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Warren Z, Veenstra-VanderWeele J, Stone W, et al. Therapies for Children With Autism Spectrum Disorders. Rockville (MD): Agency for Healthcare Research and Quality (US); 2011 Apr. (Comparative Effectiveness Reviews, No. 26.)

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Therapies for Children With Autism Spectrum Disorders.

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Introduction

Need for Evidence for Treatment of Autism Spectrum Disorders in Children

Autism spectrum disorders (ASDs) are common neurodevelopmental disorders, with an estimated prevalence of one in 110 children in the United States.1 ASDs have multiple etiologies involving both genetic and environmental risk factors. Among the environmental risk factors that may contribute to ASD risk are advanced parental age2 and prematurity.3 Disorders within the autism spectrum include Autistic Disorder, Asperger syndrome, and Pervasive Developmental Disorder, Not Otherwise Specified (PDD-NOS).

Individuals with ASDs have significant impairments in social interaction, behavior, and communication.4 These impairments include a lack of reciprocal social interaction and joint attention (i.e., the ability of the child to use nonverbal means such as pointing to direct others’ attention to something in which the child is interested); dysfunctional or absent communication and language skills; lack of spontaneous or pretend play; intense preoccupation with particular concepts or things; and repetitive behaviors or movements.5–7 Children with ASDs may also have impaired cognitive skills and sensory perception.1,4 ASDs are often accompanied by other conditions such as seizure disorders, hyperactivity, and anxiety.6,7 The expression and severity of symptoms of ASDs differ widely, and treatments include a range of behavioral, psychosocial, educational, medical, and complementary approaches8–10 that vary by a child’s age and developmental status.

The goals of treatment for ASDs focus on improving core deficits in communication, social interactions, or restricted behaviors, as changing these fundamental deficits may help children develop greater functional skills and independence.6 In addition, comprehensive treatment programs developed in the 1980s target behaviors and development more broadly instead of focusing on a specific behavior of interest.11 Positive effects seen with these approaches in terms of cognition and language have led to the suggestion that beginning intensive therapy (25 to 30 hours/week) at an earlier age may lead to greater improvements.11–13

Treatment is frequently complicated by emergent symptoms such as irritability and other co-morbid conditions that may warrant targeted treatment. There is no cure for ASDs and no global consensus on which intervention strategy is most effective.13,14 Chronic management is often required to maximize functional independence and quality of life by minimizing the core autism spectrum disorder features, facilitating development and learning, promoting socialization, reducing maladaptive behaviors, and educating and supporting families. Individual goals for treatment vary for different children and may include combinations of medical and related therapies, behavioral therapies, educational therapies, allied health therapies and complementary and alternative medicine (CAM) therapies.

The following sections briefly describe interventions discussed in the literature meeting our criteria for this review. Additional interventions for children with ASDs that did not meet criteria for our review are described in recent systematic and narrative reviews.8–10,14–20

Behavioral Interventions

Studies of behavioral interventions are addressed in this review in the broad subcategories of early intensive behavioral and developmental interventions; social skills interventions; focal play-based or interaction-based interventions; interventions focused on associated behaviors; and a small group of other behavioral interventions assessing a variety of targets. Table 1 outlines key features of behavioral interventions addressed in the report.

Table 1. Description of behavioral interventions addressed in the report.

Table 1

Description of behavioral interventions addressed in the report.

Early intensive behavioral and developmental interventions. In 1987, Ivar Lovaas published findings21 on a subgroup of children who demonstrated improvements in cognitive abilities and educational placement in response to intensive intervention based on the principles of applied behavior analysis (ABA). As a result, ASDs were re-conceptualized from largely untreatable disorders,22 to disorders marked by plasticity and heterogeneity, where there was hope for “recovery” and better outcomes for children receiving appropriate intervention. Subsequent research focused on social communication and behavioral impairments and used both highly structured approaches and natural/developmental approaches that deliver intervention within natural contexts (Floortime, the Social Communication Emotional Regulation Transactional Support model), some of which integrate approaches (Early Start Denver Model [ESDM]).

