The role of medication in autism spectrum disorder

Efforts toward early identification and treatment are an important facet of the public health work in autism spectrum disorder (ASD).

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The prevalence of ASD is rising. With the most recent estimate from the Centers from Disease Control and Prevention of 1 in 59* children aged 8 years,¹ it is important for pediatric health care providers to have an understanding of current recommendations for treatment so they can counsel and guide affected families. ASD is a heterogeneous condition, so this article seeks to touch on broad principles, recognizing that clinicians must take into account the full clinical picture of each individual and family.

It is important to acknowledge that while there is no cure for ASD, there are treatment modalities that have an evidence base for addressing specific areas that may be impaired in children with autism. While it is beyond the scope of this article to review all of the potential areas of intervention in children with ASD, it is important to be keep in mind a few important principles.

1. The best evidenced treatment for addressing challenging and problematic behavior as well as improving a host of outcomes in children with ASD is itself behavioral in nature. These treatments are based on the principles of applied behavioral analysis,² an educational and therapeutic approach which involves looking at antecedents and consequences of behaviors. This approach also looks to shape, motivate, and reinforce functional behaviors while discouraging harmful and disruptive ones.

2. Because communication often is impaired in children with ASD, providers always should investigate for possible medical causes of pain or discomfort that might explain sudden behavior change, as well as environmental changes that could be involved.

3. The overarching principle of psychopharmacology in ASD is to start low, and go slow – because children with ASD often are particularly sensitive to medication side effects.

Irritability/aggression/extreme mood lability

There are only two medications with Food and Drug Administration labeling for an autism specific condition, and those are aripiprazole and risperidone, two second-generation antipsychotic agents approved for irritability associated with ASD on the basis of randomized controlled trials (RCTs) demonstrating their efficacy.^3,4 Included under the umbrella of irritability are aggression, deliberate self-injurious behavior, extreme temper tantrums, and quick and extreme mood changes. For aripiprazole the approved ages are 6-17 years; a dosing range of 2-15 mg/day is recommended. For risperidone, the approved age range is 5-17 years; the recommended dosing range is 0.25-4 mg/day. Prior to starting either of these medications, a cardiac history should be obtained, and baseline laboratory values, particularly lipid levels and hemoglobin A_1c (HbA_1c) are recommended. All second-generation antipsychotics carry the risk of tardive dyskinesia (a movement disorder), as well as risk of weight gain and metabolic effects. Baseline weight prior to medication initiation with routine follow-up measurement is encouraged. In light of the burden of potential side effects, these medications tend to be reserved by clinicians for circumstances where there is a significant impact on functioning. Both medications are available in liquid form for children with difficulty swallowing pills.