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Evidence-Based Pharmacotherapy for Autism Spectrum Disorders

  • Child and Adolescent Psychiatry (M DelBello, Section Editor)
  • Published:
Current Treatment Options in Psychiatry Aims and scope Submit manuscript

Opinion statement

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.

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Authors and Affiliations

  1. Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, 45229-3026, USA

    Lynn McClellan DNP, CNP, Ernest Pedapati MD, Logan Wink MD & Craig Erickson CMD

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  1. Lynn McClellan DNP, CNP
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  2. Ernest Pedapati MD
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  3. Logan Wink MD
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  4. Craig Erickson CMD
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Corresponding author

Correspondence to Craig Erickson CMD.

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Conflict of Interest

Dr. McClellan has no conflict of interest.

Dr. Wink’s current research is supported by the Simons Research Foundation, Autism Speaks, Riovant Sciences Ltd, and Cures Within Reach. Dr. Wink has served as a past consultant for Otsuka.

Dr. Pedapati receives research support from the Cincinnati Children’s Hospital Research Foundation.

Dr. Erickson is a consultant to and holds equity in Confluence Pharmaceuticals and is a consultant to Neurotrope. Dr. Erickson is a past consultant to Alcobra Pharmaceuticals, the Roche Group, and Novartis. Dr. Erickson holds non-related IP held by CCHMC and Indiana University. Dr. Erickson receives research grant support from the John Merck Fund, Cincinnati Children’s Hospital Medical Center, Autism Speaks, the National Fragile X Foundation, The Roche Group, Neuren Pharmaceuticals, and Riovant Sciences Ltd.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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This article is part of the Topical Collection on Child and Adolescent Psychiatry

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McClellan, L., Pedapati, E., Wink, L. et al. Evidence-Based Pharmacotherapy for Autism Spectrum Disorders. Curr Treat Options Psych 3, 161–181 (2016). https://doi.org/10.1007/s40501-016-0077-6

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