We adopted a similar approach to the operationalization of this category as Rogers and Vismara12 in their review of “comprehensive” evidence-based treatments for early ASDs. Interventions in this category all have their basis in or draw from principles of applied behavior analysis (ABA), with differences in methods and setting. ABA is an umbrella term describing principles and techniques used in the assessment, treatment and prevention of challenging behaviors and the promotion of new desired behaviors. The goal of ABA is to teach new skills, promote generalization of these skills, and reduce challenging behaviors with systematic reinforcement. The principles and techniques of ABA existed for decades prior to specific application and study within ASDs.

We include in this category two intensive manualized (i.e., have published treatment manuals to facilitate replication) interventions: the UCLA/Lovaas model and the ESDM. These two interventions have several key differences in their theoretical frameworks and implementation, although they share substantial similarity in the frequent use of high intensity (many hours per week, one-on-one) instruction utilizing ABA techniques. They are described together here because of these similarities. We note, however, that the UCLA/Lovaas method relies heavily on one-on-one therapy sessions during which a trained therapist uses discrete trial teaching with a child to practice target skills, while the ESDM blends ABA principles with developmental and relationship-based approaches for young children.

We review ESDM, which focuses specifically on younger children, under Key Question 7 in Chapter 3 (Results), but we integrate our discussion of UCLA/Lovaas-based approaches and ESDM in Chapter 4 (Discussion) of this report, given the model’s similarity in underlying methodology.

The other treatment approaches in this section also incorporate ABA principles, and may be intensive in nature, but often have not been manualized. We have classified these approaches broadly as “UCLA/Lovaas-based” given their similarity in approach to the Lovaas model. A third particular set of interventions included here are those using the principles of ABA to focus on key pivotal behaviors rather than global improvements. These approaches emphasize parent training as a modality for treatment delivery (e.g., Pivotal Response Training, Hanen More than Words, social pragmatic intervention, etc.) and may focus on specific behaviors such as initiating or organizing activity or on core social communication skills. Because they emphasize early training of parents of young children, they are reviewed here.

Interventions intended primarily to be administered in educational settings, or studies for which the educational arm was most clearly categorized are included in the section on educational interventions.

Social skills interventions. Difficulty with social engagement has been reported since the earliest descriptions of ASDs23 and is the unique and essential aspect of ASDs that distinguishes them from other childhood disorders.4,24 The social impairment seen in ASDs takes many forms and can vary greatly from one child to the next. Therefore interventions focused on enhancing social behavior and competence in children with ASDs should be targeted with respect to the child’s age, developmental level, and peer group. Interventions for very young children may focus on teaching parents how to engage their child and encourage back-and-forth play. At preschool and early childhood levels, interventions may focus on playing with peers, understanding emotions, and learning the basics of turn-taking and initiating and responding to social interactions. In the later elementary years and into adolescence, interventions may focus more on teaching perspective-taking and social problem-solving and understanding peer group social norms. Given that social impairments are a core feature of ASDs, numerous skill-based approaches have tried to address this vulnerability through direct instruction within individual (e.g., Social Stories) or group (e.g., Skillstreaming, Children’s Friendship Training) formats. Other approaches aim to foster the development of social skills solely through structured interactions with peers (e.g., Lego therapy).

Play-/interaction-based interventions. These interventions use interactions between children and adults (either parents or researchers) to improve outcomes such as imitation or joint attention skills or the ability of the child to engage in symbolic play. They include teaching parents how to interact differently with their children within daily routines and interactions, often using standard behavior management strategies. They also include foci on generic day-to-day interactions outside of the family (Table 1).

Behavioral interventions focused on associated behaviors. Several behavioral interventions target symptoms like anger and anxiety, which are often present with ASDs (Table 1). CBT is a common treatment for anxiety symptoms in otherwise typically developing children and has more recently been adapted for and applied to children with ASDs,25–28 particularly children with higher IQs. The approach focuses on teaching cognitive skills and relaxation strategies, helping children recognize anxious feelings, and providing them with behavioral exposures in which to practice coping skills in the face of anxiety-provoking situations. The goal of treatment is to reduce generalized and specific anxiety symptoms over time.29

Challenging behaviors, such as noncompliance, tantrums, self-injury, and aggression, are also common, and parent training protocols are used to teach behavior prevention, intervention, and management strategies. Once trained, parents can act as “co-therapists,” shaping behavior to reduce negative behaviors in daily life. Parent training interventions also often have secondary targets of improving parental feelings of self-efficacy and decreasing parental stress.

