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Recent Advances in the Pharmacological Management of Behavioral Disturbances Associated with Autism Spectrum Disorder in Children and Adolescents

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Abstract

Autism spectrum disorder (ASD) is a heterogeneous neuropsychiatric condition affecting an estimated one in 36 children. Youth with ASD may have severe behavioral disturbances including irritability, aggression, and hyperactivity. Currently, there are only two medications (risperidone and aripiprazole) approved by the US Food and Drug Administration (FDA) for the treatment of irritability associated with ASD. Pharmacologic treatments are commonly used to target ASD-associated symptoms including irritability, mood lability, anxiety, and hyperactivity. However, evidence for the efficacy of many commonly used treatments is limited by the lack of large placebo-controlled trials of these medications in this population. Research into the pathophysiology of ASD has led to new targets for pharmacologic therapy including the neuroimmune system, the endocannabinoid system, and the glutamatergic neurotransmitter system. The goal of this review is to provide an overview of the current evidence base for commonly used treatments, as well as emerging treatment options for common behavioral disturbances seen in youth with ASD.

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Acknowledgements

The authors acknowledge Nicole Friedman for her efforts editing a previous draft of this article.

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  1. Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, 45229, USA

    Martine Lamy, Ernest V. Pedapati, Kelli L. Dominick, Logan K. Wink & Craig A. Erickson

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  1. Martine Lamy
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  2. Ernest V. Pedapati
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  3. Kelli L. Dominick
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Correspondence to Martine Lamy.

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Conflict of interest

The authors declare that they have no interests that compete directly with this work, though Dr. Lamy, Dr. Dominick, Dr. Wink, Dr. Pedapati, and Dr. Erickson do receive research support from various sources for other work. Dr. Lamy’s current research is supported by OVID Therapeutics and the Simon’s Foundation. Dr. Dominick’s current research is supported by OVID therapeutics and Roche Pharmaceuticals. Dr. Wink’s current and/or past research is supported by the Simons Research Foundation, Autism Speaks, Riovant Sciences Ltd, and Cures Within Reach. She is an inventor on intellectual property held by Cincinnati Children’s Hospital Research Foundation for a treatment in autism spectrum disorder. Dr. Wink has also served as a past consultant for Otsuka and Ovid. Dr. Pedapati receives research support from Cincinnati Children’s Hospital Research Foundation and the National Institutes of Health. Dr. Erickson has received current and/or past research support from the National Institutes of Health, the United States Department of Defense, the United States Centers for Disease Control, the John Merck Fund, Autism Speaks, the Simons Foundation, Cincinnati Children's Hospital Research Foundation, the FRAXA Research Foundation, the National Fragile X Foundation, the Roche Group, Seaside Therapeutics, Novartis, Neuren, Alcobra, and Indiana University School of Medicine. He is a past consultant to Alcobra, the Roche Group, and Novartis. He is a current consultant to Fulcrum Therapeutics. He holds equity interest in and is a consultant for Confluence Pharmaceuticals. He is the inventor on intellectual property held by Cincinnati Children's Hospital Research Foundation and Indiana University describing methods for diagnosis and treatment methods in autism spectrum disorder and fragile X syndrome.

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Lamy, M., Pedapati, E.V., Dominick, K.L. et al. Recent Advances in the Pharmacological Management of Behavioral Disturbances Associated with Autism Spectrum Disorder in Children and Adolescents. Pediatr Drugs 22, 473–483 (2020). https://doi.org/10.1007/s40272-020-00408-0

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