Pharmacological treatment of autism

Abstract

Although there is little evidence that any medication reliably relieves the core symptoms of autism to a major degree, most autistic patients receive some form of pharmacotherapy, much of it off-label, with the primary therapeutic aim being to improve behaviors that interfere with behavioral and educational therapies. Because of the difficulties inherent in conducting randomized, placebo-controlled studies in pediatric populations, evidence of the benefits of this strategy is limited, and most such studies have been small and of short duration. Nevertheless, evidence from controlled studies indicates that the atypical antipsychotic drug risperidone is frequently effective for ameliorating symptoms of irritability, hyperactivity, social withdrawal, and stereotypic, repetitive behavior. There remains uncertainty as to the risk of serious adverse effects with long-term risperidone treatment. Weaker evidence supports the efficacy of antidepressant and stimulant medications, and evidence is ambiguous regarding the use of anticonvulsants as mood stabilizers in autistic patients. Promising results have been reported for a number of other medications, but additional controlled studies are needed to confirm the validity of these reports. There is little evidence that any currently employed pharmacotherapy appreciably improves impaired social interactions or communication in autistic individuals. There remains a great need for research into the long-term benefits and risks of pharmacotherapy in autism.