Educational Interventions

Most children with ASD receive at least some treatment in an educational setting, beginning with preschool. For children with ASDs, educational interventions often aim at promoting personal independence and social responsibility.13 Educational interventions have focused both on traditional areas of academic progression/achievement, as well as on addressing social, cognitive, and behavioral issues in classrooms or through specialized instruction. These interventions include the Treatment and Education of Autistic and Communication related handicapped CHildren (TEACCH) program,30 early intervention center- or classroom-based instruction, and computer-based approaches.

Originally developed in the 1970s at the University of North Carolina at Chapel Hill, TEACCH involves structured teaching and therapeutic techniques that encompass a “whole life” approach. Instruction is based on the idea that individuals on the autism spectrum have difficulty in perception and understanding; the intervention therefore relies heavily on visual supports like a picture schedule and arranging the physical environment to support the individual.

Classroom- and center-based approaches include a blend of teaching strategies that rely on principles and techniques of ABA including reinforcement-based procedures such incidental teaching, discrete trial training, and Pivotal Response Training (Table 2). Other interventions such as TEACCH and language development interventions may also be incorporated in center-based treatment. Computer-based programs use technology to deliver behaviorally-based teaching in areas like language acquisition and reading skills.

Table 2. Description of educational interventions addressed in the report.

Table 2

Description of educational interventions addressed in the report.

Medical and Related Interventions

Interventions in this category are those in which a medication, supplement, or other substance is administered to a child with ASDs. Medical treatments for symptoms of ASDs comprise a variety of pharmacologic agents including antipsychotics, psychostimulants, and serotonin reuptake inhibitors (SRIs) that are generally intended to treat common comorbidities of ASDs. Modalities such as therapeutic diets, supplements, hormonal supplements, immunoglobulin, hyperbaric oxygen, and chelating agents also have been employed to treat ASDs symptoms (Table 3).

Table 3. Description of medical and related interventions addressed in the report.

Table 3

Description of medical and related interventions addressed in the report.

Antipsychotics. Antipsychotic medications generally act on the dopamine system, which is involved in regulating emotions, and potentially decrease behavioral outbursts.31,32 Whereas the older typical antipsychotic drugs act primarily on the dopamine system, newer atypical antipsychotic drugs interact with a variety of brain chemicals, such as serotonin.33,34 Although these medications were developed to treat psychosis, they have also been studied extensively for the treatment of other disorders, including mood disorders,35,36 obsessive compulsive disorder,37 and tic disorders.38

Among typical antipsychotics, haloperidol has been used since the 1980s to treat challenging behavior in children with ASDs.39 More recently, risperidone, an atypical antipsychotic that acts on both dopamine D2 and serotonin 5-HT2A receptors,34 was the first medication to receive Food and Drug Administration (FDA) approval for the treatment of irritability in children with ASDs. Aripiprazole, which has a more complex mechanism of action,34 also recently received FDA approval for irritability in children with ASDs.

Serotonin reuptake inhibitors. Serotonin is associated with mood elevation and reduced anxiety symptoms. SRIs block the serotonin transporter so that increased serotonin stays in the system.40 SRIs have come into wide use for the treatment of depression and anxiety and are some of the most commonly prescribed medications for children with ASDs.41–43 SRIs were tested for use in children with ASDs44,45 after it was noted that 30 percent of this population had elevated blood serotonin.46 Early RCTs of both comipramine44,47 and fluvoxamine48 showed improvements in multiple behaviors. Open label trials of selective SRIs in the 1990s provided further support for the idea that this class may benefit some children with ASDs, but also revealed common side effects including hyperactivity and decreased sleep.45,49 Most recent clinical trials in children with ASDs have focused on changes in repetitive behaviors with SRIs with longer half-lives, including fluoxetine, and citalopram or escitalopram, one of two component drugs contained in citalopram.49 Longer half lives can be associated with a more stable blood level over time, reducing susceptibility to the effects of missed doses.

Stimulants and other medications for hyperactivity. Psychostimulants treat hyperactivity and inattention in patients diagnosed with attention deficit hyperactivity disorder (ADHD). Stimulants studied in ASDs include methylphenidate (MPH), amphetamine, and dextroamphetamine (Table 3). All stimulant medications inhibit dopamine uptake from the synapse; amphetamine and dextroamphetamine also cause release of dopamine into the synapse.

