Treatment of ASD

The manifestation and severity of symptoms of ASD differ widely, and treatments include a range of behavioral, psychosocial, educational, medical, and complementary approaches^3–6 that vary by a child’s age and developmental status. The goals of treatment for ASD typically 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.⁷ Treatment frequently is complicated by symptoms or comorbidities that may warrant targeted intervention (e.g., significant challenging behavior, attention and hyperactivity concerns, depression, anxiety). There is no cure for ASD and no global consensus on which intervention is most effective.^{8, 9} Individual goals for treatment vary for different children and may include combinations of behavioral therapies, educational therapies, medical and related therapies, approaches targeting sensory issues, and allied health therapies; parents may also pursue complementary and alternative medicine therapies.

The antipsychotics risperidone (Risperdal) and aripiprazole (Abilify) have been specifically approved by the U.S. Food and Drug Administration (FDA) for treatment of the comorbid symptoms of irritability and challenging behaviors in ASD. No medications have been approved specifically to treat core ASD symptoms such as communication impairments. Many medications are used off–label to manage behavioral symptoms such as anxiety and hyperactivity. In addition, other treatments such as nutritional supplements or devices such as hyperbaric oxygen chambers have been used to treat symptoms of ASD, though neither supplements or hyperbaric oxygen have been approved by the FDA for ASD treatment.¹⁰

Scope of Review

This review updates findings reported in the 2011 Agency for Healthcare Research and Quality (AHRQ) review of Therapies for Children with ASD¹¹ with a focus on studies of medical interventions. We defined medical interventions broadly as interventions involving the administration of external substances to the body or use of external, nonbehavioral procedures to treat symptoms of ASD, which includes pharmacologic agents, diet therapies, vitamins and supplements, chelating agents, electroconvulsive therapy, transcranial magnetic stimulation and hyperbaric oxygen, among other modalities. We used this broad definition, developed with input from our clinical experts, in order to capture the landscape of medically-related interventions used to treat children with ASD.

We focused the review on children between 2 and 12 years of age. We chose to limit the age range to this span because a) diagnosis of ASD earlier than age 2 is less established and b) adolescents likely have substantially different challenges and would warrant different interventions than children in the preschool, elementary, and middle school age groups.

We integrate syntheses of comparative studies evaluating medical interventions addressed in our 2011 review of therapies for children with ASD¹¹ if they addressed an agent evaluated in a study identified for the current review. To ensure comprehensive coverage of the medical literature in the current update, we also included studies that had originally been excluded in the 2011 review because of sample size. We set a lower sample size inclusion criterion in the current update.

A companion review updating findings related to interventions targeting sensory challenges is available on the AHRQ Effective Health Care Web site.

Key Questions

We developed Key Questions (KQs) in consultation with Key Informants and the Task Order Officer. KQs were posted for review to the AHRQ Effective Health Care Web site.

KQs were as follows:

KQ1.

Among children ages 2–12 with ASD, what is the comparative effectiveness (benefits and harms) of medical treatments?

1. What are the effects on core symptoms (e.g., deficits in social communication and interaction; restricted, repetitive patterns of behavior, interests, or activities including hyper- or hypo- reactivity to sensory input or unusual interest in sensory aspects of the environment) in the short term (<6 months)?
2. What are the effects on commonly associated symptoms (e.g., motor, medical, mood/anxiety, irritability, and hyperactivity) in the short term (<6 months)?
3. What are the longer term effects (≥6 months) on core symptoms (e.g., social deficits, communication deficits, and repetitive behaviors)?
4. What are the longer term effects (≥6 months) on commonly associated symptoms (e.g., motor, medical, mood/anxiety, irritability, and hyperactivity)?

KQ2.

Among children ages 2–12 with ASD, what are the modifiers of outcome for different medical treatments?

1. Is the effectiveness of the therapies reviewed affected by the frequency, duration, intensity, or dose of the intervention?
2. Is the effectiveness of the therapies reviewed affected by co-interventions or prior treatment, or the training and/or experience of the individual providing the therapy?
3. What characteristics (e.g., age, symptom severity), if any, of the child modify the effectiveness of the therapies reviewed?
4. What characteristics, if any, of the family modify the effectiveness of the therapies reviewed?

KQ3.

What is the time to effect of medical interventions?

KQ4.

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

KQ5.

Is the effectiveness of medical interventions maintained across environments or contexts (e.g., people, places, materials)?

KQ6.

What evidence supports specific components of treatment with medical interventions as driving outcomes, either within a single treatment or across treatments?

Table 1 outlines population, intervention, comparator, outcomes, timing, and setting (PICOTS) characteristics for each KQ.

Table 1

PICOTS characteristics.