Evidence-Based Pharmacotherapy for Autism Spectrum Disorders

Autism spectrum disorder (ASD) is an elusively heterogeneous disorder defined by core social impairment and repetitive interests and behaviors. To date, there are no medications approved by the US Food and Drug Administration (FDA) for treatment of the defining features of this disorder. Significant progress has been made in treatment of behavioral symptoms associated with ASD, particularly irritability and hyperactivity symptoms. Risperidone and aripiprazole have the most evidence supporting their use for irritability symptoms and are the appropriate medications to choose as first line of treatment of these symptoms. Methylphenidate and guanfacine hold promise for treatment of hyperactivity symptoms in youth with ASD, based on available evidence. Repetitive behaviors and interests remain highly challenging behavioral domains in ASD, though use of risperidone is supported by the literature, and selective serotonin reuptake inhibitors may be beneficial in some individuals. There is no clear evidence to support any pharmaceutical agent for treatment of core social impairment in ASD. Ongoing research and continued understanding of pathogenic pathways may uncover novel pharmacological targets for treatment of core symptoms in this population.