Other medications studied for the treatment of ADHD have also been studied for the treatment of hyperactivity in ASDs, including atomoxetine, which inhibits norepinephrine reuptake from the synapse50–52 Guanfacine, a norepinephrine receptor alpha-2a agonist that was originally used for the treatment of high blood pressure, has also been studied for use in ASDs.53,54

Secretin. Secretin is a gastrointestinal polypeptide used to treat peptic ulcers55,56 and in the evaluation of pancreatic function. Animal studies have suggested that secretin affects the central nervous system.57,58 Interest in secretin for the treatment of symptoms of ASDs derived from a report of 3 children with ASDs given synthetic intravenous secretin during a routine endoscopy evaluation for gastrointestinal problems.59 The report noted social, cognitive and communicative gains after the first infusion and after a second infusion given weeks later.

Other medical interventions. Additional studies in the medical literature addressed medical therapies for sleep and gastrointestinal dysfunction as well as the use of hyperbaric oxygen, specialized diets, supplements, and other agents explored to address symptoms of ASDs (Table 3).

Management of sleep issues. Children with ASDs commonly sleep little or fitfully, creating stress for them and their families.60 Melatonin, a hormone associated with regulating circadian rhythms,61 and iron supplementation62 have been studied to improve disordered sleep in children with ASDs.

Management of gastrointestinal symptoms. Gastrointestinal (GI) symptoms may or may not have an increased prevalence in ASDs, with some evidence supporting increased difficulty with constipation but not other GI symptoms. Oral immunoglobulin has been considered for its potential utility in addressing GI symptoms in ASDs.63,64

Dietary supplements and restrictive diets for core symptoms of ASDs. A range of dietary supplements with potential neurologic effects show some benefit in other chronic neurological conditions and have been assessed for use in treatment of ASDs (Table 3). Magnesium-vitamin B6 and two amino acid-related compounds, L-carnosine and dimethylglycine, show some potential anticonvulsant activity in observational studies65–67 and have been tried in ASDs for potential positive behavioral effects. Reduced levels of free polyunsaturated fatty acids (PUFAs) have been reported in a range of neuropsychiatric conditions including ASDs.68,69 Supplementation with agents containing PUFAs, such as fish oil and evening primrose supplements, have been considered for their possible benefits in ASDs.

Some observational data suggest benefit of a ketogenic diet, a high fat, low carbohydrate diet, in some patients with epilepsy and seizures refractory to standard therapy,70 and this strategy has also been explored in ASDs.

Other. Amantadine, an antiviral agent,71 is thought by some to have neurologic effects that may positively affect behavior problems in ASDs. Similarly, the putative cognitive enhancer piracetam has been used in the treatment of dementia72 and has been considered for potential cognitive benefit in ASDs. Hyperbaric therapy, in which oxygen is administered in special chambers that maintain a higher air pressure, has shown possible effects in other chronic neurologic conditions73,74 and has also undergone preliminary exploration in ASDs. Cholinesterase inhibitors, such as donepezil hydrochloride and rivastigmine tartrate, inhibit an enzyme that breaks down the neurotransmitter acetylcholine; these drugs have been used to prevent further cognitive decline in Alzheimer’s disease75 and have similarly been studied for possible benefit in ASDs.

Dimercaptosuccinic acid (DMSA), used in chelation therapy, was approved by the FDA to treat lead poisoning,76 and may have similar activity against other heavy metals such as mercury.77 While no clear evidence suggests that mercury or ability to remove mercury from the body is involved in ASDs in any way, investigators have evaluated the ability of DMSA to affect ASD symptoms based upon existing off-label use in some children with autism.78

Pentoxifylline is typically used to improve blood flow in individuals with peripheral arterial disease and also inhibits the production of tumor necrosis factor, suggested as playing a role in neurological disorders; the drug also acts on the release and uptake of serotonin and dopamine and was suggested for use in autism after improvements in autistic behavior were noted in a child with an ASD receiving the medication for suspected post-traumatic brain damage.79

Allied Health Interventions

Several allied health interventions address core symptoms of ASDs as well as associated difficulties and deficits. We broadly divided allied health studies into three categories: those focused on language, sensory or auditory integration techniques including music therapy, and other approaches (including horseback riding and occupational therapy) (Table 4).

