ASDs. Fortunately, medical professionals can help manage the care of their patients with ASDs by drawing upon published literature reviews and guidelines to guide families toward evidence-based educational treatments. There is general agreement among well-researched guidelines that educational treatment should begin early and treatment goals should be comprehensive. Treatments should strive to minimize core social, communication, and behavioral deficits, and to maximize self-care, academic independence, and leisure skills, while at the same time decreasing aberrant behaviors that interfere with functioning (Myers & Johnson, 2007; Myers, 2009). Early diagnosis and early intervention are associated with best outcomes for children with ASDs. However, in the United States the average age of identification is still older than four despite the ability to identify ASDs as early as two years of age (Carr & LeBlanc, 2007).

The role of the physician should include guiding families to empirically supported educational and habilitative practices and helping them evaluate the appropriateness of educational services being offered (Myers & Johnson, 2007). Of the many educational methods available for the treatment of ASDs, some methods, such as facilitated communication, have been proven to be ineffective. Others, like auditory integration training, dolphin-assisted therapy, holding therapy, vision therapy, or therapeutic touch lack evidence to show efficacy in treating individuals with ASDs (Myers, 2009). The three general categories of early childhood educational programs most often used, and which differ in basic philosophy, are behavior analytic, developmental, and structured teaching.

There are five decades of controlled studies in university and community settings showing the effectiveness of applied behavior analysis (ABA) based interventions in helping remediate social and language impairments as well as helping children make sustained gains in IQ, academic performance, and adaptive skills, compared to children in control groups (Carr & LeBlanc, 2007; Myers & Johnson, 2007; Munshi, Gonzalez-Heydrick, Augenstein, & D’Angelo, 2011). Early and intensive behavioral interventions (or EIBI) are skills-based treatment approaches based on the science of applied behavior analysis. EIBI program models differ but share a philosophy of starting when children are very young, intensity of treatment (25-40 hours per week), a focus on communication, social, and pre-academic repertoires, and the use of teaching methods derived from the principles of operant conditioning (Carr & LeBlanc, 2007).

Such programs should be individualized and based on assessment (Carr & LeBlanc, 2007). Behavioral therapy can be provided by an early intervention program, a special education program through a school, or by therapists in private practice. Caregivers who are interested in pursuing this treatment approach should be referred to the Behavior Analyst Certification Board to locate a professional qualified to oversee such a program. Programs based on developmental theory include the relationship development intervention (RDI) and developmental, individual-difference, relationship-based model (DIR, also known as “floor-time”). Social deficits are the primary focus of both interventions, and both are popular and relatively widespread in their dissemination; however, no well-controlled studies documenting their effects have been published. Furthermore, the basic developmental theories upon which RDI and DIR are based have not been tested. When considering these interventions, the lack of empirical support should be considered.

Structured teaching is best exemplified by Project TEACCH (Treatment and Education of Autistic and Related Communication-Handicapped Children). The goal of structured teaching is to use strategies like visual supports, individual work stations which minimize distractions, and picture schedules to aid with transitions. These strategies cater to the learning styles of many individuals with autism. The National Research Council considers Project TEACCH a “plausible” intervention; however, there are currently no well-controlled studies of its outcomes.

Many educational treatments for children and adolescents with ASDs, despite their popularity, have not been adequately evaluated, and some do not meet criteria for “well-established” treatments due to a lack of robust experimental designs, independent replications or peer-reviewed published data. This does not necessarily mean that they are ineffective; however, efficacy has not been established and replicated in well-designed clinical studies (Carr & LeBlanc, 2007).

In many communities, an “eclectic” treatment approach is used which combines ABA, structured teaching, and a developmental approach. While there are many differences between the approaches, there are also areas in which they borrow from one another. For example, behavioral programs address social interactions like joint attention and imitation, borrowing from developmental approaches, as well as utilizing visual strategies borrowed from structured teaching; some developmental models and structured teaching approaches use behavioral techniques to meet their teaching goals. However, studies which have compared outcomes from the eclectic approach groups to intensive ABA groups have shown more favorable outcomes in the ABA groups, raising questions about the efficacy of eclectic educational methods.

There is a growing agreement that effective early childhood intervention for children with ASDs should include the following components (Myers & Johnson, 2007):

  • Starting early, even before a definitive diagnosis has been made;
  • Intensive teaching for at least 25 hours a week, all year long;
  • One-on-one and small group instruction, with low student-to-teacher ratios;
  • Parent or caregiver training;
  • Ongoing measurement and data analysis in order to individualize instruction as required;
  • Structured environments, including visual schedules, clear physical boundaries and predictable routines;
  • Strategies to promote generalization and maintenance of learned skills;
  • Assessment based curricula that includes functional communication, social skills, self-management, cognitive and academic skills and functional adaptive skills to increase independence;
  • Reduction of disruptive behavior using strategies that employ functional assessment.