Table 4. Description of allied health interventions addressed in the report.

Table 4

Description of allied health interventions addressed in the report.

Speech and language development. As a core feature of ASDs, communication difficulties are an important target of treatment. Frequently, verbal communication is the target of treatment, but establishing functional nonverbal communication for children who do not speak also can be the primary goal. Two approaches to increasing speech and language were identified: Responsive Education and Prelinguistic Milieu Teaching (RPMT), and the Picture Exchange Communication System (PECS). RPMT is a two-component system aimed at both parents and children. It is play-based, and encourages gestural, non-word vocal, gaze use, and later, word use for intentional communication around play, including for turn-taking, requesting and commenting.80,81 Parents are taught methods of playing with their children that are thought to facilitate communication, in particular to use linguistic mapping, in which they put into words a child’s immediately preceding nonverbal message. Once prelinguistic communication is achieved, Milieu Language Teaching is incorporated, in which prompts are used to encourage verbal imitation and questions are asked to evoke spoken communication.

PECS uses pictures or symbols to teach children to communicate spontaneously.82 The approach relies on behavioral techniques, especially reinforcement techniques. Providers prompt children to pick up and exchange a symbol/picture for a desired object. The process may include fading those prompts until competency is achieved. PECS can be used while intensive work to increase speech is in progress, and may provide an interim or additional means of communication. PECS relies on immediate positive reinforcement with the child obtaining the desired object upon successfully indicating his desire for it with the corresponding picture.

Sensory and auditory integration and music therapy. Although sensory sensitivity and dysfunction are not core features of ASDs, they are frequently described as challenges for some children with ASDs.83,84 Sensory Integration (SI) is specialized occupational therapy based on the premise that the brain’s response to basic sensory input must be normalized before higher-order processes can be addressed.85 The approach anticipates that a child who is better able to process, modulate, and integrate sensory information will then be better able to acquire higher-order skills.16 Auditory integration training (AIT) relates specifically to auditory perception. In AIT, children are repeatedly presented with modulated music according to specific protocols with a therapeutic goal of improving auditory processing, lessening auditory hypersensitivities, and increasing concentration.86,87 Finally, music therapy is at times employed with children with ASDs, hinging on speculation that children engage more with music than with speech. This treatment method is improvisational and unstructured, and practitioners purport that it can improve both verbal and nonverbal communication skills including joint attention abilities.88,89

Additional allied health interventions. A number of additional interventions including other occupational therapy techniques, horseback riding therapy, assistive devices to facilitate reading or motor skills, and movement therapy are also considered allied health approaches and may target difficulties in sensory processing as well as language and adaptive behavior.

Complementary and Alternative Medicine (CAM) Interventions

Acupuncture is an ancient Chinese medical system based on the balance of energy flows in which imbalance is thought to result in disease (Table 5). Acupuncture therapy aims to manipulate these energies through the insertion of fine needles at highly specific points related to energy flow to specific organs. Like acupuncture, massage therapy is thought to exert effects on the energy field of the body and has been used in ASDs to decrease touch aversion and improve autistic behaviors.90

Table 5. Description of CAM interventions addressed in the report.

Table 5

Description of CAM interventions addressed in the report.

Importance of This Review

While advances have been made in early diagnosis and the promotion of early intervention for ASDs,5,91,92 few current sources for the comparative effectiveness of treatment interventions exist. Clinicians and families are left to choose among the interventions in part based on what is available to them, what is covered by commercial insurance or Medicaid, or what they can afford out of pocket. Sometimes, a clinical course of action is based on the most common or popular treatments at a given time. Many therapies are not covered by insurance, and a primary reason for insurance denial from private insurers is that no evidence-based resources for this condition exist. Additionally, insurers may find it confusing to distinguish among therapies or to sort out which approaches have an evidence base and which are still experimental.

The delivery and organization of care for ASDs therefore tends to be fragmented, with pieces scattered about in the primary care, school, and specialty clinical settings, making it especially important for families and caregivers to have clear information on effectiveness of treatment components. Treatment outcomes may be highly variable across diagnostic groups and developmental stages and in the presence or absence of co-morbidities. Family context and the child’s home and school environment may also alter the effectiveness of treatment. Therapeutic approaches should therefore be tailored to an individual child to the extent possible to optimize effectiveness.92,93

Previous reviews of the literature have noted limited quality and consistency in studies assessing ASDs therapies,9,10,12,94–96 and an umbrella review found methodological weaknesses in systematic reviews of psychosocial interventions.8 While controlled trials seem to be increasing, much research is observational, generally with small sample sizes, limited followup, and limited discussion of the durability of treatment gains once active therapy ends. As the prevalence of ASDs has increased, the available treatment options have also increased, but evidence overall for many interventions can only be considered preliminary. The need for synthesized research that evaluates the evidence base for various treatments and identifies gaps in the current literature that may drive the research agenda is great.

Scope of This Evidence Report

Evidence reviews of therapeutics seek to identify and systematically summarize objective information about the evidence related to the:

  • Effectiveness of specific, well-defined treatments
  • Relative benefit of one treatment over another
  • Common side effects and serious risks of a treatment
  • Whether individual characteristics help predict who will benefit or be harmed

Key Questions and Analytic Framework

Key Questions

We focused this review on treatments for children ages 2–12 with ASDs and children younger than age 2 at risk of a diagnosis of ASD. We have synthesized evidence in the published literature to address these key questions (KQ):

KQ1. Among children ages 2–12 with ASDs, what are the short and long-term effects of available behavioral, educational, family, medical, allied health, or CAM treatment approaches? Specifically,

KQ1a.

What are the effects on core symptoms (e.g., social deficits, communication deficits and repetitive behaviors), in the short term (≤6 months)?

KQ1b.

What are the effects on commonly associated symptoms (e.g., motor, sensory, medical, mood/anxiety, irritability, and hyperactivity) in the short term (≤6 months)?

KQ1c.

What are the longer-term effects (>6 months) on core symptoms (e.g., social deficits, communication deficits and repetitive behaviors)?

KQ1d.

What are the longer-term effects (>6 months) on commonly associated symptoms (e.g., motor, sensory, medical, mood/anxiety, irritability, and hyperactivity)?

KQ2. Among children ages 2–12, what are the modifiers of outcome for different treatments or approaches?

KQ2a.

Is the effectiveness of the therapies reviewed affected by the frequency, duration, and intensity of the intervention?

KQ2b.

Is the effectiveness of the therapies reviewed affected by the training and/or experience of the individual providing the therapy?

KQ2c.

What characteristics, if any, of the child modify the effectiveness of the therapies reviewed?

KQ2d.

What characteristics, if any, of the family modify the effectiveness of the therapies reviewed?

KQ3. Are there any identifiable changes early in the treatment phase that predict treatment outcomes?

KQ4. What is the evidence that effects measured at the end of the treatment phase predict long-term functional outcomes?

KQ5. What is the evidence that specific intervention effects measured in the treatment context generalize to other contexts (e.g., people, places, materials)?

KQ6. What evidence supports specific components of treatment as driving outcomes, either within a single treatment or across treatments?

KQ7. What evidence supports the use of a specific treatment approach in children under the age of two who are at high risk of developing autism based upon behavioral, medical, or genetic risk factors?

Analytic Framework for Therapies for Children With ASDs

The analytic framework in Figure 1 summarizes the process by which families of children with ASDs make and modify treatment choices. Treatment choices are affected by many factors that relate to the care available. Treatment effectiveness may also be affected by factors related to the child (e.g., age, IQ) or the context of care. Ideally, treatment effects are seen both in the short term in clinical changes and in longer term or functional outcomes. Eventual outcomes of interest include adaptive independence appropriate to the abilities of the specific child, psychological well-being, appropriate academic engagement, and psychosocial adaptation.