Psychotropic Medications 

Psychotropic MedicationMedications that are used to produce behavioral, emotional, or cognitive changes are known as psychotropic medications. Psychotropic medications have not been proven to correct the core social communication deficits of ASDs, and they obviously do not teach skills. However, medications are sometimes effective for treating associated problem behaviors or coexisting psychiatric conditions that interfere with educational progress, socialization, health and safety, and quality of life (Myers & Johnson, 2007; Myers, 2009; Munshi, Gonzalez-Heydrick, Augenstein, & D’Angelo, 2011).

Examples of problems that might potentially be targeted with psychotropic medications include irritability, aggressive or self-injurious behavior, ADHD symptoms (inattention, distractibility, impulsivity, and hyperactivity), anxiety, mood disorders, and sleep problems. The best evidence of effectiveness of psychotropic medications for specific symptoms in people with ASDs comes from independently replicated studies involving randomized, double-blind, placebo-controlled trial designs, with adequate sample sizes and well-characterized study populations.

The atypical antipsychotic medications, risperidone and aripiprazole, are currently the only medications with U.S. Food and Drug Administration-approved labeling specific to ASDs [for the symptomatic treatment of irritability, including aggressive behavior, deliberate self-injury, and temper tantrums in children and adolescents with autism] (Myers & Johnson, 2007; Myers, 2009; Munshi, Gonzalez-Heydrick, Augenstein, & D’Angelo, 2011). There is also substantial evidence that these medications and others such as methylphenidate, guanfacine, and atomoxetine are helpful for attention-deficit/hyperactivity disorder (ADHD) symptoms in some individuals with ASDs. Some evidence supports the use of the atypical antipsychotics risperidone and aripiprazole and possibly selective serotonin reuptake inhibitors (SSRIs; such as fluoxetine or fluvoxamine) and the anticonvulsant medication, valproate, for repetitive and rigid behaviors associated with ASDs. However, the largest published controlled trial did not demonstrate that cital, a SSRI, was superior to placebo for repetitive behavior associated with autism. Melatonin may be effective for those who have difficulty falling asleep at night.

Functional assessment often reveals that problem behaviors in individuals with disabilities, including ASDs, serve as a way to reach an outcome such as attention, access to a preferred object or activity, or escape from a demand or non-preferred activity. In these cases, behavioral interventions are the most effective treatments, and they should be used before medication is considered (Myers & Johnson, 2007; Carr & LeBlanc, 2007; Myers, 2009; Carbone, Farley, & Davis, 2010; Munshi, Gonzalez-Heydrick, Augenstein, & D’Angelo, 2011). Even when medication is used, behavioral strategies are important, and there is growing evidence that the combination of behavioral intervention with medication results in better outcomes, with lower doses of medication required (Munshi, Gonzalez-Heydrick, Augenstein, & D’Angelo, 2011). In the case of rapid onset or intensification of problem behaviors, children with ASDs should be evaluated by their physicians to rule out potential medical causes, such as a hidden source of pain or discomfort. Middle ear infections, dental abscesses, reflux esophagitis, constipation, medication side effects, menstrual periods, or other medical problems may be identified and treated, and resolution of the underlying medical issue may alleviate the behavioral difficulties.

All medications can have adverse effects, and it is important for healthcare providers to only prescribe medications with which they have sufficient expertise. When the decision is made to start a therapeutic trial of medication, the specific target symptoms or behaviors for the medication should be identified, and a plan should be in place for monitoring of outcomes, including desired effects and adverse, or undesired, effects (Myers & Johnson, 2007; Myers, 2009; Carbone, Farley, & Davis, 2010). This could be done using a tool as simple as a daily behavior data sheet, counting the desired outcomes and associated signs of adverse or undesirable reactions. Once done, this can be compared to a baseline of data obtained before the medication was put in place (Munshi, Gonzalez-heydrick, Augenstein, & D’Angelo, 2011).

PlantsThe treatment reviews noted some of the common pitfalls of treatment with psychotropic medication. For example, although monotherapy (use of a single medication) is desirable, patients with complex problems are sometimes treated with more than one psychotropic medication (polypharmacy). There is very little information available about combinations of medications, and it is critical that physicians have a good understanding of the potential interactions among medications and monitor closely for adverse effects, especially if the individual being treated has limited communication skills and is unable to clearly identify if something is not right. Because of the widespread use of the Internet and the highly-variable quality of available information, it is common for parents and other care providers to be exposed to strong advocacy for treatments that have not been shown to be effective in properly designed scientific studies. It is important for physicians to be aware of the empirical evidence behind the treatments they are considering and strive to ensure that the most safe and effective interventions (based on well-designed scientific studies) are the ones that are selected (Carr & LeBlanc, 2007).

Complementary and Alternative Medicine

Complementary and alternative medicine (CAM) has been defined by the National Center for Complementary and Alternative Medicine (NCCAM) as “a group of diverse medical and health care systems, practices, and products that are not presently considered to be part of conventional medicine” (NCCAM, 2000). The NCCAM has organized CAM practices into five domains: mind-body medicine, manipulative and body-based practices, energy medicine, biologically-based practices, and alternative medical systems, such as homeopathy and naturopathy, which may utilize therapies found in the other four domains.