Figure 1 is the analytic framework to guide the literature review of autism spectrum disorders (ASDs). It summarizes the process by which families of children with ASDs make and modify treatment choices. Circled numbers indicate the Key Questions (KQs), and their placement indicates the points in the treatment process where they are likely to arise. Treatments for ASD include behavioral, educational, medical, allied health, and complementary and alternative medicine interventions (KQ1). The populations of interest are patients 2–12 years diagnosed with ASD (for KQs 1–6) and patients under 2 at risk for diagnosis of ASD (KQ7). Therapeutic modalities are chosen (KQ6). KQ3 involves identifiable changes early in the treatment process. KQ4 involves the relationship between targeted outcomes in the treatment setting and functional outcomes outside the treatment setting. KQ5 involves generalization of interventions to other contexts. KQ2, modifiers of outcome, is related to individual and therapeutic characteristics. Target outcomes in the treatment setting are language communication, academic skill development, maladaptive behaviors, distress, adaptive skills development, and social skills/interaction. Functional outcomes outside the treatment setting are adaptive independence, academic engagement/attainment, psychological well-being, and psychosocial adaptation. Long-term outcomes include quality of life, social integration, and appropriate level of independence. Harms are also considered.

Figure 1

Analytic framework for therapies for children with ASDs.

Organization of This Evidence Report

Chapter 2 describes our methods including our search strategy, inclusion and exclusion criteria, approach to review of abstracts and full publications, and our method for extraction of data into the evidence table and compiling evidence. We also describe the approach to grading of the quality of the literature and to evaluating the strength of the body of evidence.

Chapter 3 presents the results of the evidence report, synthesizing the findings by category of intervention. We report the number and type of studies identified and we differentiate between total numbers of publications and unique studies to bring into focus the number of duplicate publications in this literature in which multiple publications are derived from the same study population. We attempted to emphasize the effect of treatment on the core symptoms and commonly associated co-morbidities of ASDs. We integrate discussion of sub-questions within that for each key question because there was not adequate distinction in the literature to address them separately.

Chapter 4 discusses the results in Chapter 3 and expands on methodologic considerations relevant to each key question. We also outline the current state of the literature and challenges for future research on ASDs.

The report includes a number of appendixes to provide further detail on our methods and the studies assessed. The appendixes are as follows:

A list of abbreviations and acronyms used in the report follows the References section.

Technical Expert Panel (TEP)

We identified technical experts on the topic of ASDs in the fields of developmental disabilities, psychiatry, psychology, occupational therapy and educational research to provide assistance during the project. The TEP contributed to the Agency for Healthcare Research and Quality’s (AHRQ) broader goals of (1) creating and maintaining science partnerships as well as public-private partnerships and (2) meeting the needs of an array of potential customers and users of its products. Thus, the TEP was both an additional resource and a sounding board during the project. The TEP included eight members serving as technical or clinical experts, including representatives from our partner organizations (the nominators of the topic), the Medicaid Medical Directors and Autism Speaks. To ensure robust, scientifically relevant work, we called on the TEP to provide reactions to work in progress or possibly overlooked areas of research. TEP members participated in conference calls and discussions through e-mail to:

  • Refine the analytic framework and key questions at the beginning of the project;
  • Discuss the preliminary assessment of the literature, including inclusion/exclusion criteria;
  • Provide input on assessing the quality of the literature.

Because of their extensive knowledge of the literature, including numerous articles authored by TEP members themselves, and their active involvement in professional societies and as practitioners in the field, we also asked TEP members to participate in the external peer review of the draft report.

Uses of This Report

This evidence report addresses the key questions outlined above using methods described in Chapter 2 to conduct a systematic review of published literature. We anticipate that the report will be of value to clinicians who treat children with ASDs, including general pediatricians, developmental and behavioral pediatricians, neurodevelopmentalists, child neurologists, psychologists, psychiatrists and behavioral experts. In addition, this review will be of use to the National Institutes of Health, Centers for Disease Control and Prevention, Centers for Medicare & Medicaid Services, and the Health Resources and Services Administration–all of which have offices or bureaus devoted to child health issues. This report can bring practitioners up to date about the current state of evidence, and it provides an assessment of the quality of studies that aim to determine the outcomes of therapeutic options for the management of ASDs. It will be of interest to families affected by ASDs and the general public because of the high prevalence of ASDs and the recurring need for families and their health care providers to make the best possible decisions among numerous options. We also anticipate it will be of use to private sector organizations concerned with ASDs, as they work to guide research priorities and educate communities about ASDs.

Researchers can obtain a concise analysis of the current state of knowledge in this field. They will be poised to pursue further investigations that are needed to understand best approaches to therapies for children with ASDs.

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