Many CAM therapies from all 5 of the NCCAM domains have been advocated for the treatment of children with ASDs. The reviews that address CAM therapies state that the vast majority have been inadequately evaluated and cannot be recommended for treatment of ASDs based on the available evidence (Myers & Johnson, 2007; Carr & LeBlanc, 2007; Carbone, Farley & Davis, 2010). Potential risks of CAM treatments include direct toxic effects of biological agents or manipulative techniques, presence of contaminants, interactions with prescribed medications, interference with appropriate nutrition, interruption or postponement of valid therapies, and unwarranted expenditure of time, effort and financial resources (Myers & Johnson, 2007; Carr & LeBlanc, 2007; Carbone, Farley & Davis, 2010).

CAM interventions are sometimes divided into two categories, biological and nonbiological; although this is a misnomer because ultimately, the mechanism of action of any effective intervention would necessarily be through impacting central nervous system biology. The most thoroughly evaluated biological CAM treatment for autism, the hormone secretin, has been thoroughly evaluated and proven to be ineffective. Some of the under-evaluated biological CAM treatments that have been popular in recent years include hyperbaric oxygen, immunoregulatory interventions (such as dietary restrictions, immunoglobulins, and antiviral agents), detoxification therapies (such as chelation), various gastrointestinal treatments (such as digestive enzymes, antifungal agents, probiotics, yeast-free diets, vancomycin, and gluten- and casein-free diet), dietary supplements (large doses of vitamins, magnesium, folic acid), and even stem cell infusions. Examples of non-biological CAM therapies that have waxed and waned in popularity include auditory integration training, behavioral optometry, craniosacral manipulation, dolphin-assisted therapy, and facilitated communication, none of which has been proven to be effective.

The AAP has stated that pediatricians should: (1) critically evaluate the scientific evidence of efficacy and risk of harm of various treatments and convey this information to families, (2) help families understand how to evaluate scientific evidence and recognize unsubstantiated treatments and pseudoscience, and (3) insist that studies that examine CAM treatments be held to the same scientific standards as all clinical research (Myers & Johnson, 2007). This requires open lines of communication and families should not be discouraged from sharing information about any CAM treatments that they may be considering (Myers & Johnson, 2007). According to a recent survey, only 36-62% of caregivers who used CAM therapies for their children with ASD shared that information with their child’s primary care physician, yet they indicated that that they wanted more information on CAM therapies from physicians (Myers & Johnson, 2007). If families are reluctant to disclose CAM treatments to their child’s physicians, the physicians may inadvertently prescribe medication that has a potential interaction with the CAM treatment. When speaking with amilies, physicians should encourage families to seek additional information if:

  • The treatment is based on overly simplified scientific theories;
  • It is claimed that the therapy is effective for multiple different unrelated conditions or symptoms;
  • It is claimed that children will respond dramatically and some will be cured;
  • Support for the treatment is based on case reports and anecdotes rather than carefully designed studies;
  • There is a lack of reference to peer-reviewed -scientific literature or, denial of the need for controlled studies, or the existing literature directly contradicts the claims of proponents of the CAM treatment;
  • The treatment is said to have no potential or reported adverse effects.


All treatments should be based on sound, plausible theoretical constructs and objective scientific evidence of efficacy. When treatments are evaluated, well-designed, and appropriately controlled studies using rigorous methodologies are required to prove that the observed effects are attributable to the intervention being studied. In the published scientific literature, the evidence is strongest for behavior analytic strategies for both teaching new skills and reducing problem behaviors in children with ASDs. In some cases, when serious problem behaviors remain after a function-based approach has been utilized, a trial of psychotropic medication may be warranted to target certain specific symptoms, usually in conjunction with behavioral interventions. By providing their patients with ASDs with ongoing high-quality medical care and guiding them to effective interventions, healthcare providers can help to maximize important outcomes including functional independence and quality of life.


Carbone, P. S., Farley, M., & Davis. T. (2010). Primary care for children with autism. American Family Physician, 81, 453-460.

Carr, J. E., & LeBlanc, L. A. (2007). Autism spectrum disorders in early childhood: An overview for practicing physicians. Primary Care: Clinics in Office Practice, 34, 343-359.

Golnik, A., Ireland, M., & Borowsky, I. W. (2009). Medical homes for children with autism: A physician survey. Pediatrics, 123, 966-971.

Myers, S. M., Johnson, C. P., & American Academy of Pediatrics Council on Children With Disabilities (2007). Management of children with autism spectrum disorders. Pediatrics, 120, 1162-1182.

Munshi, K. R., Gonzalez-Heydrich, J., Augenstein, T., & D’Angelo, E. J. (2011). Evidence-based treatment approach to autism spectrum disorders. Pediatric Annals, 40, 569-574.

Myers, S. M. (2009). Management of autism spectrum disorders in primary care. Pediatric Annals, 38, 42-49.

National Center for Complementary and Alternative Medicine (2000). Expanding horizons of healthcare: five year strategic plan 2001-2005. Washington, D.C.: U.S. Department of Health and Human Services.

Please use the following format to cite this article:

Halliday, P., Houston, Z., Kinney, E., & Myers, S. M. (2012). Autism treatment reviews for physicians: The take-home messages. Science in Autism Treatment, 9(2), 1-6